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Technology Strategy Board
Driving Innovation




Stratified Medicine
in the UK
Vision and Roadmap
Stratified Medicine in the UK




Stratified Medicine in the UK
Vision and Roadmap
Summary                                           Background                                     fewer new drugs. Data shows that, starting
                                                                                                 with 5,000-10,000 candidate molecules,
A consortium of government bodies and             Even the best medicines are not equally        it can take up to 15 years, and cost more
leading charities has joined forces to            effective in all patients. Disease processes   than $800m, to get one new drug to
accelerate the development and uptake of          and treatment choices can vary from            market. The sustainability of this model
stratified medicine in the UK. The                person to person even though they may          of drug development is increasingly
Technology Strategy Board, Medical                have similar symptoms. It is estimated that    being questioned and many suggest that
Research Council (MRC), Cancer                    only 30-70% of patients respond positively     using molecular biology to select likely
Research UK (CRUK), Arthritis Research            to any particular drug. Recent advances in     responders could lead to reduced
UK (ARUK), Department of Health (DH),             science, particularly in molecular biology     development times, fewer failures, and
Scottish Government Health Directorate            and genomics, mean that it will become         lower costs. Diagnostics companies are
(SGHD) and National Institute for Clinical        increasingly possible to identify the          an important part of the solution, but
Health and Excellence (NICE) have                 underlying molecular mechanisms of             developing and commercialising robust
formed a partnership to take forward the          disease. Developing diagnostic tests that      tests can be expensive and difficult.
Stratified Medicine Innovation Platform.          indicate the molecular cause of a disease
Together they will invest around £200m            enables the development of new treatments
over five years in the area of stratified         that can more precisely target the disease,
medicine. Stratified medicine can be              as Herceptin does for certain forms of         Stratified Medicine
summarised as identifying the right               breast cancer. Predicting in advance           Innovation Platform
therapy for the right patient at the right time   which groups of patients will respond to a
in the right dose.                                particular therapy and providing treatment     Global society faces many challenges.
                                                  accordingly is known as stratified             By applying technology and innovation we
This publication sets out a shared vision                                                        can help to meet these challenges and, at
                                                  medicine (or personalised medicine).
for the UK to be the best place to develop,                                                      the same time, open up new opportunities
and have adopted, stratified medicine. It         Across the world, healthcare models are        for business. Innovation platforms focus
also outlines a technology roadmap that           facing greater challenges, both physically     on specific societal challenges where
describes nine thematic areas which, if           and financially, in providing for a growing,   government is taking action through
advanced successfully, will help accelerate       ageing population with an increasing           policy, regulation, procurement or fiscal
the development and uptake of stratified          burden of disease. The upward trend in         measures. By improving co-ordination
medicine in the UK. The vision and                spending means healthcare is becoming          between the key players from industry,
roadmap grew out of a series of workshops          a more and more significant part of a
                                                                                                 academia and government, innovation
attended by more than 100 people                  nation’s GDP, and the long-term
                                                                                                 platforms can identify barriers to meeting
representing a broad range of stakeholders        sustainability of current models of
                                                                                                 the challenge, map possible routes to
                                                  provision is increasingly questioned.
                                                                                                 overcoming the barriers, and align activities
                                                  A stratified approach has the potential
                                                                                                 to support innovative solutions. The aim is
                                                  to address some of these issues and at
                                                                                                 to deliver a step change in the ability of UK
                                                  the same time improve patient outcomes.
                                                                                                 businesses to provide solutions for the
                                                  Costs are increasing too for the supply        global marketplace, boost UK economic
                                                  side. Pharmaceutical R&D productivity          performance, and provide higher-quality,
                                                  is decreasing, meaning higher costs for        better value healthcare services.




02 | Technology Strategy Board
Vision and Roadmap




The Stratified Medicine Innovation Platform     This document presents a summary of the           A Shared Vision
(SMIP) seeks to build on the UK’s strength      views heard from these sessions in the form
within global healthcare industries and put     of a UK Vision for Stratified Medicine in 2025,   The vision is for the UK to be the best
it at the centre of the next generation of      and a roadmap to achieve it. It attempts to       place to develop, and have adopted,
medicine. It is a five-year programme to        capture some of the excellent stratification-     stratified medicine. This will benefit
accelerate the development and uptake of        related activity currently going on in the        patients by improving outcomes, help
stratified products and services. The           UK, but cannot claim to be comprehensive          physicians to select the most appropriate
                                                or an agreed consensus from all of the            therapies, increase the cost-effectiveness
innovation platform is a consortium of
                                                represented groups. The current version of        of health service provision and help
seven partner organisations all working
                                                the roadmap is a starting point. It will          business to innovate more successfully.
towards a common vision. The partners
                                                constantly evolve as new people                   This vision was developed over a series of
are Technology Strategy Board, Medical
                                                contribute to it, and better understanding        workshops which explored what the UK
Research Council (MRC), Cancer Research
                                                is developed. Those interested in engaging        landscape should look like in 2025. The
UK (CRUK), Arthritis Research UK (ARUK),
                                                further with the SMIP are encouraged to join      key aspects of the vision are given in the
Department of Health (DH), Scottish
                                                the special interest group on _Connect            box below:
Government Health Directorate (SGHD)
                                                (https://ktn.innovateuk.org/web/stratified
and National Institute for Health and
                                                -medicines-innovation-platform).
Clinical Excellence (NICE). Each of these
partners in the programme management
group serves a different constituency but
each has agreed to align activities towards        A UK Vision for Stratified Medicine
a common goal with benefits for all                K The UK should be the best place to develop, and have adopted, stratified
stakeholders. Together they will invest              medicine. This will benefit patients, provide cost-effective solutions for the NHS
around £200m in stratified medicine.                 and other healthcare providers and create opportunities for business
                                                   K There should be an increased collaborative culture throughout the sector based
Achieving the rapid development and
                                                     around shared resources, and systems should be in place for effective data
uptake of stratified medicine in the UK will
                                                     collection, sharing, governance and use across sectors (including NHS,
be a difficult and complex task. It needs all
                                                     business, academia, regulators and NICE)
components of the UK infrastructure to
work together around a common plan and             K It should be quicker and less expensive to develop new drug-diagnostic
                                                     combinations and have them licensed, and success should be reflected in
towards a common goal. The SMIP has
                                                     increased UK economic growth
started the process of building a shared
vision for stratified medicine in the UK           K It should be possible for all NHS patients to be involved in medical research if
and a plan to achieve it. An early statement         they wish, including through use of patient information and records, in order to
of a UK vision was refined and validated             inform the next generation of successful therapies
through widespread community                       K There should be a smooth reimbursement process for stratified therapies and
engagement, including through workshops              diagnostics, and an intellectual property (IP) framework that encourages innovation
where more than a hundred people from              K The UK health system should have established stratified care pathways, and
more than 70 organisations helped to                 evidence should be available to show that patient outcomes are improved where
shape and refine the vision and plan.                stratified medicine is used




                                                                                                               Technology Strategy Board | 03
Stratified Medicine in the UK




A Shared Roadmap                                                            The UK Stratified Medicine Roadmap
                                              The UK Stratified             contains a panel for each of these themes.
A technology roadmap identifies the                                         Each panel describes how things are
long-term objectives in an area and seeks
                                              Medicine Roadmap:
                                                                            perceived to be now and articulates a
to understand the possible routes to          The Nine Themes               vision of how they might be in 2025. Some
satisfy them. It can be used to guide the                                   of the barriers to achieving this vision are
                                              1. Incentivising adoption
development of new products, processes                                      presented and activities that would
or services, and to help shape the future     2. Increasing awareness       accelerate the development of stratification
direction of an emerging technology.                                        are proposed. Some of the excellent work
                                              3. Patient recruitment –
Developing a roadmap has three major                                        already in train is highlighted. Many topics
                                                 consents and ethics
uses. It helps reach a consensus about a                                    are closely interlinked and some occur in
set of needs and identifies a range of        4. Clinical trials            more than one panel: for a stratified
possible solutions to satisfy them; it                                      medicine system to work effectively in the
provides a mechanism to help forecast the     5. Data – collection,
                                                                            UK, many of these issues will have to be
required deployment of those solutions;          management and use         progressed at the same time. The
and it provides a framework to help plan      6. Regulation and standards   Stratified Medicine Innovation Platform on
and coordinate technology and system                                        its own will not be able to achieve
developments.                                 7. Intellectual property      everything that needs to be done, but an
The stratified medicine roadmap describes     8. Bio-banks and biomarkers   attempt has been made to show what our
thematic areas which, if advanced                                           own contributions might be. In order to
                                              9. Increasing the impact      save space in the following panels,
successfully, will help accelerate the
                                                 of R&D investment          acronyms are used. These are detailed in
development and uptake of stratified
medicine in the UK. The themes were                                         a glossary at the end of the document.
identified at a series of workshops
attended by a broad stakeholder
community that included commercial
companies developing therapeutics,
diagnostics and data handling solutions;
medical charities; and representatives
from the healthcare sector including
clinicians from across a number of
therapeutic areas, pathologists,
commissioners, managers and
representatives of public sector bodies.
In addition, there were academics
representing research in all of the main
topics around stratification of patients.
The roadmap captures the range of views
expressed by this broad group of
stakeholders. It calls for activity in nine
thematic areas.




04 | Technology Strategy Board
1. Incentivising adoption of SM in the NHS
                                                                                                              Barriers
                      Reimbursement mechanism for SM related Dx tests not             Budgets and processes in NHS not designed for                  A smooth integrated system: fair & beneficial to all
                      always clear leading to unequal provision                       diagnostic/drug combinations                                   stakeholders
Current Perceptions



                      Slow test adoption - commissioning structure a barrier          Only few clinical decisions based on genetic tests             Equitable access to the right tests for the right patients
                                                                                                                                                     at the right time




                                                                                                                                                                                                                  Future State
                      Infrastructure is not equally distributed and may be            Uncertain markets for point of care testing vs.
                      sub-scale in some locations                                     centralised testing                                            Infrastructure available to all with equal access to
                                                                                                                                                     treatments
                      Differing requirements for in-house tests compared to           Dx companies can struggle to provide data for
                      industry developed tests                                        independent NICE assessment                                    Tests reimbursed at a level that is cost effective and
                                                                                                                                                     incentivises new development
                      Current NHS genetics policy and services not                    Uncertain pathology capability in emerging techniques
                      designed for large-scale testing                                                                                               Patient/public engagement and acceptance
                                                                                      Aggressive national pathology cost savings may
                      Pathology budgets stretched and not linked to care              discourage adoption of new techniques                          Informed commissioning (quality, cost, turnaround etc)
                      pathways
                                                                                      No long-term strategic planning in NHS for SM services
                      Slow adoption of tests without Rx company support
                                                                                                              Enablers
                                                                       Overall framework development by             NICE diagnostics arm is an enabler for
                                                                       Human Genomics Strategy Group                value assessment
                                                                       Enhancing NHS informatics capability         National commissioning or funding for
                                                                                                                    stratified medicine, both tests and
                                                                       Increasing evidence base leading to
                                                                                                                    treatments
                                                                       more widespread clinical acceptance of
                                                                       the value of genetic tests                   Value assessment of Dx/Rx combinations
                                                                                                                    to inform commissioning
                                                                       Minimally-invasive biomarker tests




  Examples of current UK activity                                                                                                           Potential Role of SMIP
  NHS/DH initiative to improve cancer gene testing                                                                                          Help provide evidence to support policy development for
  HGSG review of commissioning, innovation and service delivery in all genetic medicine                                                     commissioning and procurement
  Ability to use Cancer Drug Fund to buy associated complex diagnostics                                                                     Help supply chain to develop new business models
  Value-based pricing review of drug procurement in NHS                                                                                     Support health economic assessment of diagnostic testing to inform
  SMIP/CRUK SMP large-scale demonstrator programme in cancer                                                                                NHS pricing
  Pathology modernisation                                                                                                                   Support CRUK SMP and other demonstrators by funding associated
  Nicholson Review of Innovation in the NHS                                                                                                 technology development

  OLS Senior Industry Group addressing business-NHS engagement                                                                              Interim support for innovative molecular diagnostics in adoption phase
  NIHR/NOCRI work to improve access for clinical trials                                                                                     Help represent businesses in NHS policy/commissioning development
  Trade Associations (ABPI, BIVDA, ABHI, etc.) work with NHS on new commissioning structures
2. Increasing stakeholder awareness
                                                                                                               Barriers
                      Evidence base not conclusive in all therapeutic areas           Lack of public awareness of the complexity of                     Patients understand stratification and accept that
                                                                                      gene-medicine interactions                                        treatment access may depend on a complex test
                      Some patients may see stratification as a method
Current Perceptions



                      of rationing                                                    Lack of high-quality, clinically approved, education              Stratification tests included in education materials
                                                                                      material for patients and practitioners                           around patient care pathway




                                                                                                                                                                                                                 Future State
                      Variable knowledge of how to apply a test in
                      clinical practice                                               Pace of change of evidence base                                   Appropriate testing for multiple biomarkers
                                                                                                                                                        incorporated in clinical care pathway
                      Emerging models for clinical decision support do                The difficulty of integrating clinical imaging and
                      not always convince clinicians                                  laboratory data
                      Test usage inconsistent across providers




                                                                                                              Enablers
                                              More awareness among front line staff of clinical utility and benefits         Development of tests for use at point of prescription (pharmacy/GP) or
                                                                                                                             patients able to test themselves
                                              Agreement and sharing of genetic medicine health economic analyses
                                                                                                                             Centralised communication resource to suit a range of health professionals
                                              Tools for physicians to provide clear interpretation of complex test results
                                                                                                                             Better engagement with patient support groups in disease specific areas
                                              Physician-driven development of education materials for healthcare
                                              professionals involved in genetic medicine                                     Patient access to global communication channels




  Examples of current UK activity                                                                                                              Potential Role of SMIP
  Education working group of the HGSG                                                                                                          Develop decision-making tools for physicians
  Various bodies already developing guidance materials                                                                                         Develop communication resource for education of health
                                                                                                                                               professionals
  Appointment of NHS Chair in Pharmacogenetics
                                                                                                                                               Involve patient support groups and Royal Colleges in SMIP
  CRUK large-scale demonstrator in cancer
                                                                                                                                               programme development
                                                                                                                                               Accelerate and communicate programmes that demonstrate validity
                                                                                                                                               of approach
                                                                                                                                               Help extend approach into other therapeutic areas
3. Patient recruitment (consents & ethics)
                                                                                                          Barriers
                      Identifying and recruiting appropriate patient cohorts        Data held in a range of databases and formats across            A streamlined process for trials consent and patient
                      can be time-consuming                                         healthcare providers                                            recruitment, and trials are safe
Current Perceptions



                      R&D approval process can be complex and time-                 Data quality may be low in data that has not been               Consent for broad and unspecified future use,
                      consuming                                                     monitored and audited                                           supported by appropriate regulation of personal data




