Expertise: Rare Disease 88 results
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
Rare disease development is entering a new era—one defined by clearer regulatory expectations, deeper patient involvement, and a growing emphasis on feasibility, flexibility, and transparency. In this eBook, we examine how evolving FDA programs, modern trial designs, and patient-centered practices...
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...
Each year on September 17th, the World Health Organization recognizes World Patient Safety Day to raise awareness and promote action to reduce patient harm. This year’s theme, “Ensuring safe care for every newborn and child,” highlights the importance of protecting...