Quantum of Effectiveness Evidence in FDA’s Approval of Orphan Drugs

Cataloguing FDA’s Flexibility in Regulating Therapies for Persons with Rare Disorders
by Frank J. Sasinowski, M.S., M.P.H., J.D., Chairman of the Board, National Organization for Rare Disorders

One of the key underlying issues facing the development of all drugs, and particularly orphan drugs, is what kind of evidence the Food and Drug Administration (FDA) requires for approval. The Federal Food, Drug, and Cosmetic [FD&C] Act provides that for FDA to grant approval for a new drug, there must be “substantial evidence” of effectiveness derived from “adequate and well-controlled investigations.” This language, which dates from 1962, provides leeway for FDA medical reviewers to make judgments as to what constitutes “substantial evidence” of a drug’s effectiveness, that is, of its benefit to patients.
The sole law that applies specifically to orphan drugs, the Orphan Drug Act of 1983, provided financial incentives for drug companies to develop orphan drugs, which is legally defined as products that treat diseases that affect 200,000 or fewer patients in the U.S. But the Orphan Drug Act, whose enactment was championed by the National Organization for Rare Disorders (NORD), did not amend or revise the statutory standards in the law for establishing that a new medicine is safe and effective for its proposed use. From a strict regulatory standpoint, the standard for orphan drugs is identical to the standard required for all other drugs, namely that “substantial evidence” demonstrates the effectiveness of the drug for its intended uses.

Read the Full Report


The NORD Press Release:


Released at Major Conference, Study Catalogues Flexibility in Orphan Drugs Approved Since 1983
Washington DC, October 11, 2011 – The National Organization for Rare Disorders (NORD) today released a landmark report documenting flexibility in the Food and Drug Administration (FDA) review of potential treatments for patients with rare diseases.

Released at the U.S. Conference on Rare Diseases and Orphan Products, the report examined the basis for FDA’s approval of 135 non-cancer “orphan drugs” – those for rare diseases – since the Orphan Drug Act was enacted in 1983 to provide incentives to encourage development of treatments for rare diseases.

This is the first study of its kind ever conducted and the first time that there has been a systematic examination of the basis for approval for any category of drug products extending over such a long period of time. The study demonstrates a decades-long pattern of flexibility in FDA review of orphan drugs.

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