                                                                                                                                                                                                             Future State
                      When patient consents are study or site-specific, data        Inconsistent processes and standards for research               Single, and internationally valid, consent and ethics
                      cannot be used for other purposes                             consents                                                        approval
                      Sometimes local ethics and R&D approval process can           Privacy concerns around data storage and use                    Primary care has important role in patient recruitment
                      inhibit larger national studies
                                                                                    Excessive caution from some (unrepresentative)                  Use of data and samples for research based on
                      Problems with national programme for IT has held back         minority pressure groups                                        ‘opt-out’ rather than ‘opt-in’
                      novel data solutions




                                                                                                          Enablers
                           Structure of NHS gives unique opportunity to deliver standardised research consent    Implementation of Academy of Medical Sciences recommendations on streamlining
                                                                                                                 research regulation
                           Novel IT solutions for capturing, communicating, storing and analysing data
                                                                                                                 Connection of ethics committee with patient groups, starting with current disease
                           Public engagement about use of samples and genetic data for research
                                                                                                                 areas of focus like cancer
                           Development of an overarching ethical framework
                                                                                                                 Policy communication, IT enablement & governance.
                           Cost of storing massive data sets decreasing
                                                                                                                 Education and public awareness campaign demonstrating wider benefits to the
                           Patient groups support non-restrictive consents and effective R&D approvals           community of participation in research
                           Development and widespread use of generic consent forms                               Collaboration in clinical trials recognised in clinician performance assessment


  Examples of current UK activity                                                                                                          Potential Role of SMIP

  Academy of Medical Science report and recommendations                                                                                    Engage with patient groups on programme development and
                                                                                                                                           benefits of consent
  Leading hospitals already running generic consent for all patient samples
                                                                                                                                           Support Government implementation of AMS recommendations
  HGSG working on this area
                                                                                                                                           Consulting with stakeholders on routine/generic consent
  Plan for Growth actions on research approvals and recruitment to studies
  NIHR Translational Research Partnerships process for research consent in inflammatory disease
  Various data access and sharing initiatives (Generation Scotland, Cancer Registries, General
  Practice Research Database)
  Improved national and international data standards are enabling data sharing
4. Clinical Trials
                                                                                                                   Barriers
                      Localisation of commissioning and independence of                 Uncertain impact of localisation agenda in healthcare                 Reduced time and cost to conduct clinical trials
                      providers leads to varied and complex implementation              and changes in commissioning
                                                                                                                                                              Reduced time to start complex genetic research
Current Perceptions



                      of national clinical trials
                                                                                        Relative higher prices for trial activities in UK versus              projects
                      NIHR involve patients group                                       BRIC countries




                                                                                                                                                                                                                       Future State
                                                                                                                                                              Reduced cost to recruit the appropriate subjects to
                      Networks such as NCRI encourage and enable                                                                                              enable stratified medicine
                      national trials
                                                                                                                                                              All patients are able to participate in clinical
                      Delays before patient recruitment starts is often the                                                                                   trials/population-based genomic research
                      time-limiting step in studies
                                                                                                                                                              Centralised solutions to access rare genetic types
                      AMS report describes the situation well                                                                                                 (e.g. web-based platform)
                                                                                                                                                              Exclusion of inappropriate patient populations on
                                                                                                                                                              grounds of safety genotypes
                                                                                                                                                              Selection of appropriate patients for efficacy testing
                                                                                                                   Enablers
                                                                          AMS report and recommendations                Develop robust online (internet)
                                                                                                                        recruitment
                                                                          National initiatives to improve trials
                                                                          environment (CRUK, NOCRI, etc)                Develop standards within NHS for
                                                                                                                        provision of patients into clinical trials
                                                                          Public increasing their levels of
                                                                          participation in research                     Better connection of infrastructure
                                                                                                                        including IT systems and Biobanks
                                                                          Faster ethics approval based on
                                                                          patient choice and critical health ‘need’




  Examples of current UK activity                                                                                                                   Potential Role of SMIP
  NIHR Clinical Research Networks, including cancer, could help in stratified medicine                                                              Similar to role in patient recruitment
  NOCRI helping industry engagement with NIHR trials infrastructure                                                                                 Help create critical mass to accelerate trials process
  NIHR common frameworks and guidelines to reduce patient recruitment times                                                                         Communicate unmet needs (prioritisation)
                                                                                                                                                    Demonstration of a consistent database of patient accessible
                                                                                                                                                    records.
                                                                                                                                                    Develop web-based recruitment tools
5. Data
                                                                                                            Barriers
                      Routine data collection and quality variable across the        Morale issues following NPfIT failure                             Population-wide consented patient data available for
                      UK, better in Scotland                                                                                                           analysis by researchers
                                                                                     Most data has not been consented for access
Current Perceptions



                      NHS Information Centre as central repository and                                                                                 Routine use of anonymised patient data sets by NHS &
                                                                                     Default position is to not make anonymised data
                      access point for data but currently not meeting                                                                                  industry to improve patient outcomes




                                                                                                                                                                                                                  Future State
                                                                                     available for research
                      researcher requirements
                                                                                                                                                       Increased use of routine data in post-marketing
                                                                                     Bespoke systems do not or cannot communicate
                      Government data transparency drive (opengov)                                                                                     surveillance
                                                                                     without significant investment
                      No national programme for IT                                                                                                     Routine collection of health economic data around SM
                                                                                     Data protection issues seen as a barrier within trusts
                      Range of IT systems in use




                                                                                                            Enablers
                                                     Massive data sets                                             Implement IT practices and routine generic consent for use
                                                                                                                   of appropriately anonymised clinical data for research
                                                     Public support for research
                                                                                                                   between institutions
                                                     IT capabilities improving
                                                                                                                   Large-scale demonstrator programmes (regional or
                                                     Encourage more open working between academia,                 therapeutic-area focused)
                                                     NHS & industry
                                                                                                                   Simplification of data protection rules around patient data
                                                     Cultural/policy encouragement to share data
                                                                                                                   Build in and embed quality systems into NHS data handling




  Examples of current UK activity                                                                                                             Potential Role of SMIP
  Generation Scotland                                                                                                                         Develop tools to allow secure data mining of patient data across
                                                                                                                                              distributed data sources
  Individual routine consents
                                                                                                                                              Demonstrate that access to data repository by NHS & industry will
  CRUK/Eastern Cancer Registry partnership & SMIP investment in data handling
                                                                                                                                              improve patient outcomes and/or make UK a much better place to
  Ongoing improvements to cancer registries                                                                                                   do research
  Establishment of Patient Reported Outcome Measures (PROMS) by Dept Health in England
6. Regulatory Framework and Development Pathway
                                                                                                           Barriers
                      It can cost over $800m to take a safe and                    Lack of smart and adaptive study designs                         Novel design of clinical trials makes smaller cohorts of
                      effective drug to market, and take 7-9 years from                                                                             patients possible
                                                                                   Lack of a single body controlling quality of
Current Perceptions



                      first in-man trials
                                                                                   diagnostic labs                                                  Coordinated regulation of pharmaceuticals industry
                      New drugs sometimes only marginally better than                                                                               (Rx) and diagnostic industry (Dx)




                                                                                                                                                                                                               Future State
                                                                                   Need for regulatory guidance development for
                      standard care but more expensive
                                                                                   drug-diagnostic combinations                                     Companies make more progress within existing
                      Different degrees of regulation for pharmaceuticals,                                                                          regulatory framework
                      diagnostic tests and laboratories/hospitals




