Patients as Partners: Improving Rare Disease Research Through Early Engagement

Patients as Partners: Improving Rare Disease Research Through Early Engagement

What happens when patients are involved in research from the very beginning, not just as participants, but as partners? In this episode of The Value Brief, host Rick Chapman [https://www.linkedin.com/in/richardchapman1/] is joined by Mousumi Bose, PhD [https://www.linkedin.com/in/mousumi-bose-phd/], Associate Professor at Montclair State University, and Kristie DeMarco [https://www.linkedin.com/in/kristie-demarco-90004b192/], Founder and President of the Global DARE Foundation [https://www.defeatadultrefsumeverywhere.org/], to discuss why meaningful patient engagement is essential to improving rare disease research, clinical trial design, and healthcare decision-making. Drawing on research expertise and lived experience, Mousumi and Kristie explore barriers to early patient engagement, the unique challenges of rare disease evidence generation, and how patient-centered research can lead to stronger evidence, better outcomes, and studies that reflect what matters most to patients and caregivers. The conversation also highlights the Rare Disease Patient Engagement (RDPE) Checklist, a practical framework designed to help researchers, patient organizations, industry leaders, and policymakers embed patient and caregiver perspectives throughout research and value assessment. Topics include patient engagement in healthcare research, rare disease evidence generation, patient-centered clinical trial design, caregiver perspectives, lived experience, building trust between researchers and patient communities, and strengthening value assessment and healthcare decision-making through patient input. Resources Rare Disease Patient Engagement (RDPE) Checklist [https://valueresearch.org/what-we-do/defining-solutions/rare-diseases/rare-disease-patient-engagement-guidance-and-checklist/]Watch the RDPE Checklist Video [https://valueresearch.org/new-video-rare-disease-patient-engagement-checklist/]Patient Collaborator Training Program [https://www.youtube.com/playlist?list=PLlXb_mFrp9Pz8YHAWZga88WbR6dqSHEXm]2025 ULF Family Conference: From Silence to Science [https://www.youtube.com/watch?v=YLDAhPw5f_o&t=1222s]

Beyond Price: Centering Patients in Drug Pricing Reform

Host: Tiffany Huth, Head of External Affairs, Center for Innovation & Value Research [https://valueresearch.org/] Guests: * Maia Laing, Chief Policy Officer, SickCells [https://sickcells.org/] * Silas Martin, Head of Access & Policy Research, Johnson & Johnson Episode Summary This episode explores how drug pricing reform can move beyond price to better reflect patient-centered value. Recorded following the 2025 Fall Policy Summit [https://valueresearch.org/events/2025-fall-policy-summit/], the discussion examines the role of lived experience, evidence, and innovation in shaping more equitable, sustainable drug pricing policy. What You’ll Hear * Why patient-centered value must be central to drug pricing reform * Where international pricing comparisons fall short for U.S. patients * How affordability, access, and innovation can coexist * The importance of patient-generated evidence alongside economic models Related Resources & Events J&J Center for U.S. Healthcare Policy Research: https://policyresearch.jnj.com/ [https://policyresearch.jnj.com/] Why Accounting for Heterogeneity Matters in U.S. Economic Analyses: https://policyresearch.jnj.com/why-accounting-for-heterogeneity-matters-in-u-s-economic-analyses [https://policyresearch.jnj.com/why-accounting-for-heterogeneity-matters-in-u-s-economic-analyses] SickCells Policy Forum (March 17–18) – Registration now open https://sickcells.org/policy-forum/ [https://sickcells.org/policy-forum/] Center’s 2026 7th Annual Methods Summit Valuing Innovation: Expanding Definitions of Value Wednesday, March 25, 2026, 9:00 AM – 5:00 PM EDT USC Capital Campus, Washington, DC  Register now to attend in-person [https://app.glueup.com/track/rd?type=campaign&lid=2&tracking_id=5402:389065:1a19c3af-cdd2-4562-ab49-016e39fb3a5c&redirect_url=https://app.glueup.com/event/2026-methods-summit-164561/&ts=1768492552&ps=d0tpOXhXdUVTODVISVJnMWN2Z2lXQjBqY0Q5WTNOTGxiVWF1ZTFQbDdCY1pSRXIxZ090REpRQzNCb0xFVVlLZG5nQXR2TXQxcTBNWmFZbUhZbFc4WWMyRXhEbU5xS1dvREpuNXRvVWtsUms9] or via livestream [https://app.glueup.com/track/rd?type=campaign&lid=3&tracking_id=5402:389065:1a19c3af-cdd2-4562-ab49-016e39fb3a5c&redirect_url=https://app.glueup.com/event/2026-methods-summit-virtual-164861/&ts=1768492552&ps=d0tpOXhXdUVTODVISVJnMWN2Z2lXQjBqY0Q5WTNOTGxiVWF1ZTFQbDdCY1pSRXIxZ090REpRQzNCb0xFVVlLZHcyL1ZjRTlGdWhzQm9Nb05haEdKdzB2eXJpTDF1Z2tEVDNadzNpeUlVUDg9].

