FDA principles for rare disease drug development

A new U.S. Food and Drug Administration initiative may help tackle persisting challenges in the development of treatments for rare diseases.

Two FDA research centers — the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research — have developed the Rare Disease Evidence Principles with the goal of speeding up the review process for the genetic conditions and clarifying the types of evidence needed to demonstrate the efficacy of a drug. Approval of rare disease treatments will require positive findings from an appropriate study as well as evidence from mechanisms and biomarkers, nonclinical models, clinical pharmacodynamic data, case reports, expanded access data or natural history studies.

Prior to the initiation of a pivotal trial, sponsors must demonstrate that the therapies being investigated are designed to treat diseases that may result in disability or mortality who have significant unmet needs and no alternative treatment options in order to qualify for the process.

Read more: FDA

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