                                                                                                           Enablers
                                                                     Regulatory guidance for co-development       Facilitate co-development of drugs and
                                                                     of diagnostics and treatment                 diagnostics by alignment of the regulatory
                                                                                                                  processes
                                                                     DH and BIS encouraging EU Commission
                                                                     to improve regulatory framework              Setting up of an expert committee
                                                                                                                  (akin to the Committee for Advanced
                                                                                                                  Therapies set up by the ATMP Regulation)




  Examples of current UK activity                                                                                                         Potential Role of SMIP
  EU Commission on the revision of the IVD directive                                                                                      Help in novel clinical study design
  NHS QIPP agenda includes work on quality and standards                                                                                  Encourage discussion with regulatory agencies
7. Intellectual Property
                                                                                                                  Barriers
                      Laboratory-developed tests using unlicensed IP in NHS                Questions around effective regulation of laboratory-             IP with enhanced licensing for NHS use acts as a driver
                      discourages development of new diagnostic tests by                   developed tests                                                  of ‘step change’ innovation
Current Perceptions



                      industry
                                                                                           No single approach to NHS licensing of test technology           Publicly funded labs are free to develop safe
                      Laboratory-developed tests can sometimes save the                                                                                     and effective tests that do not infringe on other




                                                                                                                                                                                                                      Future State
                                                                                           Patent thickets – multiple IP on sequence reduces
                      NHS money                                                                                                                             IP rights-holders
                                                                                           economic attractiveness of panel testing
                      Some NHS laboratory-developed tests are being                                                                                         Competition between commercial and laboratory-
                      technically and clinically validated and externally quality                                                                           developed tests benefits patients
                      assured
                      Current IP for gene sequences (BRCA/Myriad etc)
                      subject to legal challenge




                                                                                                                  Enablers
                                                                Patent box tax incentive                                 Develop IP protection on the test, not the
                                                                                                                         underlying gene sequence
                                                                Excellent technology platform & biomarker
                                                                science base                                             Provide a strong home market (NHS) with value
                                                                                                                         based pricing (product & service) and willingness
                                                                Business models for developing & exploiting new
                                                                                                                         to adopt new tests
                                                                & existing IP that provides fair return on
                                                                investment whilst maintaining reasonable end             A stronger domestic industry with global outlook,
                                                                user cost                                                able to capitalise on excellent science base




  Examples of current UK activity                                                                                                                 Potential Role of SMIP
  EU In-Vitro Diagnostic consultation in process                                                                                                  Provide support for diagnostic companies and co-development
  OECD guidelines for licensing genetic inventions                                                                                                Support SMEs in early IP filing
  HGC recommendations on IP and DNA diagnostics                                                                                                   Commission health economics studies
                                                                                                                                                  Support development of new business models and pricing
                                                                                                                                                  mechanisms
8. Bio-Banks and Early Biomarker Detection
                                                                                                             Barriers
                      No central overarching database of biomarkers or               Localised clinical and research databases are hard to            UK-wide database of biobanks is used for
                      biosignatures                                                  use for national level research                                  stratification studies
Current Perceptions



                      Many biosamples not clinically annotated                       Lack of technology platforms for use in clinical biomarker       All tissue collected with consent for broad clinical
                                                                                     research                                                         research and data capture




                                                                                                                                                                                                                Future State
                      Limited standardisation of tissue collection or consent
                                                                                     Require policy framework for biomarker validation                Routine, effective collaboration on biomarker ID
                      Not everyone sees the value of centralised biobanks
                                                                                     Many biomarkers do not have associated therapies                 A range of early biomarkers identified for future
                      Stand-alone, incompatible tissue and data
                                                                                                                                                      validation
                      infrastructures
                                                                                                                                                      Coordination for Biomarker research via mandated
                      UK Biobank has recruited 0.5m people
                                                                                                                                                      central database
                                                                                                                                                      In silico trials to ID biomarker



                                                                                                             Enablers
                                                          Accreditation system for biobanks                        Improved biomarker discovery and validation through
                                                                                                                   new approaches
                                                          Improve coordination of funding for biobanks
                                                                                                                   Linking of databases and biobanks
                                                          Standardisation of procedures, including sample
                                                          handling storage and consent process                     Improving IT to enable metadata approach across
                                                                                                                   multiple systems
                                                          Ensuring biobanks store comprehensive clinical data
                                                                                                                   Increase in pharma collaboration and pre-competitive
                                                          Broad & enduring consent or implement opt-out
                                                                                                                   activity
                                                          rather than opt-in for data analysis in research



  Examples of current UK activity                                                                                                            Potential Role of SMIP
  UK Biobank programme standardising access procedures                                                                                       Help develop standards for biobanks
  Generation Scotland                                                                                                                        Map existing UK databases and biobanks
  Routine generic consent at sample-acquisition in major teaching hospitals                                                                  Support development of sample handling & preservation technologies
  NIHR/NOCRI Translational Research Partnerships                                                                                             Support development of biosignature analysis and data storage
                                                                                                                                             techniques
                                                                                                                                             Encourage widespread adoption of best practice
                                                                                                                                             Help connect existing infrastructure to create critical mass
                                                                                                                                             Enable access to infrastructure to accelerate development of new tests
                                                                                                                                             and therapies
9. Increasing and Leveraging UK R&D investment
                                                                                                              Barriers
                      Developing new drugs has becoming increasingly                  Different parts of the system have different objectives            Taking drugs from discovery phase to
                      expensive                                                                                                                          commercialisation is faster and cheaper, enhancing
                                                                                      Bridging the gap between research and commercial
Current Perceptions



                                                                                                                                                         business performance and encouraging investment
                      Pharmaceutical businesses are re-evaluating their R&D           exploitation
                      investments                                                                                                                        Stratified medicine offers improved value for money for




                                                                                                                                                                                                                   Future State
                                                                                      The RAE/REF do not incentivise academics to help
                                                                                                                                                         customers including NHS
                      Turbulence in financial markets has impacted                    commercialisation
                      availability of equity and debt finance                                                                                            Improved co-ordination of public and private funding
                                                                                      The sector is extremely large and complex, so major
                      Public sector funders have not always aligned their             change is non-trivial
                      activities
                      University funding is changing
                      NHS needs to save money



                                                                                                             Enablers
                                                                      Creating a coordinated UK programme           Better coordinated UK and EU funding
                                                                                                                    streams
                                                                      Incentivise stakeholders to share
                                                                      pre-competitive data                          Understand the value of cooperative
                                                                                                                    investment
                                                                      Stakeholder support for SM
                                                                                                                    Taking an international perspective
                                                                      Full audit of UK capabilities /IT,
                                                                      biomarkers, Dx, Rx, clinical etc




  Examples of current UK activity                                                                                                               Potential Role of SMIP
  ABPI work in this area                                                                                                                        Commission health economic studies
  OSCHR work in this area                                                                                                                       Help develop new business models for stratified medicine
  Nicholson Review of innovation in the NHS                                                                                                     Ensure coordination of partner activities
  NorthWest exemplar                                                                                                                            Help create critical mass through a UK-wide programme
  Stratified Medicine Innovation Platform investments to date                                                                                   Engage in international programmes
Stratified Medicine in the UK