AI in Rare Disease Research: The Power of Patient-Centered Co-Development

In this episode, we explore how artificial intelligence (AI) is reshaping rare disease research, from speeding up diagnosis and improving data collection to guiding more effective treatment decisions. Guests Tricha Shivas (Foundation for Sarcoidosis Research) and Michael Hager (Ready, Aim, Innovate) share insights on the promise and pitfalls of AI in healthcare. They discuss genetic breakthroughs, the importance of patient-centered engagement, and why co-developing AI tools with the communities they serve is essential to reducing bias and ensuring equitable innovation. The Patient-Centered Value Research Checklist for Rare Disease [https://valueresearch.org/what-we-do/defining-solutions/rare-diseases/] is now open for public comment. We’re seeking input from researchers, HTA professionals, clinicians, industry partners, and patient advocacy leaders to ensure this tool is practical, inclusive, and grounded in real-world priorities. The comment period runs from July 21 to August 18, 2025. Learn more and submit feedback today. [https://valueresearch.org/what-we-do/defining-solutions/rare-diseases/]

Caregiving in America: Inspired by Bradley Cooper, Centering Voices and Solutions

In this special episode of The Value Brief, released in tandem with the PBS documentary Caregiving, executive produced by Bradley Cooper, we spotlight the urgent and often overlooked challenges facing caregivers in the United States. Host Dr. Richard Chapman, Chief Science Officer at the Center for Innovation & Value Research [https://valueresearch.org/], is joined by Sarita Edwards [https://www.linkedin.com/in/saritakedwards/], President and CEO of the E.WE Foundation, and Mandy Barr [https://www.linkedin.com/in/mandy-fuller-barr-9285445a/], Engagement Specialist at the Rosalynn Carter Institute for Caregivers. Together, they share personal caregiving stories and offer expert insight into the systemic barriers that leave caregivers, especially those raising medically complex children, without adequate support. This powerful conversation explores: * The emotional, financial, and professional impact of caregiving * Gaps in public policy and employer practices * Strategies to center caregiver voices in healthcare decision-making Note: This episode was recorded before the official premiere of Caregiving. The film is now available to stream on PBS.org and PBS Passport.Learn more about the Center's Economic Impacts Project. [https://valueresearch.org/what-we-do/defining-solutions/economic-impact/]

Urgency, Equity, and Engagement: Advancing Patient-Centered Value Research

What does it truly mean to practice patient-centered research, and how can we ensure patient voices drive real healthcare change? In this episode of The Value Brief, host Larragem Raines, Director of Research at the Center for Innovation and Value Research, is joined by two leaders in the field: * Dr. C. Daniel Mullins [https://www.linkedin.com/in/c-daniel-mullins-b001297/], Executive Director of the PATIENTS Program at the University of Maryland School of Pharmacy * Dr. Magdalena Harrington [https://www.linkedin.com/in/magdalena-harrington-phd-98165265/], Senior Director of Patient-Centered Outcomes Assessment at Pfizer Together they discuss: * Defining patient-centered value beyond clinical outcomes * Turning patient insights into measurable, actionable healthcare improvements * The essential role of trust, communication, and storytelling in patient engagement * Moving from patient participation to true co-creation in healthcare research * What gives them hope—and urgency—for the future of value assessment This thoughtful conversation offers practical strategies for translating lived experiences into research, improving outcomes, and advancing equity in healthcare evaluation. Listen now to learn how patient voices can drive lasting change in healthcare systems. Learn more: Methods Summit Summary [https://valueresearch.org/wp-content/uploads/2025/05/2025-Methods-Summit-Summary_FINAL.pdf]