Assessment of the clinical and cost-effectiveness evidence in the reimbursement decisions of new cancer drugs

• Background: This study aimed to describe the clinical and cost-effectiveness evidence supporting reimbursementdecisions of new cancer drugs and analyze the influence of trial characteristics and the cost per quality-adjusted lifeyears (QALYs) on the likelihood of reimbursement in Sweden.Patients and methods: Data were extracted from all appraisal dossiers for new cancer drugs seeking reimbursement inSweden and claiming added therapeutical value between the years 2010 and 2020. The data were analyzed usingdescriptive statistics, and logistic regression models were also used with the cost per QALY, study design,comparator, and evidence on final outcomes in the clinical trials as predictors of reimbursement.Results: All 60 included appraisals were based on trial evidence that assessed at least one final outcome (overallsurvival [OS] or quality of life [QoL]), although rarely as a primary outcome. Of the appraisals with a final decision(n ¼ 58), 79% were approved for reimbursement. Among the reimbursed drugs, only half had trial evidencedemonstrating improved OS or QoL. Only one drug had trial evidence supporting improvements in both OS andQoL. The average cost per QALY for reimbursed cancer drugs was estimated to be 748 560 SEK (V73 583). A highercost per QALY was found to decrease the likelihood of reimbursement by 9.4% for every 100 000 SEK (V9830)higher cost per QALY (P ¼ 0.03). For cost-effectiveness models without direct evidence of improvements in finaloutcomes, a larger QALY gain was observed compared with those with evidence mainly relying on intermediate andsurrogate outcomes.Conclusions: There are substantial uncertainties in the clinical and cost-effectiveness evidence underlyingreimbursement decisions of new cancer drugs. Decision makers should be cautious of the limited evidence onpatient-centered outcomes and the implications of allocating resources to expensive treatments with uncertainvalue for money.

Ämnesord

SAMHÄLLSVETENSKAP  -- Ekonomi och näringsliv (hsv//swe)

SOCIAL SCIENCES  -- Economics and Business (hsv//eng)

MEDICIN OCH HÄLSOVETENSKAP  -- Hälsovetenskap -- Hälso- och sjukvårdsorganisation, hälsopolitik och hälsoekonomi (hsv//swe)

MEDICAL AND HEALTH SCIENCES  -- Health Sciences -- Health Care Service and Management, Health Policy and Services and Health Economy (hsv//eng)

Nyckelord

reimbursement

health technology assessment

decision making

cancer drugs

cancer drugs

decision making

health technology assessment

reimbursement

Publikations- och innehållstyp

ref (ämneskategori)

art (ämneskategori)