Stratified Medicine                            The SMIP programme management group
                                               would greatly welcome input from other
Innovation Platform                            players. The Medicines and Healthcare
Partners                                       products Regulatory Agency (MHRA) and
                                               the National Institute for Health Research
The innovation platform seeks to make the      (NIHR)/ NIHR Office for Clinical Research
UK the best place to develop, and have         Infrastructure (NOCRI) have already
adopted, stratified medicine products and      started working closely with the innovation
services. This is an extremely ambitious       platform, and it is hoped that others would
vision that will require activities across     also like to participate. There will be areas
multiple technology areas, policy streams      where others are better placed to lead,
and government departments. Each of the        and we can support their programmes.
Stratified Medicine Innovation Platform
partners has a specific remit and can
contribute significant resources and
expertise from their core areas of
                                               How to engage with us
capability. Together, the group covers a       For the UK to achieve the vision set out in
broad spectrum of interests. By aligning       this document, significant advancements
activities, many of the issues described       in technology and changes to systems
above can be progressed.                       and processes will be required. This
                                               cannot be achieved by any single
The Technology Strategy Board aims to          organisation and will entail coordinated
help businesses to develop innovative new      activity across the entire healthcare sector.
products and services, while the Medical       This UK Stratified Medicine Vision and
Research Council funds outstanding             Roadmap can form the focal point for
research in the science base and has a         companies, public sector bodies and
major initiative in stratified medicine.       government to coordinate their efforts.
Cancer Research UK is the world’s leading      The innovation platform partners have
cancer charity dedicated to saving lives       begun this coordinated activity and would
through research, and Arthritis Research       welcome help and advice from others
UK has an interest in achieving better         whose actions could help accelerate the
outcomes for patients in its disease area:     journey towards the ultimate goal.
both have stratified medicine at the centre    A web-based community has been
of their strategies. The Department for        created to encourage engagement
Health and Scottish Government Health          (https://ktn.innovateuk.org/web/stratified
Directorate are government departments         -medicines-innovation-platform).
whose main role is to improve health and       This will be supported by the innovation
achieve better health, better care, and        platform and will highlight future
better value for all. The National Institute   workshops, funding calls and ongoing
for Health and Clinical Excellence is          discussions on specific stratified-
tasked with assessing medical                  medicine-related topics. We would also
interventions, both therapies and              encourage others to use this forum to share
diagnostics, and recommending their use        information, raise awareness, identify
based on value calculations.                   partners and generate yet more momentum.




014 | Technology Strategy Board
Vision and Roadmap




Glossary
ABHI       Association of British Healthcare Industries
ABPI       Association of British Pharmaceutical Industry
AMS        Academy of Medical Sciences
ATMP       Advanced Therapy Medicinal Products
BIS        UK Department for Business, Innovation & Skills
BIVDA      British In-vitro Diagnostic Association
BRIC       Brazil, Russia, India and China
CRUK       Cancer Research UK
CRUK SMP   Cancer Research UK Stratified Medicine Programme
DH         Department of Health
Dx         diagnostic
HGC        Human Genetics Commission
HGSG       Human Genome Strategy Group
IP         intellectual property
IT         information technology
IVD        in-vitro diagnostic
NCRI       National Cancer Research Institute
NHS        National Health Service
NICE       National Institute for Health and Clinical Excellence
NIHR       National Institute for Health Research
NOCRI      NIHR Office for Clinical Research Infrastructure
NPfIT      National Programme for IT
OECD       Organisation for Economic Co-operation and Development
OLS        Office of Life Sciences
OSCHR      Office for Strategic Co-ordination of Health Research
PROMs      patient reported outcome measures
QIPP       quality, innovation, productivity and prevention
R&D        research and development
RAE/REF    Research Assessment Exercise/Research Excellence Framework
Rx         therapeutic
SM         stratified medicine
SMIP       Stratified Medicine Innovation Platform




                                                                        Technology Strategy Board | 015
Stratified Medicine in the UK




     Technology Strategy Board
     North Star House
     North Star Avenue
     Swindon
     SN2 1UE

     Telephone: 01793 442700

     www.innovateuk.org

     © Technology Strategy Board October 2011 T11/065



016 | Technology Strategy Board

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Technology Strategy Board Vision and Roadmap

