3/10/2016

A Visual Model of 
Patient Engagement in Benefit‐
Risk Assessment through the 
Medical Product Life Cycle
March 10, 2016
12 Noon – 1:30 PM ET

Disclaimer

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Information Association Inc. All other trademarks are the
property of their respective owners.

© 2015 DIA, Inc. All rights reserved.

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Speaker Disclosure
It is DIA’s policy that all faculty participating in continuing education activities must
disclose to the program audience (1) any real or apparent conflict(s) of interest related to
the content of their presentation and (2) discussions of unlabeled or unapproved uses of
drugs or medical devices. Faculty may have disclosed one or more of the following:
grants/research support, consultancy relationships, speaker’s bureau participation,
significant equity (stock) positions, and sources of honoraria/expenses.

This educational activity may include references to the use of products for indications not
approved by the FDA. Opinions expressed with regard to unapproved uses of products
are solely those of the faculty and are not endorsed by DIA or any of the manufacturers
of products mentioned herein. Faculty for this educational activity was asked to disclose
any discussion of unlabeled or unapproved uses of drugs or medical devices.

Robyn Lim, PhD: No financial relationships

Kimberly McCleary: No financial relationships
Marilyn Metcalf: Employee GSK; stock shareholder of Centocor (part of J&J)
Meredith Smith: Employee of Amgen; stock shareholder of Abbott/AbbVie, BMS, Pfizer
Debra Michaels: Stock shareholder of Abbott/AbbVie, Sanofi
© 2015 DIA, Inc. All rights reserved.

Thank You……

Special thanks goes to PCORI,

The Patient-Centered Outcomes Research

Institute,

for the Engagement Award for Conference Support

that partially funded the DIA conference and made
the Visual Model possible

© 2015 DIA, Inc. All rights reserved.

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Panel Participants

Robyn Lim, PhD Kimberly McCleary Marilyn Metcalf, PhD

Meredith Smith, PhD, MPA Debra Michaels, MS, RDN

© 2015 DIA, Inc. All rights reserved.

Patient Engagement in Benefit‐Risk Assessment throughout the Life 
Cycle of Medical Products: Current and Future States 

COLOR KEY
• Green: denotes aspects of patient engagement in benefit‐risk assessment of medical products that are in place, with 
efforts begun
• Yellow: denotes aspects that are not now in practice but should be implemented in the medical product life cycle for 
effective and meaningful patient engagement in benefit‐risk assessment. Yellow may also denote aspects that are 
sometimes practiced but need to be applied more widely/consistently or improved upon. 

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In this model, the term “patients” may include individual patients, their family 
members, caregivers, or the organizations that represent them.  
Inputs from patients and other stakeholders into the medical product development 
cycle are multiple and diverse.  Some inputs are already incorporated or mandated 
(green shading) while other inputs are not yet linked into the cycle or only tentatively 
linked by a few people or groups (yellow shading).

In the following slides, each part of the medical product life cycle is 
described, identifying where patients, who ultimately will utilize the medical 
products, can or should be engaged in benefit‐risk decision making to assure 
best patient outcomes.
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The patient should be involved as a partner, collaborating with all other
stakeholders in the medical product development cycle. This concept is
visualized in the "Patient as Driver Steering Committee", a conceptual 
representation of the processes and practices that should involve appropriate 
engagement among  all stakeholders, including the patient, to ensure patients' 
preferences and needs remain the focal point throughout the life cycle.

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The term “unmet patient needs” encompasses the concept of 
unmet medical treatment needs as well as unmet needs for 
involvement and input into the medical product development 
process.  

Patients' needs, perspectives, preferences, and choices, collectively referred to as
"patient input," are diverse.  Patients come from many different communities, and 
we need to ask for input from all patients, including those who are remote and hard 
to reach.

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Many ideas for systematically incorporating patient input 
have been identified, but substantiated frameworks and 
methodologies to collect and utilize patient input at the 
individual patient and the public health levels are lacking.

Although some frameworks enabling such a flow of information 
are found in some areas of research, such as conducting 
structured studies, often these are focused on collection of 
patient input and lack a pathway to return the information to 
the patient community.

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A number of professionals currently provide input about 
unmet needs of patients into the development cycle.  This 
input could be improved by a better understanding of the 
development cycle and more effective communication with 
the patients whose needs are being represented. 
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A common understanding about patients' everyday experiences with 
a disease is needed to enable appropriate choices about what to 
develop and why. 

Such understanding will emerge with enhanced engagement of all 
stakeholders throughout the medical product development cycle in 
systematically collecting input from a heterogeneous sample of 
patients, coalescing it into a larger public health picture, and 
appropriately sharing the results with the community.

