RegulatoryGlossary Term
A special status granted by the FDA to drugs and biologics intended to treat rare diseases affecting fewer than 200,000 people in the United States, providing incentives for development.
Orphan Drug Designation was established by the Orphan Drug Act of 1983 to address a market failure in pharmaceutical development: rare diseases, while individually uncommon, collectively affect millions of patients, yet the limited market size for any single rare disease often made development economically unfeasible. The orphan drug program provides incentives that offset the commercial challenges of rare disease drug development, resulting in hundreds of new treatments for conditions that might otherwise have remained without approved therapies.
The incentives associated with Orphan Drug Designation are substantial and have proven effective in stimulating rare disease drug development. Designated products receive seven years of marketing exclusivity upon approval, during which the FDA will not approve the same drug for the same indication from another company, even if patent protection has expired. Additional incentives include tax credits for clinical trial costs, waiver of application fees, and eligibility for grants supporting clinical research. These benefits can significantly improve the economic viability of rare disease drug development.
Obtaining Orphan Drug Designation requires submission of a request demonstrating that the drug is intended for a disease affecting fewer than 200,000 people in the United States, or for which there is no reasonable expectation that development costs will be recovered from U.S. sales. The request must include a scientific rationale for the drug's proposed use and evidence supporting its potential benefit for the target population. Designation is granted before marketing approval and can be requested at any stage of development, though early designation maximizes the available benefits. The designation status continues through approval and the exclusivity period, provided the sponsor demonstrates good faith efforts to develop and make the drug available.
Rare disease development
"The enzyme replacement therapy received Orphan Drug Designation for treatment of the lysosomal storage disorder, which affects approximately 5,000 patients in the United States, providing seven years of marketing exclusivity upon approval."
Development incentives
"With Orphan Drug Designation in hand, the biotech company was able to claim tax credits covering 25% of clinical trial costs, improving the financial feasibility of the development program for the rare genetic condition."
A premarket submission to the FDA demonstrating that a medical device is substantially equivalent to a legally marketed predicate device and therefore does not require premarket approval.
An FDA pathway allowing approval of drugs for serious conditions based on a surrogate endpoint or intermediate clinical endpoint reasonably likely to predict clinical benefit, with post-marketing requirements to confirm the expected benefit.
A regulatory submission to the FDA requesting approval to market a biological product in the United States, demonstrating that the product meets standards for safety, purity, and potency.
An FDA program for drugs intended to treat serious conditions where preliminary clinical evidence indicates substantial improvement over available therapies on clinically significant endpoints.
An FDA program designed to expedite the development and review of drugs intended to treat serious conditions and fill an unmet medical need, providing increased communication with FDA and eligibility for Rolling Review.
A special status granted by the FDA to drugs and biologics intended to treat rare diseases affecting fewer than 200,000 people in the United States, providing incentives for development.
Orphan Drug Designation was established by the Orphan Drug Act of 1983 to address a market failure in pharmaceutical development: rare diseases, while individually uncommon, collectively affect millions of patients, yet the limited market size for any single rare disease often made development economically unfeasible. The orphan drug program provides incentives that offset the commercial challenges of rare disease drug development, resulting in hundreds of new treatments for conditions that might otherwise have remained without approved therapies.
The incentives associated with Orphan Drug Designation are substantial and have proven effective in stimulating rare disease drug development. Designated products receive seven years of marketing exclusivity upon approval, during which the FDA will not approve the same drug for the same indication from another company, even if patent protection has expired. Additional incentives include tax credits for clinical trial costs, waiver of application fees, and eligibility for grants supporting clinical research. These benefits can significantly improve the economic viability of rare disease drug development.
Obtaining Orphan Drug Designation requires submission of a request demonstrating that the drug is intended for a disease affecting fewer than 200,000 people in the United States, or for which there is no reasonable expectation that development costs will be recovered from U.S. sales. The request must include a scientific rationale for the drug's proposed use and evidence supporting its potential benefit for the target population. Designation is granted before marketing approval and can be requested at any stage of development, though early designation maximizes the available benefits. The designation status continues through approval and the exclusivity period, provided the sponsor demonstrates good faith efforts to develop and make the drug available.
Rare disease development
"The enzyme replacement therapy received Orphan Drug Designation for treatment of the lysosomal storage disorder, which affects approximately 5,000 patients in the United States, providing seven years of marketing exclusivity upon approval."
Development incentives
"With Orphan Drug Designation in hand, the biotech company was able to claim tax credits covering 25% of clinical trial costs, improving the financial feasibility of the development program for the rare genetic condition."
A premarket submission to the FDA demonstrating that a medical device is substantially equivalent to a legally marketed predicate device and therefore does not require premarket approval.
An FDA pathway allowing approval of drugs for serious conditions based on a surrogate endpoint or intermediate clinical endpoint reasonably likely to predict clinical benefit, with post-marketing requirements to confirm the expected benefit.
A regulatory submission to the FDA requesting approval to market a biological product in the United States, demonstrating that the product meets standards for safety, purity, and potency.
An FDA program for drugs intended to treat serious conditions where preliminary clinical evidence indicates substantial improvement over available therapies on clinically significant endpoints.
An FDA program designed to expedite the development and review of drugs intended to treat serious conditions and fill an unmet medical need, providing increased communication with FDA and eligibility for Rolling Review.