Enhancing the Value of Patient Input into Medicines Research & Development - #PatientsEU2019 - The Conference Forum

Enhancing the Value of Patient
Input into Medicines Research &
          Development

                Luther T. Clark, MD
           Deputy Chief Patient Officer
         Office of the Chief Patient Officer
                        MSD

                 29 January 2019

                                               #PatientsEU2019

Patient Centered/Centricity

Two separate but related contexts: medical care and research
  § Medical Care:
   o engagement of patients in managing their own health and
      healthcare
   o consideration of patient preferences and values when making
      medical decisions
  § Research:
   o inclusion of patients, caregivers, and patient advocates advising on
      all trial aspects: formulating research questions, study design, trial
      conduct, evaluating trial conduct, disseminating study results

                                                           #PatientsEU2019

Patient Engagement in Research and
              Development
• Continues to gain momentum as a way to improve
  outcomes
• Help innovations to be better solutions, not just better
  drugs or vaccines
• Aid in supporting the value of products
   o Regulators and reimbursement authorities: FDA and EMA formally
      seek patient perspectives when making registration/reimbursement
      decisions
   o Payers, prescribers, and patients: Choosing among alternatives in an
      increasingly complex ecosystem under intense financial pressures

                                                             #PatientsEU2019

Consequences of not Engaging
                Patients
Potential Opportunity Costs:
• Failure to realize greater efficiencies in recruitment and
  retention
• Making wrong choices, wasted effort pursuing wrong
  target or path; need for costly protocol amendments
• Gaps in understanding on part of patient and missed
  opportunity for education
• Gaps in understanding that can lead to gaps in trust

                                                   #PatientsEU2019

ASSESSING MEANINGFUL PATIENT ENGAGEMENT IN DRUG
                                       DEVELOPMENT: A DEFINITION, FRAMEWORK, AND RUBRIC
                                       Eleanor M. Perfetto, PhD, MS, Elisabeth M. Oehrlein, BA

ELEMENTS:

Patients as Partners: Patients, caregivers, and other relevant people (e.g., people at risk for a
disease, but do not yet have the disease) recognized as partners in the drug development process throughout
the life cycle.

Continuous Patient Engagement: PE is continuous, throughout the drug development process
and product lifecycle; not a one-time or sporadic event

Meaningful Patient Engagement: PE must be meaningful: a real interaction and dialogue, not
a “check-the-box” exercise. Patient input should come from thoughtful dialogue and patients should be able to
see how the input they provide is used in the specific studies or in the development processes.

The Right Patients are Engaged: Throughout the process, the target patient population is well
represented, and other relevant populations are considered for engagement.

The Right Time to Engage: Engagement at the appropriate time(s) throughout the process.
M-CERSI Conference on Patient-Focused Drug Development Planning Committee
University of Maryland School of Pharmacy, Department of Pharmaceutical Health Services Research, Baltimore, MD; National Health Council,
Washington DC
                                                                           6

Opportunities for Patient Input Across
               the Product Life Cycle

Discovery and     Clinical Development                               Life-Cycle Management
Pre-Clinical      (Phases I to III)            Approval and
                                               Launch                - Contributing to benefit:risk profile
- Understanding   - Understanding                                    - Supporting access and
  unmet need        patient/caregiver          - Contributing to     reimbursement
                    knowledge and experience      decisions on
- Informing                                       licensure and      - Facilitating treatment adherence
  product           with their condition and                         - Developing packaging, education
                    with clinical research        labelled
  design,                                         indications        tools, health-literate labeling,
  components,     - Designing protocols to                           medication guides
  stability,        optimize recruitment,      - Supporting launch
                                                 excellence          - Informing policy
  schedule          retention, compliance                            - Supporting manufacturing
                  - Including endpoints                              changes
                    meaningful to                                    - Guiding decisions on retaining or
                    patients/caregivers                              discontinuing licensed products

                   Across the product lifecycle: understanding value

                                                                                  #PatientsEU2019

Patient Inspired Innovation

    … listening, learning and
  collaborating with patients,
caregivers and patient groups …

                          #PatientsEU2019

Benefits of Sponsor/Patient
                Collaboration
Creates potential for developing studies that are scientifically
rigorous and more satisfying for patients

Sponsors’ learnings from patients:
§ How patients experience their diseases
§ What defines “success” from the perspective of patients
§ Impact of the trial design/conduct on patients’ lives
§ Modifications needed to increase recruitment, retention,
  compliance and trial experience
§ What data is most meaningful to patients

