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Orphan disease definition FDA

While pharmaceutical companies make the majority of their profit from treating common diseases like cancer, heart disease, and diabetes, public policies in various nations have stimulated development of orphan drugs, which treat rare medical conditions. One example of this type of regulation is the Orphan Drug Act, which gives incentive for pharmaceuticals to develop drugs for diseases that affect a small population.

The Orphan Drug Act of 1983 defines a rare disease as a condition that affects fewer than 200, 000 people in the US. The Act provides the following benefits to a medication that has obtained Orphan Drug Designation:

• Seven-year marketing exclusivity after FDA approval of a designated drug

• Tax credit equal to 50% of clinical investigation expenses

• Waiver of PDUFA filing fee

• Assistance in development

• Grant funding

On June 12, 2013, the U.S. Food and Drug Administration (FDA) issued final regulations amending the 1992 Orphan Drug regulations implemented within the Orphan Drug Act. These modifications clarified certain regulatory language and made minor improvements to address issues raised since issuance of the regulations. The key points are highlighted here. For a full review of the Final Rule, please see the following link:

In the final rule, the FDA explained that the term “medically plausible” has been misinterpreted to mean “any medically recognizable or clinically distinguishable subset of persons with a particular disease or condition” and thus could lead to “artificially narrow subsets” and permit a non-rare disease to be “artificially subdivided into smaller groups for establishing subsets that are under the prevalence limit for designation.” This would result in “artificial orphan populations” obtaining orphan-drug designation and this would “divert resources away from research and development for true orphan diseases.” As a result, the term ‘medically plausible” has been removed from 21 CFR 316.20 (b)(6).

FDA has added a definition for “orphan subset” as follows: “Orphan subset of a non-rare disease or condition (“orphan subset”) means that use of the drug in a subset of persons with a non-rare disease or condition may be appropriate but use of the drug outside of that subset (in the remaining persons with the non-rare disease or condition) would be inappropriate owing to some property(ies) of the drug, for example, drug toxicity, mechanism of action, or previous clinical experience with the drug.” An orphan subset is a regulatory concept that is specific to Orphan Drug Regulations and not a medically recognizable or clinically distinguishable subset of persons with a particular disease.

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