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 ORCID: 0000-0002-7192-5751, Neez, Emilie and Mossialos, Elias ORCID: 0000-0001-8664-9297 (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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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:	25 Oct 2024 17:27
URI:	http://eprints.lse.ac.uk/id/eprint/103355

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