All patients are different, and data collected during product development or Randomised Clinical Trials (RCT) does not always paint the full picture of everyday patients. RWE insights complement the manufacturing process and RCT findings, adding more value and providing real-world impact. While together data from the manufacturing process and RWD paint a fuller picture. Due to the limitations of the study design, data from the manufacturing process and RCTs are inadequate for demonstrating an intervention’s long-term safety and effectiveness. Moreover, it is possible to compare multiple product or interventions in RWE.

1. Real-World Evidence: Patient Outcomes
By Mehdi Ehtesham
June 2018

2. Agenda
Concepts
Usage And Solutions
Source of Data
Available Datasets
Mobile Health and Wearable Technologies
Regulatory Agencies and Post-approval Studies
Business Development
Marketing Strategy
Research Proposal and Contracts
Project Management
Project Delivery
Challenges vs. Opportunities

3. Concept of Real-World Evidence (RWE)
All patients are different, an data collected during
product development or Randomised Clinical Trials
(RCT) does not always paint the full picture of every
day patients. RWE insights complement
manufacturing process and RCT findings, adding
more value and providing real-world impact. While
together data from manufacturing process and RWD
paint a fuller picture.
Due to limitations with the study design, data from
manufacturing process and RCTs are inadequate for
demonstrating an intervention’s long-term safety and
effectiveness. Moreover, it is possible to compare
multiple product or interventions in RWE.

4. Usage of Real-World Evidence

5. Source of Data
Medical claims
•Initially created for
financial administrative
purposes.
•Patient-level information
on diagnosis, treatment
types, providers and
costs
•High level of coverage
patient’s activities
Electronic
medical records
•More comprehensive
clinical data
•Include info on non-
prescription (Over-the-
counter)
•Additional healthcare info
(labs, risk-factors, nurses
etc.)
•Not limited to only insured
population
•Not widely adopted
Patient registries
•Commercial and research
purposes
•To understand clinical
practice, identify unmet
clinical need, disease
natural history and
treatment.
•To evaluate the
effectiveness if
treatments, meet post-
marketing commitments
in terms of qualification
and characterisation of
safety endpoints
•To demonstrate the
economic value, quality of
life benefits of a particular
treatment/product
Chart review
•Pre-recorded patient data
are extracted involves
medical information
Physician, patient
and public
surveys
•Should be well designed,
structured standardised
and validated
Mobile health
and wearable
technologies
•Mobile health apps and
wearables, particularly
those that collect patient-
and consumer-generated
health data, can fill some
of these data gaps by
providing real-world,
more meaningful, high
frequency and/or
longitudinal data.

6. All Available Datasets
• Electronic Health Records
• Pharmacy Datasets
• Medical Claims Datasets
• National Registries

7. Mobile Health and Wearable Technologies
Mobile health and wearable technologies has the potential to enable progress in better
evidence in health care. “Patient/consumer-facing” apps and wearables that collect
patient-generated health data can provide a low-cost mechanism for gathering real-
world, high frequency and/or longitudinal data on measurable outcomes that people
care about. As mobile health and wearable technologies are increasingly adopted for
clinical use and in the consumer space, the data may be reused as part of real-world
evidence generation. Such data reuse could also become an additional source of
revenue for medical device manufacturer, beyond their value to payer or provider
systems, for use in predictive analytics and improving quality of care. But despite some
promising examples, the impact of mobile health and wearable technologies on
research is limited today.
There is not yet a clear path for how mobile health and wearable technologies can
reliably and efficiently elicit, validate and transmit relevant data and such data are
currently not being collected on a sustained and longitudinal basis. Consequently, this is
a crucial time for mobile health and wearable technologies, with growing attention to
overcoming the challenges of interoperability, common data elements and data
definitions in order to allow disparate data streams to combine to create actionable
insights for improving or maintaining an individual’s health and treating disease.
• Create collaborations between the patient, clinical, and research communities as well
as medical device manufactures to advance the science on collecting and using
mobile health and wearable technologies data for evidence generation.
• Enable developers and companies to build their products on a strong standardised
base in the pre-competitive space.
• Ensure that users of patient/consumer-facing technologies understand and can more
efficiently consent to how their data may be used, and are kept informed of research
developments resulting from their data donations.