                                                   #PatientsEU2019

Examples of Engagements for
           Collecting Insights
§ Patient Dialogues
 o Facilitated input sessions (live or e-channel) with patients and/or
   caregivers to obtain their insights on specific topics to inform trial
   design and strategy
§ Patient Input Forums (PIFs)
 o Live interviews with physician and patient to discuss a “real-world”
   patient journey for a given disease or condition
§ Individual Patient/Advocate Forums
 o Live forums/meetings with individual patients/advocates
§ Patient Advisory Boards
§ Other Engagements (i.e. Surveys, Market Research)

                                                           #PatientsEU2019

Best Practice Considerations
§ Clarity about questions to be asked, why asked, and how the
  info obtained will be used
§ Third-party vendors with expertise in patient engagement
  can be valuable
§ Patients may be blinded to or aware of the sponsor company
   o When patients blinded to the company, there may be less bias
§ Consideration of needs of key populations; ensure patients
  engaged representative of affected populations
§ Types of engagements
   o In-person: researchers can interact directly with patients to
     understand potential issues with trial design, recruitment, retention
   o Virtual or e-meetings: larger numbers of patients can be reached in
     shorter time; no geographic limitations

                                                           #PatientsEU2019

Patient Dialogue: Case Example
§ Half-day 4-hour workshop
§ 8 Participants/patients with breast cancer
§ One aspect of a program to better engage
  patients and caregivers
§ Purpose of Workshop:
   o to understand patients’ needs and incorporate them
     into decision making processes for research and
     development.
   o To obtain input into design features of a clinical study
§ Tone was informal and creative.
§ Activities designed to stimulate lively discussion
  relating to motivations, barriers, and behaviors
  of this population.
                                                  #PatientsEU2019

Patient Dialogue: High Level Findings

Patient Dialogue: Key Learnings

§ Current patient perspectives and expectations of treatment
§ Understanding of patient knowledge and perceptions
  regarding clinical trials
§ Gained insight into patients’ source preferences for
  information about clinical trials and perceptions of the
  credibility of these sources.
§ How patients behave when evaluating whether to participate
  in a clinical trial, including:
      - Drivers of decision making;
      - Role of influencers (i.e., family) in this decision.
      - Barriers to participation in a clinical trial.
§ Increased understanding of patient perceptions of key
  aspects of clinical trial design
                                                               #PatientsEU2019

Merck Changes The Paradigm On Clinical Trials
“When you begin to look at a trial from the
patient’s perspective and consider the
complexity of the trial and what we are
asking them to do, is it any wonder that so
many patients are refusing to participate?
And is it any wonder why so many patients
choose to withdraw from a clinical trial?”

“Patients have a voice, and that voice is
very important. It needs to be embraced
and not just as an industry buzzword. It will
be a very valuable contribution to how we
run clinical trials in the future.”

Mark Travers, Global Head of Monitoring Excellence and
Interim Head of North America Clinical Operations, Merck.
Clinical Leader, May 11, 2016

                                                            #PatientsEU2019

Merck Changes The Paradigm On Clinical Trials

§ Mindset Shifting:

§ Making it easier for partners:

§ Embracing new technologies
  and platforms: remote
   patient/physician interactions; more
   efficient medication delivery; point of
   care diagnostics; wearable devices
   for trials; piloting of system using a
   sensor, tablet and Wi-Fi; eConsent;
   providing information in multimedia
   approach, using text, pictures, or
   cartoons
Mark Travers, Global Head of Monitoring Excellence and
Interim Head of North America Clinical Operations, Merck.
Clinical Leader, May 11, 2016

Pa Partnershipsrships
                                                       • Patients,
                    • Academic                      Caregivers and            • Community
                     Research                      Patient Advocacy               Based
                      Centers                        Organizations            Organizations

   • Investigator                                                                          • Industry
        Sites

•“Coming together is a beginning; keeping together is progress; working together is success” – Henry Ford

The Goal
… end to end, global, bi-directional
engagement with patients/caregivers to
optimize and realize greater innovation and
efficiency

… putting the individual patient and caregiver
at the center of our decision-making.

                                     #PatientsEU2019

Key Takeaways
§ Listening to patients and caregivers is critical for inspiring
  and informing effective and relevant medical and scientific
  innovation
§ Sponsor/Patient collaboration creates potential for studies
  that are both scientifically rigorous and more satisfying for
  patients
§ Potential consequences for not engaging patients include
  lost opportunity costs (efficiencies, gaps in understanding
  and patient trust)

                                                   #PatientsEU2019

Thank You!

             #PatientsEU2019