AIM: To determine the survival outcomes of New Zealand patients with unresectable or metastatic melanoma treated with pembrolizumab or nivolumab.
METHODS: This is a national retrospective cohort study. Patients with advanced unresectable or metastatic melanoma who received publicly funded immune-checkpoint inhibitors (ICIs) from 2017 to 2019 were included. Individual patient data were extracted from national administrative databases. The primary endpoint was OS, and secondary endpoints included OS by age, duration of treatment, posttreatment survival, and 30-day mortality from last pharmaceutical claim.
RESULTS: Five hundred ninety-seven patients were included, with a median follow-up of 25 months. One-year OS was 72%, the 2-year OS estimate was 60%, and median OS not reached. Survival did not differ by dichotomized age (≥70 vs. <70 year old), hazard ratio (HR) .94 (95% confidence interval (CI): .72-1.22; p = .62). Median duration of treatment was 9.0 months (95% CI: 7.9-10.1). Median post-treatment survival for the subgroup who had ceased treatment was 12.0 months (95% CI: 9.0-14.0). For the sample as a whole, the estimated 30-day mortality from last pharmaceutical claim was 15.7%.
CONCLUSION: OS in our New Zealand real-world population is comparable to pivotal clinical trials and real-world data (RWD) from other countries. These findings support the achievement of health gains from use of ICI in advanced unresectable and metastatic melanoma.
OBJECTIVE: To investigate the evidence available: 1) on government initiatives to mandate transparency in drug pricing worldwide, 2) on the reported effects of drug pricing transparency initiatives on drug price, and 3) on the limitations and barriers of the implementation of drug pricing transparency.
METHODS: Databases such as Medline-Ovid, Cochrane Central Register, PubMed, and Science Direct were used to search for relevant literature from inception to February 2018. A manual search of grey literature such as policy papers, governmental publications, and websites was also performed to obtain the information that was not available in the articles. Using narrative synthesis, the results were critically assessed and summarized according to its context of drug pricing approaches.
RESULTS: Of the 4382 relevant articles located, 12 studies met the inclusion criteria for drug price transparency initiatives. Only 3 studies reported the outcomes on the regulation of drug prices. Two studies in South Africa showed that price transparency initiatives did not necessarily reduce drug prices. Another study in the Philippines indicated a reduction in medicines' price based on the effects of government-mediated access prices. The limitations and barriers in price transparency initiatives include fragmentation of the healthcare system and nondisclosure of discounts and rebates by pharmaceutical companies.
CONCLUSION: Drug pricing transparency initiatives have been implemented in many countries and commonly coexist with a country's pricing policies. Nevertheless, due to sparse evidence, the effect of drug price transparency initiatives on price control is still inconclusive.