Clinical Benefits of the Accelerated Approval Program in Oncology

OBJECTIVES: Estimate the clinical benefits of early access to breast and non-small cell lung cancer (NSCLC) treatments approved under the Accelerated Approval Program (AAP), which FDA instituted in 1992 to allow for conditional approval of drugs shown to improve surrogate measures within serious conditions with unmet medical needs.

METHODS: All breast and NSCLC drugs granted accelerated approval and later converting to traditional approval were identified from the FDA. IQVIA data was used to estimate the number of patients treated with each drug for the period between accelerated approval and traditional approval. Incremental progression-free and overall survival benefits relative to the comparator were obtained from each confirmatory trial as reported in the prescribing information or resulting publication. Survival benefits for the total population utilizing each drug were calculated to estimate the societal benefits associated with early access, stratified by cancer type and payer type.

RESULTS: 233,305 breast cancer patients and 97,749 NSCLC patients gained earlier access to ten breast cancer and ten NSCLS therapies because of the AAP. For breast cancer patients, this resulted in over 85,000 years of progression-free survival, and nearly 160,000 years of life gained. Lung cancer patients had 27,987 additional years of progression-free survival, with overall survival estimates unavailable as trials are ongoing. Within breast cancer, 55% of the gains were in individuals with commercial insurance or Medicaid, whereas in lung cancer over 70% of the gains were in the Medicare population.

CONCLUSIONS: The Accelerated Approval Program has succeeded in making life-extending therapies available to patients sooner. These findings, likely an underestimate as they only consider two diseases and ignore the AAP’s potential to increase the odds a product will be developed and the number of indications pursued per product, highlight that the AAP is a valuable pathway for patients and should be protected.