PhRMA, Novartis weigh in on FDA’s 3-year exclusivity guidance
The Pharmaceutical Research and Manufacturers of America (PhRMA) said the US Food and Drug Administration’s (FDA) recent draft guidance on marketing exclusivity for new clinical investigations provides essential clarity on the types of applications that are eligible for the incentive. However, both PhRMA and Novartis highlighted that additional clarification is still necessary on some topics.
The guidance released in March, presented in a question-and-answer format, outlines the statutory and regulatory criteria for eligibility to obtain this three-year exclusivity and clarifies the processes for applicants to request it. (RELATED: FDA drafts guidance explaining 3-year clinical investigation exclusivity for new drugs, Regulatory Focus 4 March 2026).
Under the 1984 Hatch-Waxman Amendments, certain new drug applications (NDAs) and NDA supplements are eligible for three years of marketing exclusivity. This exclusivity prevents any abbreviated new drug applications (ANDAs) from being approved during that time. The FDA specifies that NDAs and supplemental NDAs (sNDAs) qualifying for this exclusivity must include information from new clinical investigations that are not merely bioavailability studies. The guidance provides clarity on what qualifies as a new clinical investigation.
PhRMA supports guidance
PhRMA stated that the guidance provided needed clarity on the types of investigations that may qualify for three-year exclusivity. For example, the guidance clarifies that even if the drug used in the clinical investigation is not the same as that approved in the application, it is still considered the same in certain situations.
The guidance states that “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.”
The group said it “supports FDA’s position because it reflects the practical realities of drug development. It recognizes that clinical investigations may appropriately use product variants—even where those differ from the final approved product—and that such efforts can nonetheless generate evidence essential to approval of the product ultimately approved and meeting the threshold for 3-year exclusivity.”
PhRMA also said it supports FDA’s clarification in the guidance that certain studies with pharmacokinetic (PK) endpoints may receive three-year exclusivity. In the draft guidance, FDA confirms that “an investigation assessing both PK/bioavailability and clinical safety and/or effectiveness” may “qualify as a clinical investigation for 3-year exclusivity.”
Yet PhRMA requested that FDA provide additional guidance on whether the scope of three-year exclusivity applies to certain antibiotics.
The group states that “the Draft Guidance expressly states that it does not address issues concerning eligibility of certain antibiotics for 3-year exclusivity under section 505(v) of the FDCA. PhRMA would also find additional guidance on FDA’s interpretation of section 505(v) helpful and requests FDA issue further guidance on this topic.”
Novartis sought clarification on whether new pediatric efficacy supplements for oncology drugs can qualify for three-year marketing exclusivity.
The company states that “the guidance is silent on whether new pediatric efficacy supplements for Molecularly Targeted Oncology Drugs (under Amendments to Sec. 505B of the FD&C Act) are in scope of 3-year exclusivity for drug products. Novartis proposes that the FDA add a Q&A outlining their position on the applicability of new clinical investigation exclusivity for pediatric molecularly targeted oncology products.”