  • 1. Technology Strategy Board Driving Innovation Stratified Medicine in the UK Vision and Roadmap
  • 2. Stratified Medicine in the UK Stratified Medicine in the UK Vision and Roadmap Summary Background fewer new drugs. Data shows that, starting with 5,000-10,000 candidate molecules, A consortium of government bodies and Even the best medicines are not equally it can take up to 15 years, and cost more leading charities has joined forces to effective in all patients. Disease processes than $800m, to get one new drug to accelerate the development and uptake of and treatment choices can vary from market. The sustainability of this model stratified medicine in the UK. The person to person even though they may of drug development is increasingly Technology Strategy Board, Medical have similar symptoms. It is estimated that being questioned and many suggest that Research Council (MRC), Cancer only 30-70% of patients respond positively using molecular biology to select likely Research UK (CRUK), Arthritis Research to any particular drug. Recent advances in responders could lead to reduced UK (ARUK), Department of Health (DH), science, particularly in molecular biology development times, fewer failures, and Scottish Government Health Directorate and genomics, mean that it will become lower costs. Diagnostics companies are (SGHD) and National Institute for Clinical increasingly possible to identify the an important part of the solution, but Health and Excellence (NICE) have underlying molecular mechanisms of developing and commercialising robust formed a partnership to take forward the disease. Developing diagnostic tests that tests can be expensive and difficult. Stratified Medicine Innovation Platform. indicate the molecular cause of a disease Together they will invest around £200m enables the development of new treatments over five years in the area of stratified that can more precisely target the disease, medicine. Stratified medicine can be as Herceptin does for certain forms of Stratified Medicine summarised as identifying the right breast cancer. Predicting in advance Innovation Platform therapy for the right patient at the right time which groups of patients will respond to a in the right dose. particular therapy and providing treatment Global society faces many challenges. accordingly is known as stratified By applying technology and innovation we This publication sets out a shared vision can help to meet these challenges and, at medicine (or personalised medicine). for the UK to be the best place to develop, the same time, open up new opportunities and have adopted, stratified medicine. It Across the world, healthcare models are for business. Innovation platforms focus also outlines a technology roadmap that facing greater challenges, both physically on specific societal challenges where describes nine thematic areas which, if and financially, in providing for a growing, government is taking action through advanced successfully, will help accelerate ageing population with an increasing policy, regulation, procurement or fiscal the development and uptake of stratified burden of disease. The upward trend in measures. By improving co-ordination medicine in the UK. The vision and spending means healthcare is becoming between the key players from industry, roadmap grew out of a series of workshops a more and more significant part of a academia and government, innovation attended by more than 100 people nation’s GDP, and the long-term platforms can identify barriers to meeting representing a broad range of stakeholders sustainability of current models of the challenge, map possible routes to provision is increasingly questioned. overcoming the barriers, and align activities A stratified approach has the potential to support innovative solutions. The aim is to address some of these issues and at to deliver a step change in the ability of UK the same time improve patient outcomes. businesses to provide solutions for the Costs are increasing too for the supply global marketplace, boost UK economic side. Pharmaceutical R&D productivity performance, and provide higher-quality, is decreasing, meaning higher costs for better value healthcare services. 02 | Technology Strategy Board
  • 3. Vision and Roadmap The Stratified Medicine Innovation Platform This document presents a summary of the A Shared Vision (SMIP) seeks to build on the UK’s strength views heard from these sessions in the form within global healthcare industries and put of a UK Vision for Stratified Medicine in 2025, The vision is for the UK to be the best it at the centre of the next generation of and a roadmap to achieve it. It attempts to place to develop, and have adopted, medicine. It is a five-year programme to capture some of the excellent stratification- stratified medicine. This will benefit accelerate the development and uptake of related activity currently going on in the patients by improving outcomes, help stratified products and services. The UK, but cannot claim to be comprehensive physicians to select the most appropriate or an agreed consensus from all of the therapies, increase the cost-effectiveness innovation platform is a consortium of represented groups. The current version of of health service provision and help seven partner organisations all working the roadmap is a starting point. It will business to innovate more successfully. towards a common vision. The partners constantly evolve as new people This vision was developed over a series of are Technology Strategy Board, Medical contribute to it, and better understanding workshops which explored what the UK Research Council (MRC), Cancer Research is developed. Those interested in engaging landscape should look like in 2025. The UK (CRUK), Arthritis Research UK (ARUK), further with the SMIP are encouraged to join key aspects of the vision are given in the Department of Health (DH), Scottish the special interest group on _Connect box below: Government Health Directorate (SGHD) (https://ktn.innovateuk.org/web/stratified and National Institute for Health and -medicines-innovation-platform). Clinical Excellence (NICE). Each of these partners in the programme management group serves a different constituency but each has agreed to align activities towards A UK Vision for Stratified Medicine a common goal with benefits for all K The UK should be the best place to develop, and have adopted, stratified stakeholders. Together they will invest medicine. This will benefit patients, provide cost-effective solutions for the NHS around £200m in stratified medicine. and other healthcare providers and create opportunities for business K There should be an increased collaborative culture throughout the sector based Achieving the rapid development and around shared resources, and systems should be in place for effective data uptake of stratified medicine in the UK will collection, sharing, governance and use across sectors (including NHS, be a difficult and complex task. It needs all business, academia, regulators and NICE) components of the UK infrastructure to work together around a common plan and K It should be quicker and less expensive to develop new drug-diagnostic combinations and have them licensed, and success should be reflected in towards a common goal. The SMIP has increased UK economic growth started the process of building a shared vision for stratified medicine in the UK K It should be possible for all NHS patients to be involved in medical research if and a plan to achieve it. An early statement they wish, including through use of patient information and records, in order to of a UK vision was refined and validated inform the next generation of successful therapies through widespread community K There should be a smooth reimbursement process for stratified therapies and engagement, including through workshops diagnostics, and an intellectual property (IP) framework that encourages innovation where more than a hundred people from K The UK health system should have established stratified care pathways, and more than 70 organisations helped to evidence should be available to show that patient outcomes are improved where shape and refine the vision and plan. stratified medicine is used Technology Strategy Board | 03
  • 4. Stratified Medicine in the UK A Shared Roadmap The UK Stratified Medicine Roadmap The UK Stratified contains a panel for each of these themes. A technology roadmap identifies the Each panel describes how things are long-term objectives in an area and seeks Medicine Roadmap: perceived to be now and articulates a to understand the possible routes to The Nine Themes vision of how they might be in 2025. Some satisfy them. It can be used to guide the of the barriers to achieving this vision are 1. Incentivising adoption development of new products, processes presented and activities that would or services, and to help shape the future 2. Increasing awareness accelerate the development of stratification direction of an emerging technology. are proposed. Some of the excellent work 3. Patient recruitment – Developing a roadmap has three major already in train is highlighted. Many topics consents and ethics uses. It helps reach a consensus about a are closely interlinked and some occur in set of needs and identifies a range of 4. Clinical trials more than one panel: for a stratified possible solutions to satisfy them; it medicine system to work effectively in the provides a mechanism to help forecast the 5. Data – collection, UK, many of these issues will have to be required deployment of those solutions; management and use progressed at the same time. The and it provides a framework to help plan 6. Regulation and standards Stratified Medicine Innovation Platform on and coordinate technology and system its own will not be able to achieve developments. 7. Intellectual property everything that needs to be done, but an The stratified medicine roadmap describes 8. Bio-banks and biomarkers attempt has been made to show what our thematic areas which, if advanced own contributions might be. In order to 9. Increasing the impact save space in the following panels, successfully, will help accelerate the of R&D investment acronyms are used. These are detailed in development and uptake of stratified medicine in the UK. The themes were a glossary at the end of the document. identified at a series of workshops attended by a broad stakeholder community that included commercial companies developing therapeutics, diagnostics and data handling solutions; medical charities; and representatives from the healthcare sector including clinicians from across a number of therapeutic areas, pathologists, commissioners, managers and representatives of public sector bodies. In addition, there were academics representing research in all of the main topics around stratification of patients. The roadmap captures the range of views expressed by this broad group of stakeholders. It calls for activity in nine thematic areas. 04 | Technology Strategy Board
