Rare Disease
Expertise: Rare Disease 86 results
Expert speakers discuss operational, ethical, and regulatory factors that shape effective data-return strategies.
Rare Disease
2026 Rare Disease Day Forum
Rare Disease
Returning Patient Data in Rare Disease Studies
Returning individual results to participants is gaining momentum as a core expression of patient-centered research. In rare disease development, where participants often contribute significant time, personal risk, and emotional investment, access to personal study data is increasingly seen as part...
Rare Disease
World Symposium
Learn how evolving FDA programs, modern trial designs, and patient-centered practices are reshaping rare disease development.
Navigating rare disease drug development requires coordinated, adaptive strategies as science and regulation evolve in parallel. Small, geographically dispersed patient populations, limited natural history data, and heterogeneous disease progression make trial design and execution unusually complex. At the same time,...
Since PDUFA VII was signed into law in September 2022, the U.S. Food and Drug Administration (FDA) has introduced a series of initiatives aimed at addressing the persistent challenges of rare disease development. Collectively, these efforts form a more complete...
In rare disease research, every patient counts. Recruitment is challenging, retention even harder, and each data point is invaluable. Yet rigid trial protocols often create unnecessary barriers for patients, families, and sites alike. For rare conditions where participants are geographically...