Can Early U.S. Adoption of Cancer Drugs Inform HTA Decision-Making?


Jonsson P1, Mpofu P2, Kent S3, Copeland A2, Groves B4, Bargo D2, Ramsey S5, Baxi S2, Adamson B2
1National Institute for Health and Care Excellence, Manchester, UK, 2Flatiron Health, New York, NY, USA, 3National Institute for Health and Care Excellence, Manchester, LAN, UK, 4National Institute for Health and Care Excellence (NICE), London, LON, UK, 5Hutchinson Institute for Cancer Outcomes Research, Fred Hutchinson Cancer Research Center, Seattle, WA, USA

OBJECTIVES: International differences in time-to-approval of new drugs could enable use of real-world data from the US to inform technology appraisals (TAs). To understand availability of data, we evaluated prescribing after FDA approval and preceding NICE appraisal within a network of US cancer clinics.

METHODS: We evaluated prescribing patterns for drugs with NICE TAs for oncology published between 1/1/2014 and 12/31/2019 using the electronic health record (EHR)-derived de-identified Flatiron Health database. We measured time from FDA approval to manufacturer submission to NICE and publication of final guidance. For instances where FDA preceded NICE appraisal, we identified the number of patients starting the new drug after FDA approval and corresponding follow-up time available at each NICE milestone.

RESULTS: We included 60 NICE TAs for drugs across 11 cancers. Median time from FDA to NICE submission and final guidance publication was 5.6 and 18.5 months, respectively. At the time of manufacturer submission to NICE, an average of 147 real-world patients had received the drug of interest on-label after FDA approval with a median of 4.5 months of follow-up time, increasing to 269 patients and 6.4 months of follow-up time at the time of NICE final guidance publication. Patient utilization numbers were higher in those topics recommended for use in the Cancer Drug Fund compared to recommended for routine commissioning.

CONCLUSIONS: The time from FDA approval to NICE guidance may provide an opportunity to inform reimbursement decisions with real-world US patients. Opportunities to use this data will vary by cancer type, the nature of uncertainties identified — most frequently cited for cancer products are longer term measures such as overall survival, progression-free survival, as well as quality of life — and will depend on whether the data is reflective of UK patient characteristics, treatment settings, and clinical pathways.

Conference/Value in Health Info

2021-05, ISPOR 2021, Montreal, Canada

Value in Health, Volume 24, Issue 5, S1 (May 2021)




Economic Evaluation, Health Technology Assessment

Topic Subcategory

Cost-comparison, Effectiveness, Utility, Benefit Analysis, Decision & Deliberative Processes



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