Orphan Drug Designation

Orphan Drug Act (ODA) grants a special status or designation to those drugs or biological products that are used to treat rare diseases or conditions when a sponsor request for it. This designation is referred as Orphan Designation or Orphan Status. To qualify for this designation, the drug and disease/condition must meet criteria mentioned in ODA and implementing regulations of FDA at 21 CFR Part 316.

This designation provides the sponsor many development incentives for the drug as per ODA, which includes tax credits for qualified clinical testing. The marketing application of a prescribed drug product that has received orphan designation is not subject to prescription drug consumer fee, unless it is used for other indication than the rare disease.

If a sponsor wants to submit a request for orphan designation of a drug, they can do so by providing information mentioned in 21 CFR 316.20 and 316.21. Sponsors who are submitting request for orphan designation for a same product and for same rare disease, must submit their own information and data to support their request. The granting of orphan designation does not alter the requirements and criteria of standard regulatory and of getting marketing approval. The safety and efficacy of a drug must be developed via adequate and controlled studies.

Some of the drugs to obtain Orphan Drug Designation (ODD) are as follows:

  • Vorinostat has got ODD for treatment of T-cell non-Hodgkin's lymphoma.
  • Rituximab has got ODD for treatment of patients with anti-neutrophil cytoplasmic antibody-associated vasculitis (Wegener's Granulomatosis, Microscopic Polyangiitis, and Churg-Strauss Syndrome) and for other orphan diseases as well.

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