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The collection of patient input through formal trials is well‐established and 
includes diverse disease‐specific scales and quality of life measures. While 
these scales are designed to elicit patient self‐reported data (e.g., pain 
level, impact on well‐being), they are not designed to assess patients’ 
treatment preferences and goals. 

A pathway to effectively communicate differences between
the patients' and other stakeholders' perspective on the
relevance of a scale is needed early in the development
cycle. This would help in keeping the patients’ perspectives and
needs as the focal point of medical product development.
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The reciprocal flow of communication among all stakeholders and medical 
product research programs is a fundamental aspect of robust patient 
engagement early in the development cycle.
Research 

The heterogeneous nature of patients’ needs 
could be fed into the development cycle by 
patients well versed in the disease state and 
possessed of common terminology and 
understanding of the medical product 
development cycle. An ongoing, reciprocal flow of information
between patients and other stakeholders in 
the process is important to keeping patients 
engaged in the cycle.
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Funding is critical for
research and 
development of
medical products, but 
also for financing the 
development of a 
patient engagement 
framework and 
methods.  Funding is 
needed as well to 
continuously improve 
our efforts to engage 
with patients.

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Research sponsors are evolving 
toward increasing patient 
partnership in 
proactive 
planning
rather 
than only 
reacting 
to a study 
experience 
or a  developed 
treatment. 

These views have led to 
increased incorporation 
of patients’ perspectives 
and needs into early 
planning and into 
proposed label language 
discussions with 
regulatory agencies.
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Upon completion of studies supporting a marketing authorization application, the 
product undergoes regulatory review. Increasingly, as evidenced by current and 
evolving legislation, regulatory bodies are recognizing the importance of obtaining 
patients’ input regarding their disease, including the risks and uncertainty tradeoffs 
they are willing to make for certain treatment benefits.

Enriching the data review package with multi‐faceted patient input 
would help ensure that approved medical products holistically 
address the needs important to patients.
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Inclusion of patient input 
into regulatory decision 
making needs to become 
an integral and formal 
part of the process. 

While regulatory bodies 
have begun to 
incorporate some patient 
Structured methods 
input, such input has 
and scientific 
been predominantly 
approaches to
anecdotal and 
gathering substantial
idiosyncratic as opposed 
evidence on patients'
to systematic and 
input are lacking. 
representative in nature. 
Some strides to fill this 
gap are emerging, but 
greater effort is 
needed to align on a 
common language for 
patient engagement 
and on methodologies
acceptable to gather 
and analyze those data.
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As a medicine enters the market, patient involvement could be harnessed to a greater 
extent to help manage and monitor the medical product's performance through: 
enhancing decision support for patients to help ensure the right patients are getting the 
right medicine/product in the right way; further developing patient registries to build or 
enhance real‐world data repositories for effectively querying a medicine/product's
performance and understanding how it is best managed in real‐world patients.

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After approval, the medicine is 
used by a more diverse 
population of patients than 
those who may have 
participated in clinical trials.  
Information about the 
medicine becomes more 
complex to understand.  At the 
same time, opportunities 
continue to identify/ 
characterize subpopulations of 
patients who might best 
benefit from or
be most susceptible
to the negative effects of 
the medicine. This would 
allow for refined labelling and 
use, as necessary, to maintain 
a positive benefit‐risk balance 
for all stakeholders involved
in managing patient care.

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Real world evidence allows a 
more structured way of 
monitoring a medicine’s 
performance as more patients 
have the chance to take the 
medicine.

Innovations in real‐world
evidence data platforms,
governance systems and
database linkage potential are
currently opening up, to an
unprecedented degree,
opportunities to capture and
consider real‐world patients'
experiences and perspectives
about drug performance.

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Information collected from patients in the real world should be 
fed back into the development cycle at critical junctures, such 
as funding decisions, research, approval, or reimbursement 
processes, thereby allowing for iterative, coordinated decision‐
making by key stakeholders. A structured process for robustly 
gathering this information is lacking.
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Pulling the lens back, we can see how patient input should be integrated into 
benefit‐risk decision making throughout the entire medical product life cycle. 

This medical product development story began with the patient at the center and has
provided a vision for increasing and improving patient engagement for informing the
benefit‐risk assessment of medical products. Achieving this vision would positively
impact people and communities everywhere by providing medicines/products that
best meet the needs of those taking them.

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Ask

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Send Us Your Comments

• View/download the Visual Model:

http://www.diaglobal.org/en/resources/tools-and-downloads#Visual-Model

• Send us your comments on the Visual Model or

on patient engagement in benefit-risk
assessment:
PEBRModel@diaglobal.org

Questions to Consider:

• What other advances have been made in including the

patient voice in benefit-risk decision making?

• What changes are most critical to assure the inclusion

of patient input in benefit-risk decision making?

• What are the challenges to making such improvements?

• How can we address the challenges?

• How can this Visual Model be used or improved in

making changes and addressing challenges?

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