Caution needed in introduction of provisional approvals for medicines

Abstract

The Australian government recently released its response to the Review of Medicines and Medical Devices Regulation, accepting most recommendations. One recommendation involves the introduction of provisional approvals for perceived lifesaving and innovative new treatments, allowing these to be approved on the basis of a more limited data dossier on the condition that further safety and efficacy data (including real world evidence) is collected to determine whether full approval should be granted. However, experience with similar schemes overseas raises significant questions about the safety and efficacy of products made available via these pathways; these risks are compounded by factors such as the challenges associated with the collection and use of “real world” data and the difficulties of withdrawing products from the market once patients and clinicians become familiar with them. Although there are a number of good reasons to provide patients with earlier access to medicines on the basis of provisional evidence (including providing treatment options to patients with serious illnesses and hope to those in desperate situations) we must exercise caution in the introduction of accelerated approval pathways in order to protect both current and future patients from potentially harmful and futile treatments, and ensure that healthcare systems use their resources wisely.