Overview

• Called the Rare Disease Evidence Principles (RDEP), the process brings CDER and CBER into a joint review structure with input from a Rare Disease Policy and Portfolio Council.
• Eligibility is tightly limited to conditions driven by a known genetic defect that affect fewer than 1,000 people in the U.S., progress rapidly to significant disability or death, and lack adequate alternative therapies.
• Approvals may rely on one adequate and well-controlled study that can be single-arm, backed by strong confirmatory evidence such as mechanistic or biomarker data, relevant nonclinical models, clinical pharmacodynamic findings, case reports, expanded access data, or natural history studies.
• Sponsors must apply before initiating their pivotal trial, after which the FDA will convene discussions on study design and supporting evidence and may hold patient listening sessions when appropriate.
• Analysts welcome clearer expectations yet question how much faster approvals will become since single-arm strategies are already common and many programs will not qualify, though platform approaches could gain predictability.