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 reimbursement decisions of new cancer drugs and analyze the influence of trial characteristics and the cost per quality-adjusted life years (QALYs) on the likelihood of reimbursement in Sweden. PATIENTS AND METHODS: Data were extracted from all appraisal dossiers for new cancer drugs seeking reimbursement in Sweden and claiming added therapeutical value between the years 2010 and 2020. The data were analyzed using descriptive 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 (overall survival [OS] or quality of life [QoL]), although rarely as a primary outcome. Of the appraisals with a final decision (n = 58), 79% were approved for reimbursement. Among the reimbursed drugs, only half had trial evidence demonstrating improved OS or QoL. Only one drug had trial evidence supporting improvements in both OS and QoL. The average cost per QALY for reimbursed cancer drugs was estimated to be 748 560 SEK (€73 583). A higher cost per QALY was found to decrease the likelihood of reimbursement by 9.4% for every 100 000 SEK (€9830) higher cost per QALY (P = 0.03). For cost-effectiveness models without direct evidence of improvements in final outcomes, a larger QALY gain was observed compared with those with evidence mainly relying on intermediate and surrogate outcomes. CONCLUSIONS: There are substantial uncertainties in the clinical and cost-effectiveness evidence underlying reimbursement decisions of new cancer drugs. Decision makers should be cautious of the limited evidence on patient-centered outcomes and the implications of allocating resources to expensive treatments with uncertain value for money.

Colorectal cancer screening with fecal immunochemical testing or primary colonoscopy: An analysis of health equity based on a randomised trial.

Background: We have addressed health equity attained by fecal immunochemical testing (FIT) and primary colonoscopy (PCOL), respectively, in the randomised controlled screening trial SCREESCO conducted in Sweden. Methods: We analysed data on the individuals recruited between March 2014, and March 2020, within the study registered with ClinicalTrials.gov, NCT02078804. Swedish population registry data on educational level, household income, country of birth, and marital status were linked to each 60-year-old man and woman who had been randomised to two rounds of FIT 2 years apart (n = 60,123) or once-only PCOL (n = 30,390). Furthermore, we geo-coded each study individual to his/her residential area and assessed neighbourhood-level data on deprivation, proportion of non-Western immigrants, population density, and average distance to healthcare center for colonoscopy. We estimated adjusted associations of each covariate with the colonoscopy attendance proportion out of all invited to respective arms; ie, the preferred outcome for addressing health equity. In the FIT arm, the test uptake and the colonoscopy uptake among the test positives were considered as the secondary outcomes. Findings: We found a marked socioeconomic gradient in the colonoscopy attendance proportion in the PCOL arm (adjusted odds ratio [95% credibility interval] between the groups categorised in the highest vs. lowest national quartile for household income: 2·20 [2·01-2·42]) in parallel with the gradient in the test uptake of the FIT × 2 screening (2·08 [1·96-2·20]). The corresponding gradient in the colonoscopy attendance proportion out of all invited to FIT was less pronounced (1·29 [1·16-1·42]), due to higher proportions of FIT positives in socioeconomically disadvantaged groups. Interpretation: The unintended risk of exacerbating inequalities in health by organised colorectal cancer screening may be higher with a PCOL strategy than a FIT strategy, despite parallel socioeconomic gradients in uptake. Funding: This work was supported by the Swedish Cancer Society under Grant 20 0719. CB and US provided economic support from the Swedish Research Council for Health, Working life, and Welfare under Grant 2020-00962.