Objectives: The unmet need in non-small cell lung cancer (NSCLC) is evidenced by four targeted therapies, crizotinib, ceritinib, osimertinib and alectinib, which were all brought to patients early through conditional marketing authorization (CMA). We aimed to characterize evidence generation for these early access products and its impact on access. Methods: Volume, type and timelines of evidence generation was identified and compared between the four products. The scope of evidence includes early access programs (EAPs), expanded access programs, pivotal trial extension studies, and real world evidence. Time to positive health technology assessment (HTA) outcome was assessed in 4 major European markets. Results: All products received CMA based on ongoing phase II or I/II trials and required phase III data for full MA, which were ongoing when CMA was granted. Official EAPs were initiated in Europe for all products. In addition, expanded access program(s) were conducted for other geographies. Open-label extension studies collecting long-term data was identified for all products except osimertinib where an extension study was not initiated yet as the phase III is still ongoing. A range of observational studies and registries have been initiated for all 4 products to collect efficacy and safety data, as well as patient reported outcomes (PRO), pharmacoeconomics and data on real world clinical practice such as biomarker testing and treatment patterns. Time from CMA submission to first positive (with restrictions) HTA outcome was faster for all products versus standard MA benchmark (26.8 months), with osimertinib being quickest (15.8 months) and alectinib slowest (25.7 months). Conclusions: These 4 case studies showed that in diseases with high unmet need, companies are using a full range of evidence generation opportunities to complement the main clinical trials. This additional commitment to a broader and earlier understanding of the product may have a positive impact on the market access outcome.
van Hooijdonk, I. E., Lai, L., & van Engen, A. (2018). PCN30 - EVIDENCE GENERATION IN A EARLY ACCESS STRATEGY FOR TARGETED ONCOLOGY THERAPIES: A COMPARISON IN NON-SMALL CELL LUNG CANCER. Value in Health, 21, S19–S20. https://doi.org/10.1016/j.jval.2018.09.112