  • 5. 1. Incentivising adoption of SM in the NHS Barriers Reimbursement mechanism for SM related Dx tests not Budgets and processes in NHS not designed for A smooth integrated system: fair & beneficial to all always clear leading to unequal provision diagnostic/drug combinations stakeholders Current Perceptions Slow test adoption - commissioning structure a barrier Only few clinical decisions based on genetic tests Equitable access to the right tests for the right patients at the right time Future State Infrastructure is not equally distributed and may be Uncertain markets for point of care testing vs. sub-scale in some locations centralised testing Infrastructure available to all with equal access to treatments Differing requirements for in-house tests compared to Dx companies can struggle to provide data for industry developed tests independent NICE assessment Tests reimbursed at a level that is cost effective and incentivises new development Current NHS genetics policy and services not Uncertain pathology capability in emerging techniques designed for large-scale testing Patient/public engagement and acceptance Aggressive national pathology cost savings may Pathology budgets stretched and not linked to care discourage adoption of new techniques Informed commissioning (quality, cost, turnaround etc) pathways No long-term strategic planning in NHS for SM services Slow adoption of tests without Rx company support Enablers Overall framework development by NICE diagnostics arm is an enabler for Human Genomics Strategy Group value assessment Enhancing NHS informatics capability National commissioning or funding for stratified medicine, both tests and Increasing evidence base leading to treatments more widespread clinical acceptance of the value of genetic tests Value assessment of Dx/Rx combinations to inform commissioning Minimally-invasive biomarker tests Examples of current UK activity Potential Role of SMIP NHS/DH initiative to improve cancer gene testing Help provide evidence to support policy development for HGSG review of commissioning, innovation and service delivery in all genetic medicine commissioning and procurement Ability to use Cancer Drug Fund to buy associated complex diagnostics Help supply chain to develop new business models Value-based pricing review of drug procurement in NHS Support health economic assessment of diagnostic testing to inform SMIP/CRUK SMP large-scale demonstrator programme in cancer NHS pricing Pathology modernisation Support CRUK SMP and other demonstrators by funding associated Nicholson Review of Innovation in the NHS technology development OLS Senior Industry Group addressing business-NHS engagement Interim support for innovative molecular diagnostics in adoption phase NIHR/NOCRI work to improve access for clinical trials Help represent businesses in NHS policy/commissioning development Trade Associations (ABPI, BIVDA, ABHI, etc.) work with NHS on new commissioning structures
  • 6. 2. Increasing stakeholder awareness Barriers Evidence base not conclusive in all therapeutic areas Lack of public awareness of the complexity of Patients understand stratification and accept that gene-medicine interactions treatment access may depend on a complex test Some patients may see stratification as a method Current Perceptions of rationing Lack of high-quality, clinically approved, education Stratification tests included in education materials material for patients and practitioners around patient care pathway Future State Variable knowledge of how to apply a test in clinical practice Pace of change of evidence base Appropriate testing for multiple biomarkers incorporated in clinical care pathway Emerging models for clinical decision support do The difficulty of integrating clinical imaging and not always convince clinicians laboratory data Test usage inconsistent across providers Enablers More awareness among front line staff of clinical utility and benefits Development of tests for use at point of prescription (pharmacy/GP) or patients able to test themselves Agreement and sharing of genetic medicine health economic analyses Centralised communication resource to suit a range of health professionals Tools for physicians to provide clear interpretation of complex test results Better engagement with patient support groups in disease specific areas Physician-driven development of education materials for healthcare professionals involved in genetic medicine Patient access to global communication channels Examples of current UK activity Potential Role of SMIP Education working group of the HGSG Develop decision-making tools for physicians Various bodies already developing guidance materials Develop communication resource for education of health professionals Appointment of NHS Chair in Pharmacogenetics Involve patient support groups and Royal Colleges in SMIP CRUK large-scale demonstrator in cancer programme development Accelerate and communicate programmes that demonstrate validity of approach Help extend approach into other therapeutic areas
  • 7. 3. Patient recruitment (consents & ethics) Barriers Identifying and recruiting appropriate patient cohorts Data held in a range of databases and formats across A streamlined process for trials consent and patient can be time-consuming healthcare providers recruitment, and trials are safe Current Perceptions R&D approval process can be complex and time- Data quality may be low in data that has not been Consent for broad and unspecified future use, consuming monitored and audited supported by appropriate regulation of personal data Future State When patient consents are study or site-specific, data Inconsistent processes and standards for research Single, and internationally valid, consent and ethics cannot be used for other purposes consents approval Sometimes local ethics and R&D approval process can Privacy concerns around data storage and use Primary care has important role in patient recruitment inhibit larger national studies Excessive caution from some (unrepresentative) Use of data and samples for research based on Problems with national programme for IT has held back minority pressure groups ‘opt-out’ rather than ‘opt-in’ novel data solutions Enablers Structure of NHS gives unique opportunity to deliver standardised research consent Implementation of Academy of Medical Sciences recommendations on streamlining research regulation Novel IT solutions for capturing, communicating, storing and analysing data Connection of ethics committee with patient groups, starting with current disease Public engagement about use of samples and genetic data for research areas of focus like cancer Development of an overarching ethical framework Policy communication, IT enablement & governance. Cost of storing massive data sets decreasing Education and public awareness campaign demonstrating wider benefits to the Patient groups support non-restrictive consents and effective R&D approvals community of participation in research Development and widespread use of generic consent forms Collaboration in clinical trials recognised in clinician performance assessment Examples of current UK activity Potential Role of SMIP Academy of Medical Science report and recommendations Engage with patient groups on programme development and benefits of consent Leading hospitals already running generic consent for all patient samples Support Government implementation of AMS recommendations HGSG working on this area Consulting with stakeholders on routine/generic consent Plan for Growth actions on research approvals and recruitment to studies NIHR Translational Research Partnerships process for research consent in inflammatory disease Various data access and sharing initiatives (Generation Scotland, Cancer Registries, General Practice Research Database) Improved national and international data standards are enabling data sharing
  • 8. 4. Clinical Trials Barriers Localisation of commissioning and independence of Uncertain impact of localisation agenda in healthcare Reduced time and cost to conduct clinical trials providers leads to varied and complex implementation and changes in commissioning Reduced time to start complex genetic research Current Perceptions of national clinical trials Relative higher prices for trial activities in UK versus projects NIHR involve patients group BRIC countries Future State Reduced cost to recruit the appropriate subjects to Networks such as NCRI encourage and enable enable stratified medicine national trials All patients are able to participate in clinical Delays before patient recruitment starts is often the trials/population-based genomic research time-limiting step in studies Centralised solutions to access rare genetic types AMS report describes the situation well (e.g. web-based platform) Exclusion of inappropriate patient populations on grounds of safety genotypes Selection of appropriate patients for efficacy testing Enablers AMS report and recommendations Develop robust online (internet) recruitment National initiatives to improve trials environment (CRUK, NOCRI, etc) Develop standards within NHS for provision of patients into clinical trials Public increasing their levels of participation in research Better connection of infrastructure including IT systems and Biobanks Faster ethics approval based on patient choice and critical health ‘need’ Examples of current UK activity Potential Role of SMIP NIHR Clinical Research Networks, including cancer, could help in stratified medicine Similar to role in patient recruitment NOCRI helping industry engagement with NIHR trials infrastructure Help create critical mass to accelerate trials process NIHR common frameworks and guidelines to reduce patient recruitment times Communicate unmet needs (prioritisation) Demonstration of a consistent database of patient accessible records. Develop web-based recruitment tools
  • 9. 5. Data Barriers Routine data collection and quality variable across the Morale issues following NPfIT failure Population-wide consented patient data available for UK, better in Scotland analysis by researchers Most data has not been consented for access Current Perceptions NHS Information Centre as central repository and Routine use of anonymised patient data sets by NHS & Default position is to not make anonymised data access point for data but currently not meeting industry to improve patient outcomes Future State available for research researcher requirements Increased use of routine data in post-marketing Bespoke systems do not or cannot communicate Government data transparency drive (opengov) surveillance without significant investment No national programme for IT Routine collection of health economic data around SM Data protection issues seen as a barrier within trusts Range of IT systems in use Enablers Massive data sets Implement IT practices and routine generic consent for use of appropriately anonymised clinical data for research Public support for research between institutions IT capabilities improving Large-scale demonstrator programmes (regional or Encourage more open working between academia, therapeutic-area focused) NHS & industry Simplification of data protection rules around patient data Cultural/policy encouragement to share data Build in and embed quality systems into NHS data handling Examples of current UK activity Potential Role of SMIP Generation Scotland Develop tools to allow secure data mining of patient data across distributed data sources Individual routine consents Demonstrate that access to data repository by NHS & industry will CRUK/Eastern Cancer Registry partnership & SMIP investment in data handling improve patient outcomes and/or make UK a much better place to Ongoing improvements to cancer registries do research Establishment of Patient Reported Outcome Measures (PROMS) by Dept Health in England
