Presentation (CTI)

OBJECTIVES: Although most pivotal oncology trials generate PRO data, there is limited evidence on regulatory and reimbursement outcomes and ultimately the return on investment (ROI) of collecting PRO data across Europe. Therefore, as a first step to estimating ROI, we attempted to quantify the impact PRO data might have on regulators’ and payers’ decision making in 5 European major markets.
METHODS: New indication approvals by EMA and FDA between 2011 and 2024 in oncology were identified using IQVIA’s Market Access Insights platform. The pivotal study design, endpoints, PRO regulatory labelling claim and HTA/reimbursement outcomes in selected countries (England, Germany, France, Italy, and Spain) were extracted. HTA/reimbursement outcomes were compared between products sets with different PRO results.
RESULTS: 93 novel oncology products that launched with RCTs were identified. Out of these 85 products (91%) had one or more PRO measure in the trial. 25 of 93 products (27%) achieved a PRO claim or mention in the FDA (3 of 25 products) and/or EMA label (all 25 products), 23 products (24%) received a PRO-based added benefit rating by Germany’s G-BA, and 10 products (11%) achieved both a regulatory label/mention and a PRO-based G-BA added benefit rating. The 38 products with either a label claim and/or a PRO based G-BA benefit rating were more likely to be reimbursed across England, Germany, France, Italy and Spain vs the 55 products that achieved neither (79% vs 42%). Additionally, the products with either a label claim and/or a PRO based G-BA rating were more likely to achieve considerable added benefit rating in Germany (76% vs 33%).
CONCLUSIONS: These results suggest that well-designed investment in a PRO strategy can increase the probability of demonstrating treatment benefit with PROs, positively impacting reimbursement and HTA outcomes.