Rare disease programs force teams to confront operational realities much earlier in development, particularly when advanced modalities, limited patient populations, and complex site requirements intersect.

In this episode of The Emerging Biotech Leader, hosts Kim Kushner and Ramin Farhood are joined by Brendan Slagle, Senior Director, Medical at SSI Strategy to discuss why operational feasibility needs to be built into study design from the beginning rather than addressed later during execution.

Site readiness, patient burden, follow-up care, delivery logistics, and cross-functional alignment all become critical much earlier in development, especially in studies where there is little room for operational inefficiency.

Breaking down silos across clinical operations, medical affairs, regulatory, investigators, and patient communities becomes critical in these settings. Waiting too long to involve these perspectives can create studies that are scientifically sound but difficult to execute in practice.

Designing studies that patients and caregivers can realistically participate in becomes critical for generating meaningful data and sustaining enrollment over time. Generating meaningful data depends on designing studies that patients and caregivers can realistically participate in, while balancing scientific ambition with operational feasibility. Many of these lessons increasingly apply beyond rare disease as clinical development grows more complex across therapeutic areas.

Successful programs depend not only on scientific innovation, but on early alignment, operational realism, and a clear understanding of what patients and sites can actually sustain.

What can rare disease development teach the rest of the industry about trial execution? Listen to the full episode.