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