IMPORTANCE Numerous cancer drugs have received accelerated approval from the US Food and Drug Administration (FDA) based on clinical trial outcomes that are otherwise not acceptable for traditional FDA approval; the accelerated approval process allows outcomes based on surrogate measures that are only reasonably likely to estimate clinical benefits. In England, the National Institute for Health and Care Excellence (NICE) evaluates the clinical benefits and cost-effectiveness of drugs after they have received regulatory approval and issues recommendations regarding their coverage in the National Health Service (NHS). However, the level of concordance between European and FDA decision-making in the context of drugs qualifying for FDA accelerated approval is unknown. OBJECTIVE To compare FDA accelerated approval decisions for cancer drugs with NICE coverage decisions. DESIGN, SETTING, AND PARTICIPANTS This retrospective cohort study compared cancer drug indications that received FDA accelerated approval from December 11, 1992, to May 31, 2017, with the same set of drug indication pairs in England until August 31, 2019. Data from European Public Assessment Reports developed by the European Medicines Agency (EMA) and public appraisal documents from NICE were used to determine NHS coverage recommendations. National Institute for Health and Care Excellence (NICE) public appraisal documents were analyzed for drug indications, characteristics of clinical evidence, cost-effectiveness, and coverage decisions. Data were analyzed from September 1 to December 31, 2019.
Background: England's National Institute for Health and Care Excellence (NICE) and the US' Institute for Clinical and Economic Review (ICER) both conduct cost-effectiveness evaluations for new cancer drugs to help payers make drug coverage decisions. However, NICE and ICER assessments have been noted to reach different conclusions. We aim to better understand the degree to which their recommendations diverge and what drives these apparent differences. Methods: We compared the methods and results of publicly available cost-effectiveness evaluations performed by ICER and NICE of similarly assessed cancer drugs. Assessments were compared based on incremental cost-effectiveness ratio, comparator treatment, price, recommendation, and the design of the economic evaluation. Findings: Among 11 commonly assessed cancer drugs, ICER and NICE were in concordance for 7 evaluations and in discordance on the cost-effectiveness and coverage decisions for 4 drugs. Most new cancer drugs were not cost-effective in either the US (7/11) or England (7/11). Furthermore, NICE's capacity to negotiate price discounts and access schemes result in much lower cost per QALY valuations and more favourable recommendations than those of ICER for similarly assessed cancer drugs. Interpretations: NICE and ICER employ similar health technology assessment (HTA) methodologies and were aligned with most recommendations, finding that many new and expensive cancer drugs are cost ineffective. Growing use of ICER assessments will continue to send stronger price signals to manufacturers that cancer drugs with low value for money will be viewed less favourably by private insurers. NICE provides an important reminder of how much lower other countries pay for drugs when comparative effectiveness and valuebased pricing are integrated into public drug coverage decisions.
At present, pay for prescription models are insufficient at containing costs and improving access to medicines. Subscription financing through tenders, licensing fees and unrestricted or fixed volumes can benefit stakeholders across the supply chain. Pharmaceutical manufacturers can reduce the need for marketing expenses and gain certainty in revenue. This will decrease costs, improve predictability in budget expenditure for payers and remove price as a barrier of access from patients. Inherently, low- and middle-income countries lack the purchasing power to leverage price discounts through typical price arrangements. These markets can realise substantial savings for branded and generic medicines through subscription financing. Procuring of on-patent and off-patent drugs requires separate analysis for competition effects, the length of contract and encouraging innovation in the medicine pipeline. Prices of competitive on-patent medicines and orphan drugs can be reduced through increased competition and volume. Furthermore, pooling expertise and resources through joint procurement has the potential for greater savings. Incentivising research and development within the pharmaceutical industry is essential for sustaining a competitive market, preventing monopolies and improving access to expensive treatments. However, technical capacity, forecasting demand and the quality of generic medicines present limitations which necessitate government support and international partnerships. Ultimately, improving access requires progressive financing mechanisms with patients and cost containment in mind.
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