To review clinical and cost-effectiveness evidence underlying reimbursement decisions relating to drugs whose authorization mainly is based on evidence from prospective case series.
A systematic review of all new drugs evaluated in 2011-2016 within a health care profession-driven resource prioritization process, with a market approval based on prospective case series, and a reimbursement decision by the Swedish Dental and Pharmaceutical Benefits Agency (TLV). Public assessment reports from the European Medicines Agency, published pivotal studies, and TLV, Scottish Medicines Consortium and National Institute of Health and Care Excellence decisions and guidance documents were reviewed.
Six drug cases were assessed (brentuximab vedotin, bosutinib, ponatinib, idelalisib, vismodegib, ceritinib). The validity of the pivotal studies was hampered by the use of surrogate primary outcomes and the absence of recruitment information. To quantify drug treatment effect sizes, the reimbursement agencies primarily used data from another source in indirect comparisons. TLV granted reimbursement in five cases, compared with five in five cases for Scottish Medicines Consortium and four in five cases for National Institute of Health and Care Excellence. Decision modifiers, contributing to granted reimbursement despite hugely uncertain cost-effectiveness ratios, were, for example, small population size, occasionally linked to budget impact, severity of disease, end of life and improved life expectancy.
For drugs whose authorization is based on prospective case series, most applications for reimbursement within public health care are granted. The underlying evidence has limitations over and above the design per se, and decision modifiers are frequently referred to in the value-based pricing decision making.
Suicide is the leading cause of death among young men aged 15?29 in Greenland, but few epidemiological studies have described this problem. We aimed to summarise descriptive epidemiological studies of suicide in young men in Greenland compared with other demographic groups in Denmark and Greenland to inform future suicide prevention strategy. We searched PubMed, PsycINFO, and Embase using an agreed search strategy to identify English-language papers describing suicide epidemiology in Greenlandic men aged 15?29. We followed PRISMA guidelines in screening and appraising eligible publications. Eight articles fulfilled inclusion criteria of 64 meeting search criteria. Findings covering 1970?2011 supported a dramatic rise in suicide rates in Greenlandic men aged 15?24 from 1976, who remained the highest-ranking demographic group over 1976?2011 compared with men and women of all age groups in Denmark and Greenland. Highest rates recorded were almost 600 per 100,000 per year in men aged approximately 20?23 over 1977?1986. No studies described suicide epidemiology after 2011, and no studies described risk factors for suicide in young men. Given the very high suicide rates recorded for young men over 1976?2011, such studies will be essential for informing the development and evaluation of appropriate preventive interventions.