Mp57-09 Time to Approval of Novel Cancer Medicines Granted Accelerated Approval and Implications for Reform of the Accelerated Approval Pathway

You have accessJournal of UrologyHealth Services Research: Value of Care: Cost and Outcomes II (MP57)1 May 2024MP57-09 TIME TO APPROVAL OF NOVEL CANCER MEDICINES GRANTED ACCELERATED APPROVAL AND IMPLICATIONS FOR REFORM OF THE ACCELERATED APPROVAL PATHWAY Thomas Hwang and Ariadna Tibau Thomas HwangThomas Hwang and Ariadna TibauAriadna Tibau View All Author Informationhttps://doi.org/10.1097/01.JU.0001009420.83948.eb.09AboutPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookLinked InTwitterEmail Abstract INTRODUCTION AND OBJECTIVE: The Accelerated Approval (AA) pathway was established to speed development and approval of drugs for life-threatening conditions with limited treatment options. In December 2022, the US Congress enacted new policies to ensure that confirmatory studies for AA medicines are completed in a timely fashion. Here, we evaluate factors associated with conversion of AA to regular approval by the US FDA, including magnitude of clinical benefit as measured by European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). METHODS: We identified all new medicines approved by the US Food and Drug Administration (FDA) for treatment of adult patients with genitourinary (GU) and non-GU solid tumors through the AA pathway from 1992 to 2020, with follow up through January 2022. FDA drug labels and clinical trial reports were reviewed to apply ESMO-MCBS v1.1 grades to both initial and confirmatory studies. High clinical benefit was defined as a grade of A or B for trials of curative intent and 4 or 5 for those of palliative intent. Withdrawn AA indications, as determined by FDA, were considered as low benefit. Two-tailed statistical significance was defined as p<0.05. RESULTS: From 1992 to 2020, the FDA granted AA to 46 cancer drugs for 74 indications. The median time between AA and regular approval of oncology drugs was 3.7 years (range: 0.8-12.6 years). Of these 74 indications, 42 (57%) had clinical benefit confirmed in post-approval clinical trials and were converted to regular approval. Indications with high clinical benefit according to ESMO-MCBS were associated with shorter times to conversion to regular approval (median 2.5 vs 4.8 years; p=0.007). Similarly, indications with overall survival benefit were associated with shorter times to regular approval (median 2.6 vs 3.7 years; p=0.04). CONCLUSIONS: Cancer drugs with greater clinical benefit were associated with faster conversion to full FDA approval. New policy reforms to the AA pathway should prioritize further expediting market access of therapies with high clinical benefit as well as expedited completion of confirmatory studies. Source of Funding: Swiss Cancer Research Foundation © 2024 by American Urological Association Education and Research, Inc.FiguresReferencesRelatedDetails Volume 211Issue 5SMay 2024Page: e941 Advertisement Copyright & Permissions© 2024 by American Urological Association Education and Research, Inc.Metrics Author Information Thomas Hwang More articles by this author Ariadna Tibau More articles by this author Expand All Advertisement PDF downloadLoading ...