Why do so many rare disease families, especially families of color, still feel unseen in research, and what would it take to build clinical trials that truly meet them where they are? In this episode, Laura Brencher, Leapcure’s VP of Advocacy, sits down with Dini Rao, co-founder of Colors of Hope and a Prader-Willi Syndrome (PWS) advocate, to explore what equitable research looks like when day-to-day caregiving is already a full-time job.

Dini shares how Colors of Hope was born from a simple but powerful goal; helping families feel less alone. Dini discusses how that community lens translates directly into better research participation: clearer education, trusted messengers, and study experiences designed around real constraints. Dini points to evidence that physician workforce diversity can have measurable health impacts, specifically, findings related to racial gaps in cardiovascular mortality (ref 1). Together, Laura and Dini unpack why representation isn’t just a “nice to have,” how sponsors and sites can reduce friction without sacrificing quality, and why listening to caregivers is essential to ethical, patient-centered progress in rare disease research.

In this episode, we cover:

• How community-building can remove barriers to research participation and reduce isolation for families
• What “patient-centered” means when caregivers are balancing complex, round-the-clock needs
• Why diversity in rare disease research requires trust, cultural humility, and sustained outreach
• Practical ways to make trials more accessible (communication, logistics, support) while protecting quality

To learn more about Colors of Hope, check out https://www.pwscolorsofhope.org/

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Reference:

1. Alsan, Marcella; Garrick, Owen; Graziani, Grant. “Does Diversity Matter for Health? Experimental Evidence from Oakland.” American Economic Review, 109(12), Dec 2019. https://www.aeaweb.org/articles?id=10.1257/aer.20181446

Pulmonary fibrosis trials are advancing quickly, but many studies still struggle to enroll and complete on time. In this episode, Zach Gobst sits down with Dr. Aaron Milstone, a pulmonologist and principal investigator, to talk about what enrollment really looks like from the site side and what it takes to support patients through participation with dignity, clarity, and trust.

Dr. Milstone shares how pulmonary research has become increasingly competitive, why thoughtful pre-screening against inclusion/exclusion criteria protects both patients and study quality, and how practical logistics for patients, like travel and lodging support, can be the difference between someone being able to participate or not. The conversation highlights what happens when recruitment is treated as a partnership with sites and patients, not just a pipeline.

In this episode, we cover:

• Why pulmonary fibrosis trials are especially competitive for enrollment and what that means for sites and sponsors
• The role of careful pre-screening to ensure the right patients are approached for the right studies
• How travel and lodging support can reduce participation barriers without compromising trial rigor
• What builds (and erodes) trust for patients considering research participation
• Why enrollment success is essential to completing studies and generating evidence patients can rely on

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Why are pregnant and breastfeeding women still so often excluded from clinical research, and what does that mean for real-world decision-making? In this episode, Laura Brencher, Leapcure's VP of Advocacy, sits down with Marie Teil, UCB's Global Head of Special Patient Populations, to unpack the long-standing evidence gap for women of childbearing age and what it takes to close it responsibly.

Marie shares why inclusion has to be intentional from the start, how earlier data generation supports more informed conversations between patients and clinicians, and what “ethical” truly looks like when patients are left without evidence. The conversation touches on growing global momentum, including evolving ICH guidance aimed at supporting the inclusion of pregnant and breastfeeding individuals in clinical trials and what leaders across the ecosystem can do today to move the field forward.

In this episode, we cover:

• Why excluding pregnant/breastfeeding populations creates real-world risk and uncertainty
• Designing inclusive studies as a default, not an afterthought
• Building trust-driven, evidence-based approaches that support patients and physicians
• Progress in the field and why upcoming global guidelines matter
• A principle to carry forward: protect women through research, not from research

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