ASSESSMENT OF HEALTH TECHNOLOGY APPRAISALS TO IDENTIFY KEY DRIVERS FOR REIMBURSEMENT OF ONCOLOGY DRUGS WITH ONLY PHASE 2 CLINICAL DATA
Author(s)
Duran A1, Morlock R2, Lie X3, Dekkers R3, Gani R4, van Engen A3, Holmstrom S5
1Astellas Pharma Europe Ltd., Chertsey, UK, 2Astellas Pharma Global Development, Inc., Northbrook, IL, USA, 3QuintilesIMS Consulting Services, Hoofddorp, The Netherlands, 4QuintilesIMS Consulting Services, Berkshire, UK, 5Astellas Pharma Europe, B.V., Leiden, The Netherlands
OBJECTIVES: Drugs can obtain marketing authorisation with only Phase 2 data if the data is considered promising, a Phase 3 trial is not considered feasible or there is a high unmet need. Here we identify and review challenges faced by manufacturers of oncology drugs approved with only Phase 2 data in Health Technology Appraisals (HTAs). METHODS: Oncology drugs approved on basis of Phase 2 data since 2010 were identified on the European Medicines Agency (EMA) website and were ranked based on inclusion of overall survival data, a control arm, were deemed a targeted therapy, had orphan disease status or conditional marketing authorisation. HTAs from Germany, France, UK, Australia, Canada, Sweden, Italy and Spain for the top-ranked products were then identified using QuintilesIMS proprietary HTA Accelerator and reviewed to identify payer’s rationale for reimbursement decisions. RESULTS: Twenty-five oncology drugs were licenced with Phase 2 data alone since 2010. The nine top-ranked products had 51 relevant HTA reports. 47% of the reports recommended unrestricted access, 16% had restrictions, 16% were rejections and 22% provided no reimbursement recommendation. The most frequent positive payer comments related to clinical benefit, good safety/tolerability and innovation. The most frequent negative payer comments related to safety risk, lack of comparator, lack of subgroup data, and limited comparative benefit. Lack of Phase 3 data was not directly cited, but uncertainty and insufficient powering were identified as a payer concern. Many trials were single arm and payers struggled to use these results to assess clinical benefit. In three cases, resubmissions with additional Phase 3 data led to improved reimbursement outcomes. CONCLUSIONS: The review found that using Phase 2 alone is not an absolute barrier to reimbursement, but the uncertainty stemming from a less comprehensive evidence base may influence payers’ decisions. Payer comments related mostly to good efficacy and safety data, and robust comparative effectiveness.
Conference/Value in Health Info
2017-11, ISPOR Europe 2017, Glasgow, Scotland
Value in Health, Vol. 20, No. 9 (October 2017)
Code
PCN331
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Oncology