Current regulatory status in China of drugs withdrawn from the US FDA accelerated pathway: implications for regulation globally

In 1992, in an effort to bring novel drugs to market more quickly and meet patient needs in a timely manner, the US Food and Drug Administration (FDA) pioneered an accelerated approval initiative on the basis of surrogate endpoints that are reasonably predictive of clinical benefit for the treatment of serious or life-threatening diseases. 1 US Food and Drug AdministrationAccelerated approval program. https://www.fda.gov/drugs/nda-and-bla-approvals/accelerated-approval-programDate accessed: October 24, 2023 Google Scholar By contrast to regular approval, accelerated approval requires post-market confirmatory clinical trials to establish the clinical benefit of the drug, then a decision is made as to whether the approval should be converted to a traditional approval or whether the drug should be withdrawn from the market. 2 US Food and Drug AdministrationExpedited programs for serious conditions––drugs and biologics. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologicsDate accessed: October 24, 2023 Google Scholar In the past 3 years, this expedited pathway has attracted extensive criticism, including failure to complete confirmatory clinical trials in a timely manner, 3 Fashoyin-Aje LA Mehta GU Beaver JA Pazdur R The on- and off-ramps of oncology accelerated approval. N Engl J Med. 2022; 387: 1439-1442 Crossref PubMed Scopus (33) Google Scholar , 4 Gyawali B Kesselheim AS Ross JS The accelerated approval program for cancer drugs—finding the right balance. N Engl J Med. 2023; 389: 968-971 Crossref PubMed Scopus (9) Google Scholar use of surrogate endpoints (eg, progression-free survival) in confirmatory clinical trials, 5 Parikh RB Hubbard RA Wang E et al. Exposure to US cancer drugs with lack of confirmed benefit after US Food and Drug Administration accelerated approval. JAMA Oncol. 2023; 9: 567-569 Crossref PubMed Scopus (6) Google Scholar and failure to withdraw drugs quickly from the markets when they have been shown to not have clinical benefit. 6 Akhade A Sirohi B Gyawali B Global consequences of the US FDA's accelerated approval of cancer drugs. Lancet Oncol. 2022; 23: 201-203 Summary Full Text Full Text PDF PubMed Scopus (10) Google Scholar Concerns have also been raised that cancer drugs given accelerated approval by the FDA could lead to difficult decisions in other countries, especially in some low-income and middle-income countries that do not have their own regulatory agencies or that have weak regulatory capacity. 6 Akhade A Sirohi B Gyawali B Global consequences of the US FDA's accelerated approval of cancer drugs. Lancet Oncol. 2022; 23: 201-203 Summary Full Text Full Text PDF PubMed Scopus (10) Google Scholar , 7 Cramer A Sørup FKH Christensen HR Petersen TS Karstoft K Withdrawn accelerated approvals for cancer indications in the USA: what is the marketing authorisation status in the EU?. Lancet Oncol. 2023; 24: e385-e394 Summary Full Text Full Text PDF PubMed Scopus (3) Google Scholar