USA Law and Practice Contributed by: Daniel Kracov, David Marsh and Alice Ho, Arnold & Porter
In addition, the 2018 “Right to Try” Act theoretically permits certain eligible terminally ill patients to have broad access to eligible investigational drugs in cer- tain 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 traditional IND pathway. There is also a very limited Humanitarian Device Exemption (HDE) pathway for approval of a Humani- tarian Use Device (HUD) intended to benefit patients in the treatment or diagnosis of a disease or condition that affects – or is manifested in – not more than 8,000 individuals in the USA per year. 3.6 Ongoing Obligations Imposed by Marketing Authorisations Virtually every drug, biological or device product is subject to ongoing requirements relating to establish- ment registration, product listing, compliance with cGMP/quality systems, track-and-trace requirements, and safety reporting/adverse-event reporting regula- tions. In certain cases, the FDA may require closer, ongoing oversight of a drug or biologic under a risk evaluation and mitigation strategy (REMS) or may mandate post-market studies or trials. 3.7 Third-Party Access to Pending Applications for Marketing Authorisations While the FDA does release approval and “complete response” (ie, not ready for 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 information on approved products may be obtained via the FDA’s Drugs@FDA website. Often, extensive information about pending applica- tions is released in the form of briefing papers and presentations used at FDA Advisory Committee meetings. The FDA does not reveal the existence of pending INDs or IDEs unless the sponsor has publicly acknowledged the filings. Third parties may submit requests for information under the Freedom of Information Act (FOIA); how- ever, there are a variety of exceptions from disclo- sure, as well as a major FDA backlog of requests.
Most importantly, the FDA has an obligation under the FOIA to refrain from publication of trade secrets or confidential commercial or financial information. Sponsors/applicants are afforded an opportunity to review potential releases of information and request confidential treatment under those FOIA exceptions. 4. Regulatory Reliance and Fast-Track Registration Routes 4.1 Fast-Track Registration Routes There is an array of expedited programmes for the registration of medicines and medical devices. These programmes include the following. • Fast-track designation for drug and biological products for serious conditions where said prod- ucts demonstrate the potential to address an unmet medical need. • Designation as a breakthrough therapy in the case of drugs for serious conditions where preliminary clinical evidence indicates that the drug may dem- onstrate substantial improvement on a clinically significant endpoint over available therapies, or in the case of the breakthrough devices programme that applies to designated devices that provide for more effective treatment or diagnosis of life-threat- ening or irreversibly debilitating human disease or conditions, among other criteria. • Accelerated approval for products that treat a serious condition, provide a meaningful advan- tage over available therapies, and demonstrate a significant impact on a surrogate endpoint that is reasonably likely to predict clinical benefit or on a clinical endpoint that can be measured earlier than irreversible morbidity or mortality – although the clinical effectiveness of such products must be confirmed in post-market studies. • Priority review for drugs that treat a serious condi- tion and, if approved, would provide a significant improvement in safety or effectiveness (or if the FDA is presented with a priority review voucher). • The Commissioner’s National Priority Review Voucher, which is a non-transferable voucher awarded to applicants to accelerate review and approval of specific products that align with one of five critical US national health priorities. Benefits of
405 CHAMBERS.COM
Powered by FlippingBook