RE:RE:RE:Clinical Trial Size in Orphan and Rare Diseases Innovative strategies for accelerating orphan drug development and FDA approval are crucial to bringing life-changing therapies to patients with orphan/rare diseases.
FDA guidance states that where a potential surrogate endpoint exists that is correlated with the primary endpoint, and the primary endpoint itself is difficult or slow to ascertain, an adaptive design can be based on the potential surrogate endpoint.
For example, in a trial with a primary endpoint of overall survival in which median survival time and tumor response (e.g. complete or partial response) is anticipated to predict clinical benefit, adaptive features such as sample size reassessment could be based on tumor response rather than mortality. ‘
Phase II/III trials that are adaptive in design, randomized controlled, and using surrogate clinical endpoints to predicitively determine treatment outcome were considered valuable alternatives to a relatively small sample size and overall survival in orphan diseases and rare cancers by 80% of oncologists.
By employing adaptive trial designs, utilizing real-world data, forming collaborative research networks, and engaging with the FDA early in the process, pharmaceutical companies can overcome the inherent challenges associated with orphan drug development.
ONCYs Phase 1/2 Goblet study was randomized, adaptive, biomarker driven and involving the surrogate endpoints of ORR, PFS and mOS that has been presented beforehand ... which satisfy the FDA and 80% of oncologists surveyed on an accelerated appproval pathway for rare cancers.