Drugs

Rare Diseases Drug Development Draft Guidance

FDA has issued a draft guidance, Rare Diseases: Common Issues in Drug Development, to assist sponsors of drug and biological products for the treatment or prevention of rare diseases in conducting more efficient and successful drug development programs. “Although the statutory requirements for marketing approval for drugs to treat rare and common diseases are the same and issues discussed in this guidance are encountered in other drug development programs,” the document says, “these issues are frequently more difficult to address in the context of a rare disease for which there is often limited medical and scientific knowledge, natural history data, and drug development experience.”

The draft revises and replaces an 8/2015 drug guidance by updating the natural history studies section; inclusion of issues for evaluation and validation of surrogate biomarkers; description of nonclinical flexibility; additional information on external controls and early randomization; addition of a safety section; retitled chemistry, manufacturing, and controls section to pharmaceutical quality considerations; additional information on changes to drug substance or manufacturing process with clarification on areas of flexibility; and inclusion of an additional considerations section addressing several topics — participation of patients, caretakers, and advocates, consideration of pediatric issues, and interactions with FDA.

The guidance addresses the importance of several elements in development programs for rare diseases: adequate description and understanding of the disease’s natural history; adequate understanding of the pathophysiology of the disease and the drug’s mechanism of action; nonclinical-pharmacotoxicology and human toxicology considerations to support the proposed clinical investigation or investigations; selection or development of outcome assessments and endpoints; evidence to establish safety and effectiveness; drug manufacturing considerations during drug development; participation of patients, caretakers, and advocates in development programs; and interactions with FDA.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome