Global consequences of the US FDA's accelerated approval of cancer drugs.

Abstract

The accelerated approval system is a fast-track drug approval pathway created by the US Food and Drug Administration (FDA) in 1992 in response to the HIV-AIDS crisis to allow expedited access to life-saving drugs and fulfil an unmet medical need. Nowadays, this pathway is most commonly used for cancer drug approval. 1 Sachs RE Gavulic KA Donohue JM Dusetzina SB Recent trends in Medicaid spending and use of drugs with US Food and Drug Administration accelerated approval. JAMA Health Forum. 2021; 2e213177 Google Scholar In theory, the accelerated approval pathway strikes a balance between early access to therapies and the need for robust evidence generation. This pathway allows drugs to be approved early on the basis of improvement in surrogate outcomes that are reasonably likely to translate to clinical benefit. This approval is conditional, with a mandate that drugs are tested in a post-approval confirmatory trial to substantiate the presumed clinical benefit, in the absence of which approval will be withdrawn. Several challenges and limitations of this pathway for the US regulatory and clinical environment have been previously described. 2 Gyawali B Ross JS Kesselheim AS Fulfilling the mandate of the US Food and Drug Administration's accelerated approval pathway: the need for reforms. JAMA Intern Med. 2021; 181: 1275-1276 Google Scholar Here, we discuss the often-overlooked consequences of the accelerated approval pathway for global oncology. Consequences of US FDA approval decisions in high-income countriesAkhade and colleagues1 highlight the global consequences of approving cancer medicines on the basis of uncertain evidence through the US Food and Drug Administration (FDA)'s accelerated approval pathway. Traditionally, the concern for US regulatory standards has remained inside the USA. However, Akhade and colleagues show that the consequences can be far reaching. Their Comment has substantial implications, especially considering the nomination of a new FDA commissioner and the opportunity that this nomination brings to enforce a suitable efficacy standard. Full-Text PDF