Season 3 Episode 6: The State of Patient Engagement: From a Patient Advocate

This video provides an in-depth exploration of the critical role of patient engagement in clinical trials and pharmaceutical development, featuring a discussion between Manny Vazquez, Director of Strategy for Clinical Data at Veeva Systems, and Trishna Bharadia, a patient engagement consultant and advocate. The conversation is anchored by Trishna's personal journey, stemming from a life-changing multiple sclerosis diagnosis in 2008, which fueled her mission to champion patient-centered healthcare. The discussion highlights the increasing regulatory pressure from bodies like the FDA and EMA for sponsors to demonstrate patient preference integration into clinical research, underscoring the shift towards a more patient-centric paradigm in the life sciences.

The podcast delves into key themes such as good patient engagement practices, diversity, equity, and inclusion (DEI) in healthcare and research, and patient involvement in scientific publications. Trishna emphasizes that patient centricity, while a buzzword, holds immense value when implemented correctly. Effective patient engagement leads to better shared decision-making, accelerates medicines to market, makes clinical trials more suitable for participation, and improves adherence rates—a significant challenge in chronic illness management. She argues that the investment in patient engagement is minimal compared to the overall cost and time of drug development, yielding substantial returns in trial recruitment, retention, and ultimately, successful product development.

Throughout the discussion, Trishna provides compelling real-world examples to illustrate the profound impact of early and consistent patient involvement. She recounts the failure of an inhaled insulin product that, despite strong scientific backing, was withdrawn due to poor patient acceptance, primarily because practical patient needs (e.g., bulky inhaler, trust in administration) were not considered during design. Another example highlights a spasticity medication that, while effective in trials, failed in real-world use because patients with motor skill challenges could not self-administer it effectively. These cases underscore that patients, living with their conditions daily, identify critical factors that sponsors often overlook, from trial logistics (like icy car parks affecting site visits) to the practicalities of medication disposal in diverse geographic settings. The conversation also touches on the challenges of contracting, ensuring diversity of patient voices, and integrating patients into the publication process, advocating for a shift in mindset from "can't" to "how can we."

Key Takeaways:

• Regulatory Imperative for Patient Engagement: The FDA and EMA are increasingly demanding evidence of patient involvement and consideration of patient preferences in drug development and clinical research, making patient engagement a regulatory necessity, not just a best practice.
• Strategic Value of Early Patient Involvement: Engaging patients early and consistently throughout the product lifecycle (from research priorities and target product profiles to clinical trial design and technology selection) significantly improves trial recruitment, retention, and the likelihood of successful market adoption.
• Patient Engagement as an Investment: The time and financial resources required for effective patient engagement are a "drop in the ocean" compared to the overall cost of drug development, with returns far outweighing the investment through more efficient trials and better-accepted products.
• Define Objectives for Engagement Methods: Sponsors should clearly define their objectives before engaging patients, then select the most appropriate method (e.g., surveys for broad views, advisory boards for in-depth discussion, 1-on-1 interviews for specific insights) rather than defaulting to a single approach.
• Overcoming the "Can't" Mindset: A fundamental shift in organizational mindset from immediately thinking "we can't" (due to time, resources, legal, or compliance concerns) to "how can we make this happen" is crucial for fostering patient engagement.
• Addressing Challenges in R&D Engagement: Key challenges include streamlining contracting processes (using plain language templates), ensuring diversity of patient voices (through flexible working, training, language support, and considerate scheduling), and integrating patient input into scientific publications.
• Pragmatic Trial Design: Clinical trial design, particularly inclusion/exclusion criteria, should be more pragmatic and aligned with real-world patient populations, considering comorbidities, varying BMIs, and practical daily living challenges that can impact participation and adherence.
• Real-World Usability is Paramount: Drug efficacy proven in controlled trial settings does not guarantee real-world effectiveness if practical aspects of administration or daily use are not considered with patient input, as demonstrated by the spasticity medication example.
• Importance of Localization and International Representation: Designing trials and products with an understanding of diverse international patient circumstances, including environmental factors, infrastructure, and cultural nuances, is essential for global accessibility and success.
• Expanding Clinical Trial Awareness: Awareness of clinical trials remains a significant issue, often limited by HCP knowledge. Patient groups, educational initiatives (like the Fuse project), and accessible online resources are vital for empowering patients with information beyond traditional channels.
• Patient Involvement in Scientific Publications: Patients who contribute to the concept or design of clinical research should be considered for authorship on scientific publications, aligning with ICMJE criteria, though internal company processes often create barriers to this.
• Ethical Imperative for Patient-Centered Design: Beyond regulatory and commercial benefits, there is an ethical obligation to involve patients in the development of treatments that directly impact their lives, ensuring solutions are truly beneficial and usable.
• Equitable Access to Healthcare: The ultimate goal should be equitable access to healthcare and treatment, ensuring patient outcomes are not dictated by socio-economic factors, geography, language, or health literacy, thereby unlocking the full potential of modern medicine.

Tools/Resources Mentioned:

• Veeva Systems: The host's company, a leading platform in the pharmaceutical industry, particularly for CRM and clinical data.
• MRCT (Multi-Regional Clinical Trials Center): Mentioned for providing a brilliant glossary of clinical trial terms with lay language definitions and explanations.
• Fuse (Pistoia Alliance): A nonprofit organization focused on health and data science, involved in a project to raise awareness about clinical trial data transparency through infographics and videos.
• International Council of Medical Journal Editors (ICMJE): Their criteria for authorship are referenced in the context of patient involvement in scientific publications.

Key Concepts:

• Patient Centricity: A philosophy of designing healthcare around the patient's needs, preferences, and values.
• Patient Engagement: The active involvement of patients in various stages of healthcare, research, and drug development.
• Good Patient Engagement Practice: Methodologies and strategies for effectively and ethically involving patients, ensuring their input is meaningful and impactful.
• DEI in Healthcare and Research: Ensuring diversity, equity, and inclusion in patient engagement practices to represent a broad spectrum of patient experiences and needs.
• Pragmatic Trial Design: Designing clinical trials to better reflect real-world clinical practice and patient populations, often involving broader inclusion criteria and fewer rigid procedures.
• Site Centricity: A focus on optimizing the experience and efficiency for clinical trial sites, often seen as complementary to patient centricity.
• Health Literacy: The degree to which individuals have the capacity to obtain, process, and understand basic health information and services needed to make appropriate health decisions.

Examples/Case Studies:

• Inhaled Insulin Failure: A science-driven development program for inhaled insulin failed commercially despite FDA approval because patient preferences regarding the bulky inhaler, difficulty in dismantling, inconvenience, and distrust in the administration route were not considered early enough.
• Spasticity Medication Usability Issue: A medication for spasticity, effective in clinical trials when administered by a nurse, failed in real-world use because patients with motor skill issues could not effectively self-administer it with the puffer device at home, leading to its withdrawal and redesign.
• Winter Site Visit Drop-offs: A clinical trial experienced a drop in patient attendance during winter months because patients with mobility issues were afraid of falling on iced-over car parks when trying to reach the site entrance, a practical concern overlooked in trial design.
• Rural Medication Disposal: An example of a patient advocate in rural India highlighting the challenge of disposing of medical waste like syringes due to the lack of appropriate infrastructure, emphasizing the need for localization in product and trial design.