USA Law and Practice Contributed by: Daniel Kracov, David Marsh and Alice Ho, Arnold & Porter
3.2 Marketing Authorisation for Biologic Medicinal Products Drug products are approved via New Drug Applica- tions (NDAs). Additional indications, dosage forms, etc, may be added via NDA supplements. Biological products are approved in a virtually identical process via Biologics Licence Applications (BLAs). The stand- ard for approval is “substantial evidence” of safety and effectiveness (technically, “safety, purity and potency” for biologics), resulting from at least one – and typical- ly more – adequate and well-controlled clinical stud- ies. The typical drug or biologic review process takes ten months after initial acceptance for filing (a 60-day period); however, a priority review of six months is given to certain drugs and biologics intended to treat serious or life-threatening conditions, and under the recently introduced, non-transferable Commissioner’s Priority Review Voucher programme certain designat- ed products may receive greatly accelerated review. Substantial user fees – USD4,682,003 in fiscal year 2026 for an NDA or BLA containing clinical data – are required to facilitate a review of applications. 3.3 Period of Validity of Marketing Authorisations There is no mandatory re-authorisation process per se for approved products in the absence of changes to the product that require a supplemental filing before implementation. However, the FD&C Act and FDA regulations include processes for the withdrawal or revocation of an approval based upon a significant safety or effectiveness issue or non-compliance with approval requirements. These processes can be expe- dited in certain scenarios, such as an applicant’s fail- ure to confirm the efficacy of an accelerated approval product in a post-market study, or where there is an imminent hazard. In general, a marketing authorisation may not be revoked merely because the product has not been placed on the market – although a failure to market an orphan drug could result in a loss of orphan exclusivity. 3.4 Procedure for Obtaining a Marketing Authorisation As noted in 3.2 Marketing Authorisation for Biologic Medicinal Products , the pathways for approval of drugs consist of:
• the submission of an NDA (including a 505 (b)(2) NDA relying on data for which the applicant does not have a right of reference); and • the Abbreviated New Drug Application (ANDA) for generic products, which demonstrates equivalence to a reference listed drug. A biologic is licensed via the submission of a BLA; however, that process is largely the equivalent of an NDA submission. A biosimilar application demon- strates that, based on the totality of the evidence, the biosimilar is either “highly similar” to – or interchange- able with – a reference biologic, although the FDA is generally waiving the need for separate interchange- ability switching studies. The FDA is authorised to require paediatric studies of drugs or biologics when other approaches are insuffi- cient to ensure that the products are safe and effective for use in children. The agency may also issue a writ- ten request for paediatric research and, if the sponsor fulfils the data request, it may obtain six months of paediatric exclusivity. As noted, changes to an existing marketing authorisa- tion may be obtained through supplements or amend- ments to existing applications. As regards medical devices, the submission of additional 510 (k) submis- sions can result in the clearance of significant changes to previously cleared device products. A PMA may also be supplemented or amended. In many cases, the transfer of a clearance or approval without manu- facturing site or significant product changes requires only fairly simple notifications to the FDA. 3.5 Access to Pharmaceuticals and Medical Devices Without Marketing Authorisations The FDA maintains regulations permitting expanded access to investigational products. Such expanded access to INDs and IDEs may relate to an individual patient (often called a “compassionate use”) or may allow broader use by patients not eligible for controlled clinical trials, depending upon the seriousness of the disease and the availability of alternative treatments. Sponsors of such INDs may not charge patients for the investigational drug without specific authorisation from the FDA permitting cost recovery only.
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