Introduction to outcomes research

THE POWER OF REAL-WORLD INFORMATION
TCD OUTCOMES RESEARCH © 2016 | 1

OBJECTIVES
It is NOT to create OR specialists ….
Use a product/industry life-cycle
perspective ….
Using OR smartly ....

• Framing Outcomes Research (OR)
• Techniques
• Case studies
• Application
CONTENTS:

1. FRAMING OUTCOMES RESEARCH
In your view:
• Why are you here today?
• What is OR?
• Where, if at all, should OR reside in
your organization?
FRAMING OR

DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS
| 6TCD OUTCOMES RESEARCH © 2016
• New questions from regulators, geographies, insurers, payers, patients, providers
• Disruptive new technologies: New pressures on price (NDOH Pricing Committee)
• New pressures on illustrating value
• Internal and external validity
MARKET ACCESS AND
REIMBURSEMENT CHALLENGES
FRAMING OR

EVOLUTION OF EVIDENCE
Dynamic Solutions to Dynamic QuestionsTM
Source: http://www.quintiles.com/~/media/library/presentations/the-emerging-role-of-real-world-studies-and-registries.pdf
FRAMING OR

FRAMING OR
DRUG DEVELOPMENT CYCLE

FRAMING OR
COMMERCIALIZATION CYCLE
High-value vs.
non-high-value
assets

FRAMING OR
DYNAMIC SOLUTIONS TO
DYNAMIC QUESTIONS™

FRAMING OR
THEREFORE …
• What is OR? • What makes it
different?
• How can OR contribute
towards policy?
?

FRAMING OR
• Outcomes Research “seeks to understand the
end results of particular health care practices
and interventions” on individual patients and
populations
• Often includes:
»» Clinical impact
»» Economic impact
»» Financial impact
»» Funding impact
»» Market impact
»» Public health impact
Clinical impact
Economic impact
Financial impact
Funding impact
Market impact
Public health impact
WHAT IS OR?
?

FRAMING OR
1. The Question It Asks
A hallmark of outcomes research is the breadth of issues it addresses. Outcomes research touches all aspects
of health care delivery, from the clinical encounter itself to questions of the organization, financing and
regulation of the health care system. Each of these factors plays a role in the outcome of care, or the ultimate
health status of the patient. Understanding how they interact requires collaboration among a broad range
of health services researchers, such as physicians and nurses, economists, sociologists, political scientists,
operations researchers, biostatisticians and epidemiologists.
2. The Setting It Studies
Outcomes research evaluates the results of the health care process in the real-life world of the doctor’s office,
hospital, health clinic and even the home. This contrasts with traditional randomized controlled studies,
funded mainly through the National Institutes of Health, which test the success of treatments in controlled
environments. These are called efficacy studies. Research in real-life settings is called effectiveness research.
WHAT MAKES IT DIFFERENT?

FRAMING OR
3. The Health Status Measures It Uses
Traditionally, studies have measured health status, or health outcomes, in terms of physiological measurements
– through laboratory test results, complication rates (e.g. infections) or death. These measures alone do not
adequately capture health status. A patient’s functional status, well-being, and satisfaction with care must
compliment the traditional measures.
4. The Methods It Uses
Constraints of money and the time it takes to conduct large, long term clinical trials is a major challenge of
outcomes research. One strategy of overcoming this barrier is to use existing computerized databases, such as
those available from Medicare, Medicaid, private insurance companies, and major employers. Another strategy
is to use simple questionnaires that patients can use to evaluate their own health and health care. Outcomes
research also uses meta-analysis, a technique to summarize comparable findings from multiple studies.
These approaches complement the more standard research strategies involving the collection and analysis of
information from medical records and other clinical and management data.
WHAT MAKES IT DIFFERENT?

FRAMING OR
CONTRIBUTION TO POLICY
• Global policy trends …
• Country policy
• Funder policy
• Clinical policy
Bridge the gap between clinical and commercial
functions, throughout the product life-cycle. By infusing
insights from the marketplace in the form of real-world
data into the development process, companies can better
align their development efforts with unmet patient needs
and accelerate time to market with the right evidence
package. In the post-marketing realm, collecting more
data on real patients in a real-world setting is becoming
the new mandate.

FRAMING OR TECHNIQUES
2. OUTCOMES RESEARCH TECHNIQUES

TECHNIQUES
Our armamentarium:
• Systematic review and Meta-analysis
• Health economics
• RWD analysis
• Observational studies
• Comparative effectiveness trials
• Patient reported outcomes
• Phase IIIB and IV studies

TECHNIQUES: SCIENTIFIC METHODOLOGY
Just because it is OR, does not mean we do not need scientific rigor …
The research question: Why are red cherries black?
Hypothesis: Because they’re green.
• Protocol development, PI and GCP
• Regulatory
• Ethics
• Project management
• Site management, CRF, data collection
• Data management
• Biostatistics
• Medical writing: Clinical study Report
• Value messages related to different stakeholders

TECHNIQUES: RWD ANALYSIS
Examples:
• Retrospective data review
»» Medical schemes claims data
»» Expanded access programs
• Retrospective chart review
Types of questions answered:
• Total cost of illness
• Patient flow modeling
• Adherence
• Data inputs for PE modeling
• Epidemiology
• Medicine initiation and switching rates
• Competitor analysis
• Local patient outcomes

TECHNIQUES: RWD ANALYSIS
HOW?:
• Research question
• Primary and secondary objectives
• Data specification
• SAP
• Data collection and analysis
• Results
• Impact and value messages

TECHNIQUES: OBSERVATIONAL STUDIES
Involves the direct observation of phenomena in
their natural setting
Examples:
• Registries
• Epidemiological studies

