FDA drafts guidance explaining 3-year clinical investigation exclusivity for new drugs

In a new draft guidance, the US Food and Drug Administration (FDA) has provided details on the clinical investigation requirements that sponsors of new drugs must meet to obtain three years of marketing exclusivity for their products.

Under the 1984 Hatch-Waxman Amendments, certain new drug applications (NDAs) or supplements to NDAs qualify for three-year marketing exclusivity, which prevents any abbreviated new drug applications (ANDAs) from being approved during that period. FDA notes that NDAs and sNDAs that qualify for exclusivity must also include information from new clinical investigations that are not bioavailability studies, are essential to the approval of the NDA or sNDA, and are conducted or sponsored by the applicant.

On 4 March, FDA published a draft question-and-answer guidance to clarify requirements for receiving the drug marketing exclusivity. In addition to stating what applications are eligible for the three-year exclusivity and whether sNDAs qualify as well, the agency explains that non-bioavailability studies may qualify as clinical investigations to support the drug marketing exclusivity, though it notes that the drug in the clinical investigation does not need to be the same as the drug that was approved for the application.

“For example, a clinical investigation that used a similar or earlier version of the drug product in development, which contains the same active moiety or combination of active moieties as the version of the drug in the approved application, may potentially qualify the approved drug product for 3-year exclusivity,” said FDA. “To illustrate, if a clinical investigation used a granule formulation of a drug, but the NDA at issue is seeking approval for a tablet dosage form of that drug, which could be crushed and administered similarly to the granule formulation, the clinical investigation nevertheless may qualify the tablet for 3-year exclusivity, provided the statutory and regulatory criteria are met.”

FDA also emphasized that trials that only administer a placebo to study participants would generally not be considered a clinical investigation that can be used for the purpose of obtaining marketing exclusivity. The agency also explained what clinical trial information it expects applicants to provide in their NDA applications.

“To show that the NDA contains new clinical investigations, the applicant must include a certification that to the best of its knowledge: (1) each of the clinical investigations is an investigation in humans; (2) the results of which have not been relied on by FDA to demonstrate substantial evidence of effectiveness of a previously approved drug product for any indication or of safety for a new patient population; and (3) do not duplicate the results of another investigation that was relied on by the Agency to demonstrate the effectiveness or safety in a new patient population of a previously approved drug product (see QE.1 on the process for requesting 3-year exclusivity),” said FDA.

The FDA said that it considers a clinical investigation essential to the approval of a marketing authorization if it does not have enough information to decide whether the drug under review is safe and effective without it. The agency said it does not consider an investigation essential for approval just because the sponsor conducted and submitted it in its application or supplement.

Ultimately, if a sponsor thinks they are eligible for the three-year exclusivity, FDA said they must submit a list of all relevant published studies or publicly available reports of clinical investigations, a certification that they conducted a thorough scientific literature search, and an explanation as to why the studies or reports identified from the literature search are insufficient for FDA to review their product.

Draft guidance

FDA drafts guidance explaining 3-year clinical investigation exclusivity for new drugs

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