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Association between the use of surrogate measures in pivotal trials and health technology assessment decisions: a retrospective analysis of NICE and CADTH reviews of cancer drugs

Pinto, Ashlyn, Naci, Huseyin, Neez, Emilie and Mossialos, Elias (2020) Association between the use of surrogate measures in pivotal trials and health technology assessment decisions: a retrospective analysis of NICE and CADTH reviews of cancer drugs. Value in Health, 23 (3). pp. 319-327. ISSN 1098-3015

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Identification Number: 10.1016/j.jval.2019.10.010

Abstract

Objective: To assess whether using surrogate versus patient-relevant endpoints in pivotal trials of cancer drugs was associated with health technology assessment recommendations in England (National Institute for Health and Care Excellence [NICE]) and Canada (Canadian Agency for Drugs and Technologies in Health [CADTH]). Methods: Cancer drug approvals from 2012 to 2016 were categorized by demonstrating benefit on overall survival (OS), progression-free survival, disease response, or having no comparator. Approvals were analyzed by benefit category and health technology assessment recommendation. The association between benefit (surrogate vs OS) and recommending a drug was examined using descriptive statistics and linear probability models controlling for unmet need, orphan designation, and cost-effectiveness. Results: Of 42 cancer indications that NICE recommended, 15 (36%) demonstrated OS benefit. Cancer indications with OS benefit were less likely to receive a recommendation from NICE than those without (P = .04). In linear probability models, availability of OS benefit was no longer associated with a recommendation from NICE (P = .32). Cost-effective cancer drugs had a 55.6% (95% CI: 38.9%-72.3%) higher probability of receiving a recommendation from NICE than those that were not. In Canada, 15 of 37 (41%) cancer indications that were recommended showed OS benefit. There was no detectable association between surrogate measures and CADTH recommendations based on descriptive statistics (P = .62) or in linear probability models (P = .73). Conclusion: When cost-effectiveness was considered, pivotal trial endpoints were not associated with NICE recommendations. Pivotal trial endpoints, unmet need, orphan status, and cost-effectiveness did not explain CADTH recommendations.

Item Type: Article
Official URL: https://www.sciencedirect.com/journal/value-in-hea...
Additional Information: © 2019 ISPOR – The Professional Society for Health Economics and Outcomes Research.
Divisions: Health Policy
Subjects: R Medicine > RA Public aspects of medicine > RA0421 Public health. Hygiene. Preventive Medicine
R Medicine > RC Internal medicine > RC0254 Neoplasms. Tumors. Oncology (including Cancer)
Date Deposited: 13 Feb 2020 08:15
Last Modified: 28 Jun 2020 23:29
URI: http://eprints.lse.ac.uk/id/eprint/103355

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