8. Regulatory Agencies and Post-Approval Studies: FDA
Elements to Include in a Post-Approval Study Protocol
FDA recommends you include the following elements in a post-approval study protocol:
• Background (e.g. Regulatory history, brief description of device, indications for use)
• Purpose of study
• Study objectives and hypotheses
• Study design
• Study population (including subject inclusion and exclusion criteria and definition and source of comparator group)
• Sample size calculation (statistically justified and based on study hypothesis)
• Primary and secondary endpoints (including definitions for study endpoints, success criteria, list of adverse events/complications, standard operating procedures for a
determination of relatedness with device and/or the procedure)
• Length of follow-up, follow-up schedule, description of baseline and follow-up assessments
• Description of data collection procedures (including recruitment plans, enrolment targets, plans to minimize losses to follow-up, follow-up rate targets, quality
assurance, and control)
• Statistical analysis
• Data collection forms, informed consent forms, and IRB approval forms
• Reporting requirements for interim and final reports
• Study milestones/timeline elements, including:
o Expected date of study initiation
o Expected monthly number of study sites with IRB approvals
o Expected date of initiation of subject enrolment
o Expected number of subjects enrolled per month
o Expected date for subject enrolment completion
o Expected date to complete follow-up of all study participants
o If applicable, information related to intermediate milestones (e.g. Evaluation of surrogate endpoints in a study that also measures clinical benefits).

9. Business Development Process
Marketing
& sales
plans
• Online marketing
• Arrange meetings
• Keep updating
clients
Meet those
needs
better than
competition
• How can
MedixGlobal
differentiate itself
from other
competitors
specific
needs of
client
• Address the gap
• Identify objectives
• Key business
problem
Define
target client
• Identify a target
product (e.g.
Inhaler)
• fully search about
device profile

10. Marketing Strategies
Find and contact the
correspondent or
relevant person
Identity key business
problem or opportunities
Establish a connection
(e.g. sending an email
and giving a call)
Arrange a meeting and
present the proposal:
understanding of
objectives, timelines and
budgets

11. Setup and Manage the Research Proposal and Contracts
Objectives:
The most important step in
the market research process
is defining the goals of the
project. In this step we
usually sit with the client to
understand the root question
that needs to be informed by
market research.
Methodology:
- Primary vs. secondary
- Retrospective/prospective
- Participants’ characteristics
- Inclusion/exclusion criteria
- Sample size
- Data collection/mining
- Data analysis
Timetable and fees:
List of all activities and
services
Terms & conditions:
- Termination and
suspension
- Payment and fees
- Intellectual property rights
- Warranties and disclaimers
- Limitation on liability
- Indemnity, confidentiality
- Force majeure

12. Project Management of Multidisciplinary Team
Team work
- Work with multiple
teams of experts
- Develop study
frameworks
Breakdown the
project into
small tasks
- Creating a Gantt
table or using current
project management
system
- Define tasks for
each responsible
colleagues and
stakeholder
- Set a deadline for
each task, track
performance of the
tasks
Updating the
client
Sending regular
updates about project
development and
process to the
stakeholder and
clients
Improve the
services
- Track the
performance of
overall projects
- Get feedback from
the client and
stakeholders
- Ask for suggestions
to improve services
Problem
solving
- Troubleshooting
barriers
- Find solutions

13. Project Delivery
Quality control
• ensuring that the
report/manuscript to meet
the needs of the client
and address all objective
and specifications of the
research proposal.
Creating report
• Academic vs.
commercial, follow the
guidelines (journal
guidelines). Usually two
reports should be created
for different groups of
audiences including
technical and non-
technical readers.
Presentation
• Check it with a colleague
before sending the file,
customise it based on
audiences, make sure
that we are aware all
aspects of that project.
summarise all key
findings, understandable
and not too complicated.

14. MedixGlobal Capabilities
• More than 10,000 of panel of healthcare professionals
• Team of experts
• Advanced analytical expertise
• Creating reports from a commercial prospective
Proficient in primary market research
• Programming google cloud software to analyse data
• Real-time data analytics
Machine learning process
• Get access to syndicated reports
• Delivered more than 2500 projectsBeing in the field of pharma industry since
2000
• Surveys
• CATI data collection
Installing an in-house data collection system

15. Challenges vs. Opportunities
Limited
access to
data
Incomplete
data
Enormous
data
volume
Real World
Evidence
Challenges
MedixGlobal
capabilities
Infrastructu
re
Panel of
patients
Experts

16. Aligning the Stakeholder Needs
• Evidence of positive clinical,
humanistic and economic outcomes
• Minimal side effects
• Cure the disease
• Improve quality of the life
• Affordable care
• Maximum treatment safety and
effectiveness
• Reduced treatment costs
• Address gaps
• Feedback from payers and patients
Pharma: Providers:
Payers:Patients:

17. Thank ou