USA Law and Practice Contributed by: Daniel Kracov, David Marsh and Alice Ho, Arnold & Porter
of the evidence, the biosimilar is either “highly similar” to – or interchangeable with – a refer- ence biologic. The FDA is authorised to require paediatric stud- ies of drugs or biologics when other approaches are insufficient to ensure that the products are safe and effective for use in children. The agency may also issue a written request for paediat- ric research and, if the sponsor fulfils the data request, it may obtain six months of paediatric exclusivity. As noted, changes to an existing marketing authorisation may be obtained through supple- ments or amendments to existing applications. As regards medical devices, the submission of additional 510(k) submissions 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 manufacturing site or significant product chang- es 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 “com- passionate use” ) or may allow broader use by patients not eligible for controlled clinical tri- als, depending upon the seriousness of the disease and the availability of alternative treat - ments. Sponsors of such INDs may not charge patients for the investigational drug without spe- cific authorisation from the FDA permitting cost recovery only.
In addition, the 201 “Right to Try” Act permits certain eligible terminally ill patients to have broad access to eligible investigational drugs in certain circumstances when manufacturers are willing to supply. To date, most companies have shown a reluctance to permit their products to be used via this pathway in lieu of the more tra- ditional IND pathway. There is also a very limited Humanitarian Device Exemption (HDE) pathway for approval of a Humanitarian Use Device (HUD) intended to benefit patients in the treatment or diagnosis of a disease or condition that affects – or is mani- fested in – not more than 8,000 individuals in the USA per year. 3.6 Marketing Authorisations for Pharmaceuticals and Medical Devices: Ongoing Obligations Virtually every drug, biological or device prod- uct is subject to ongoing requirements relating to establishment registration, product listing, compliance with cGMPs/quality systems, track- and-trace requirements, and safety reporting/ adverse-event reporting regulations. In certain cases, the FDA may require closer, ongoing oversight of a drug or biologic under a risk eval- uation and mitigation strategy (REMS) or may mandate post-market studies or trials. 3.7 Third-Party Access to Pending Applications for Marketing Authorisations for Pharmaceuticals and Medical Devices While the FDA does release approval letters and – after review for redaction of confidential and trade-secret information – summary review and approval documents, it does not currently publish “complete response letters” that reject an application under review. Available informa- tion on approved products may be obtained via the FDA’s Drugs@FDA website. Often, exten-
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