Clinical Development and HTA Approval of MULTI-Indication Oncology Products: Evidence from Germany, France, England, Scotland, the US, Canada, and Australia.
Author(s)
Mills M*;Michaeli D;Miracolo A, Kanavos P
London School of Economics and Political Science, London, UK
OBJECTIVES It is unclear if current pricing and reimbursement policies adequately capture the incremental value of individual indications for multi-indication products. This study aims to map the launch sequence of multi-indication products across seven countries and compares indications in terms of quality of clinical evidence, regulatory approval timelines and HTA approval timelines.
METHODS Twenty-five multi-indication products were identified with FDA approvals of multiple indications between 2009 and 2019. The FDA approval date was taken as a measure of ‘global launch’. Regulatory agency websites, clinicaltrials.gov, and health technology assessment websites in Germany, France, England, Scotland, Canada, the USA and Australia were screened to extract data on marketing authorisations, clinical trial characteristics, and HTA recommendations for all authorised indications. Descriptive statistics and survival analysis were conducted to identify differences between first and subsequent indications in terms of clinical development time, HTA approval time, and quality of clinical evidence.
RESULTS A total of 113 indications were identified across the 25 multi-indication products. The first globally launched indication was more likely to have regulatory approval and receive HTA approval across all settings than subsequent indications (p<0.001). First indications were more likely to have orphan designations (p<0.001), undergo priority review (p<0.001), have phase II, single arm pivotal trial designs (p<0.001), and use surrogate outcomes as a primary endpoint (p<0.001). No significant differences were detected in clinical development time of first vs subsequent indications. Subsequent indications have significantly shorter HTA approval timelines in England, Germany, Canada, and Australia.
CONCLUSIONS Given similar clinical development and HTA approval timelines, it is unlikely that manufacturers are systematically delaying the launch of indications to prioritise first launch of a “high value” indication. However manufacturers and payors do not fully align on the value of indication extensions, leading to a greater probability of subsequent indications not launching within individual settings.
METHODS Twenty-five multi-indication products were identified with FDA approvals of multiple indications between 2009 and 2019. The FDA approval date was taken as a measure of ‘global launch’. Regulatory agency websites, clinicaltrials.gov, and health technology assessment websites in Germany, France, England, Scotland, Canada, the USA and Australia were screened to extract data on marketing authorisations, clinical trial characteristics, and HTA recommendations for all authorised indications. Descriptive statistics and survival analysis were conducted to identify differences between first and subsequent indications in terms of clinical development time, HTA approval time, and quality of clinical evidence.
RESULTS A total of 113 indications were identified across the 25 multi-indication products. The first globally launched indication was more likely to have regulatory approval and receive HTA approval across all settings than subsequent indications (p<0.001). First indications were more likely to have orphan designations (p<0.001), undergo priority review (p<0.001), have phase II, single arm pivotal trial designs (p<0.001), and use surrogate outcomes as a primary endpoint (p<0.001). No significant differences were detected in clinical development time of first vs subsequent indications. Subsequent indications have significantly shorter HTA approval timelines in England, Germany, Canada, and Australia.
CONCLUSIONS Given similar clinical development and HTA approval timelines, it is unlikely that manufacturers are systematically delaying the launch of indications to prioritise first launch of a “high value” indication. However manufacturers and payors do not fully align on the value of indication extensions, leading to a greater probability of subsequent indications not launching within individual settings.
Conference/Value in Health Info
2020-11, ISPOR Europe 2020, Milan, Italy
Value in Health, Volume 23, Issue S2 (December 2020)
Code
HTA4
Topic
Clinical Outcomes, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Comparative Effectiveness or Efficacy, Pricing Policy & Schemes, Reimbursement & Access Policy, Systems & Structure
Disease
Drugs, Oncology