  • 10. 6. Regulatory Framework and Development Pathway Barriers It can cost over $800m to take a safe and Lack of smart and adaptive study designs Novel design of clinical trials makes smaller cohorts of effective drug to market, and take 7-9 years from patients possible Lack of a single body controlling quality of Current Perceptions first in-man trials diagnostic labs Coordinated regulation of pharmaceuticals industry New drugs sometimes only marginally better than (Rx) and diagnostic industry (Dx) Future State Need for regulatory guidance development for standard care but more expensive drug-diagnostic combinations Companies make more progress within existing Different degrees of regulation for pharmaceuticals, regulatory framework diagnostic tests and laboratories/hospitals Enablers Regulatory guidance for co-development Facilitate co-development of drugs and of diagnostics and treatment diagnostics by alignment of the regulatory processes DH and BIS encouraging EU Commission to improve regulatory framework Setting up of an expert committee (akin to the Committee for Advanced Therapies set up by the ATMP Regulation) Examples of current UK activity Potential Role of SMIP EU Commission on the revision of the IVD directive Help in novel clinical study design NHS QIPP agenda includes work on quality and standards Encourage discussion with regulatory agencies
  • 11. 7. Intellectual Property Barriers Laboratory-developed tests using unlicensed IP in NHS Questions around effective regulation of laboratory- IP with enhanced licensing for NHS use acts as a driver discourages development of new diagnostic tests by developed tests of ‘step change’ innovation Current Perceptions industry No single approach to NHS licensing of test technology Publicly funded labs are free to develop safe Laboratory-developed tests can sometimes save the and effective tests that do not infringe on other Future State Patent thickets – multiple IP on sequence reduces NHS money IP rights-holders economic attractiveness of panel testing Some NHS laboratory-developed tests are being Competition between commercial and laboratory- technically and clinically validated and externally quality developed tests benefits patients assured Current IP for gene sequences (BRCA/Myriad etc) subject to legal challenge Enablers Patent box tax incentive Develop IP protection on the test, not the underlying gene sequence Excellent technology platform & biomarker science base Provide a strong home market (NHS) with value based pricing (product & service) and willingness Business models for developing & exploiting new to adopt new tests & existing IP that provides fair return on investment whilst maintaining reasonable end A stronger domestic industry with global outlook, user cost able to capitalise on excellent science base Examples of current UK activity Potential Role of SMIP EU In-Vitro Diagnostic consultation in process Provide support for diagnostic companies and co-development OECD guidelines for licensing genetic inventions Support SMEs in early IP filing HGC recommendations on IP and DNA diagnostics Commission health economics studies Support development of new business models and pricing mechanisms
  • 12. 8. Bio-Banks and Early Biomarker Detection Barriers No central overarching database of biomarkers or Localised clinical and research databases are hard to UK-wide database of biobanks is used for biosignatures use for national level research stratification studies Current Perceptions Many biosamples not clinically annotated Lack of technology platforms for use in clinical biomarker All tissue collected with consent for broad clinical research research and data capture Future State Limited standardisation of tissue collection or consent Require policy framework for biomarker validation Routine, effective collaboration on biomarker ID Not everyone sees the value of centralised biobanks Many biomarkers do not have associated therapies A range of early biomarkers identified for future Stand-alone, incompatible tissue and data validation infrastructures Coordination for Biomarker research via mandated UK Biobank has recruited 0.5m people central database In silico trials to ID biomarker Enablers Accreditation system for biobanks Improved biomarker discovery and validation through new approaches Improve coordination of funding for biobanks Linking of databases and biobanks Standardisation of procedures, including sample handling storage and consent process Improving IT to enable metadata approach across multiple systems Ensuring biobanks store comprehensive clinical data Increase in pharma collaboration and pre-competitive Broad & enduring consent or implement opt-out activity rather than opt-in for data analysis in research Examples of current UK activity Potential Role of SMIP UK Biobank programme standardising access procedures Help develop standards for biobanks Generation Scotland Map existing UK databases and biobanks Routine generic consent at sample-acquisition in major teaching hospitals Support development of sample handling & preservation technologies NIHR/NOCRI Translational Research Partnerships Support development of biosignature analysis and data storage techniques Encourage widespread adoption of best practice Help connect existing infrastructure to create critical mass Enable access to infrastructure to accelerate development of new tests and therapies
  • 13. 9. Increasing and Leveraging UK R&D investment Barriers Developing new drugs has becoming increasingly Different parts of the system have different objectives Taking drugs from discovery phase to expensive commercialisation is faster and cheaper, enhancing Bridging the gap between research and commercial Current Perceptions business performance and encouraging investment Pharmaceutical businesses are re-evaluating their R&D exploitation investments Stratified medicine offers improved value for money for Future State The RAE/REF do not incentivise academics to help customers including NHS Turbulence in financial markets has impacted commercialisation availability of equity and debt finance Improved co-ordination of public and private funding The sector is extremely large and complex, so major Public sector funders have not always aligned their change is non-trivial activities University funding is changing NHS needs to save money Enablers Creating a coordinated UK programme Better coordinated UK and EU funding streams Incentivise stakeholders to share pre-competitive data Understand the value of cooperative investment Stakeholder support for SM Taking an international perspective Full audit of UK capabilities /IT, biomarkers, Dx, Rx, clinical etc Examples of current UK activity Potential Role of SMIP ABPI work in this area Commission health economic studies OSCHR work in this area Help develop new business models for stratified medicine Nicholson Review of innovation in the NHS Ensure coordination of partner activities NorthWest exemplar Help create critical mass through a UK-wide programme Stratified Medicine Innovation Platform investments to date Engage in international programmes
  • 14. Stratified Medicine in the UK Stratified Medicine The SMIP programme management group would greatly welcome input from other Innovation Platform players. The Medicines and Healthcare Partners products Regulatory Agency (MHRA) and the National Institute for Health Research The innovation platform seeks to make the (NIHR)/ NIHR Office for Clinical Research UK the best place to develop, and have Infrastructure (NOCRI) have already adopted, stratified medicine products and started working closely with the innovation services. This is an extremely ambitious platform, and it is hoped that others would vision that will require activities across also like to participate. There will be areas multiple technology areas, policy streams where others are better placed to lead, and government departments. Each of the and we can support their programmes. Stratified Medicine Innovation Platform partners has a specific remit and can contribute significant resources and expertise from their core areas of How to engage with us capability. Together, the group covers a For the UK to achieve the vision set out in broad spectrum of interests. By aligning this document, significant advancements activities, many of the issues described in technology and changes to systems above can be progressed. and processes will be required. This cannot be achieved by any single The Technology Strategy Board aims to organisation and will entail coordinated help businesses to develop innovative new activity across the entire healthcare sector. products and services, while the Medical This UK Stratified Medicine Vision and Research Council funds outstanding Roadmap can form the focal point for research in the science base and has a companies, public sector bodies and major initiative in stratified medicine. government to coordinate their efforts. Cancer Research UK is the world’s leading The innovation platform partners have cancer charity dedicated to saving lives begun this coordinated activity and would through research, and Arthritis Research welcome help and advice from others UK has an interest in achieving better whose actions could help accelerate the outcomes for patients in its disease area: journey towards the ultimate goal. both have stratified medicine at the centre A web-based community has been of their strategies. The Department for created to encourage engagement Health and Scottish Government Health (https://ktn.innovateuk.org/web/stratified Directorate are government departments -medicines-innovation-platform). whose main role is to improve health and This will be supported by the innovation achieve better health, better care, and platform and will highlight future better value for all. The National Institute workshops, funding calls and ongoing for Health and Clinical Excellence is discussions on specific stratified- tasked with assessing medical medicine-related topics. We would also interventions, both therapies and encourage others to use this forum to share diagnostics, and recommending their use information, raise awareness, identify based on value calculations. partners and generate yet more momentum. 014 | Technology Strategy Board
  • 15. Vision and Roadmap Glossary ABHI Association of British Healthcare Industries ABPI Association of British Pharmaceutical Industry AMS Academy of Medical Sciences ATMP Advanced Therapy Medicinal Products BIS UK Department for Business, Innovation & Skills BIVDA British In-vitro Diagnostic Association BRIC Brazil, Russia, India and China CRUK Cancer Research UK CRUK SMP Cancer Research UK Stratified Medicine Programme DH Department of Health Dx diagnostic HGC Human Genetics Commission HGSG Human Genome Strategy Group IP intellectual property IT information technology IVD in-vitro diagnostic NCRI National Cancer Research Institute NHS National Health Service NICE National Institute for Health and Clinical Excellence NIHR National Institute for Health Research NOCRI NIHR Office for Clinical Research Infrastructure NPfIT National Programme for IT OECD Organisation for Economic Co-operation and Development OLS Office of Life Sciences OSCHR Office for Strategic Co-ordination of Health Research PROMs patient reported outcome measures QIPP quality, innovation, productivity and prevention R&D research and development RAE/REF Research Assessment Exercise/Research Excellence Framework Rx therapeutic SM stratified medicine SMIP Stratified Medicine Innovation Platform Technology Strategy Board | 015
  • 16. Stratified Medicine in the UK Technology Strategy Board North Star House North Star Avenue Swindon SN2 1UE Telephone: 01793 442700 www.innovateuk.org © Technology Strategy Board October 2011 T11/065 016 | Technology Strategy Board