TECHNIQUES: OBSERVATIONAL STUDIES
Registries:

FRAMING OR
A PATIENT REGISTRY is an organized system that uses observational study methods to collect uniform
data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease,
condition, or exposure, and that serves one or more predetermined scientific, clinical, or policy Purposes.
A REGISTRY DATABASE is a file (or files) derived from the registry.
FOCUSED on the outcomes, effectiveness, comparative clinical effectiveness, and appropriateness of
pharmaceuticals, devices, and health care services.
DEFINITIONS
TECHNIQUES

FRAMING OR
PROCESS
Creating registries:
• Planning
• Design
• Data elements
• Use
• Data sources
Source:
https://effectivehealthcare.ahrq.gov/ehc/products/420/1897/
registries-guide-3rd-edition-vol-1-140430.pdf
TECHNIQUES

FRAMING OR
PROCESS
The key points to consider in designing a registry include:
• formulating a research question;
• choosing a study design;
• translating questions of clinical interest into measurable exposures and
outcomes;
• choosing patients for study, including deciding whether a comparison
group is needed;
• determining where data can be found; and
• deciding how many patients need to be studied and for how long.
TECHNIQUES

FRAMING OR
PROCESS
TECHNIQUES
Ethical considerations:
• Ethics, data ownership, privacy
• Patient informed consent
• Data protections (FDA CRF Part 11 compliance)
http://www.fda.gov/RegulatoryInformation/Guidances/ucm125067.htm

FRAMING OR
PROCESS
TECHNIQUES
Operating registries:
• Recruiting and retaining patients
• Data collection and QA
• AE’s recording and reporting
• Data analysis
• Medical writing

SCIENTIFIC RIGOR
https://www.graceprinciples.org/doc/GRACE-Checklist-031114-v5.pdf

Static Registries to Dynamic eRegistriesTM

Static vs. Dynamic Registries

• Paper based CRF
• Transcription into e-CRF
• Significant monitoring
• Data management cumbersome
• Takes time: high touch, low tech
• Reporting is laborious
Static Registries

E-REGISTRY:

Computational Model + E-Registry = Dynamic e-Registry
DYNAMIC e-REGISTRIES

DYNAMIC e-REGISTRIES
• Automated descriptive analyses:
Simple descriptive statistics
• Automated predictive analyses:
Predict heterogeneity of treatment effect; predict
primary/secondary outcomes
• Automated prescriptive analyses:
Machine learning - Prescribe what patient
characteristics will provide best outcomes
• Dashboards:
Create bespoke dashboards as required by stakeholders
• Automated exposure to stakeholders:
All technology platforms

TECHNIQUES: COMPARATIVE EFFECTIVENESS TRIALS (CER)
“While the implementation of CER has
implications across global healthcare
systems, the US focus on CER points to the
growing influence of payers and their desire
to control spending.”1
1. Nellesen et al. Pharmacoeconomics 2010; 28 (10): 789-798, 1170-7690/10/0010-0789/$49.95/0

TECHNIQUES: COMPARATIVE EFFECTIVENESS TRIALS (CER)
“In an era marked by increasing prominence
of CER evidence, successful pharmaceutical,
biotechnology and medical device manufacturers
will need to develop products with unambiguous
evidence of economic as well as clinical value
relative to alternatives.”1
1. Nellesen et al. Pharmacoeconomics 2010; 28 (10): 789-798, 1170-7690/10/0010-0789/$49.95/0

FRAMING OR
“CER is defined as research comparing interventions in the real world settings”1
1. http://www.med.upenn.edu/sleepctr/documents/FederalCoordinatingCoucilforCER_2009.pdf
CER DEFINITION
TECHNIQUES

• Non-inferiority trials • Superiority trials • Observational studies
CER EXAMPLES

TECHNIQUES: PATIENT REPORTED OUTCOME STUDIES (PRO)
“PRO is a health outcome directly reported by the patient
who experienced it.”

TECHNIQUES: PATIENT REPORTED OUTCOME STUDIES (PRO)
Examples:
• Symptoms (impairment)
• Functioning (disability)
• General health perceptions
• Quality of life
• Rating health care

FRAMING OR TECHNIQUES CASE STUDIES
3. CASE STUDIES
Question:
• Can you think of a specific clinical/competitive issue that
might be addressed by using OR techniques?
• Would you like to use your specific problem as a case
study for this session?

SETTING THE CASE STUDY
CONTEXT:
• As product manager, you are informed that, in 2018,
casestudymab, your company’s MAB in the treatment
of prostate cancer, will be launched in SA
• You are told it will cost approximately US$40,000 per
treatment
• There are at least 3 competitors in this setting in SA
• Their comparative cost is about US$25,000 per
treatment
Using the knowledge
gained in this session,
how will you proceed?

FRAMING THE RESPONSE
Activity Activity plan
Pre-launch activities
Launch activities
Post-registration activities

FRAMING OR TECHNIQUES CASE STUDIES APPLICATION
4. APPLICATION

4. APPLICATION
CONTEXT:
• Clinical affairs
• Medical affairs
• Regulatory affairs
• Market access
• Commercial

4. APPLICATION
THE BIG OR OBJECTIVE:
• It is part of your competitive strategy …
• It is about building a comprehensive portfolio of real world evidence …
• That can be used by all departments …
• To reach commercial objectives of market access and reimbursement
...
• Using S.M.A.R.T. Goals.

“In times of change the learners shall inherit
the earth … while the learned will find
themselves perfectly equipped to manage a
world that no longer exist” – Eric Hoffer
tienie.stander@tcd-global.com
www.tcd-global.com

Introduction to outcomes research

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