Pcn188 the Importance of Overall Survival as a Clinical Endpoint in Fda vs Ema Regulatory Approval

Abstract

The Comparative Non-Small Cell Lung Cancer (NSCLC) Health Outcomes Model uses a Markov cohort model approach to estimate the population health outcomes of US patients with locally advanced or metastatic NSCLC under different access policies. The model comprises a base case which simulates health outcomes in the US, and five additional scenarios in which US access conditions are replaced by policies from five comparator countries (Australia, Canada, France, South Korea and the United Kingdom). Under each scenario the model controls for differences in regulatory approval timelines to focus on the timeliness and comprehensiveness of reimbursement coverage. Results: Model outcomes indicate that US patients diagnosed with locally advanced and metastatic NSCLC between 2006 and 2017 have gained 201,700 life years in total due to access to innovative medicines. US patients would lose half of this survival benefit if they instead experienced the access conditions of the other comparator countries. Significant variation in outcomes existed between the access conditions of different countries, with the Australian system leading to the largest decrease in life years (-74%). Conclusions: The difference in health outcomes among the scenarios demonstrates how access policies can impact the population health of NSCLC patients. Our study supports the importance of continued timely access to cancer medicines in the US and underscores the potential health impacts from adopting more restrictive approaches used by foreign countries.