Health technology assessment in Japan: a work in progress.

Background: In Japan, pharmacoeconomic requirements for list-price adjustment were institutionalized in April 2019 following provisional implementation of a new Health Technology Assessment (HTA) program 2016-2019. Since April 2019, submission of cost-effectiveness evidence to the Central Social Insurance Medical Council (Chuikyo) as part of the Japanese Ministry of Health, Labour, and Welfare has been mandatory for selected pharmaceuticals and medical devices.Methods: Based on a review of publications and commentaries since April 2019, together with views from a group of experts on key issues to be addressed, this report provides an update on recent HTA developments and key challenges still to be addressed.Results and Discussion: Japan's new HTA program is a first step toward development of a universal healthcare system that can be sustainable for many years into the future. Currently, Japan's HTA program requires provision of incremental cost-effectiveness ratios (ICERs) as evidence, with quality-adjusted life years as the preferred outcome measure. Prices can be adjusted both upward and downward according to the degree of the ICER estimate. Japan is the first country to have adopted an algorithmic method for "ICER-based" pricing; however, HTA measures that extend beyond a single ICER estimate are needed to take full advantage of HTA in the future. In particular, generation of evidence of value should support changes to the healthcare system so that incentives for innovation are not diminished while industry and government are not overburdened by the generation or assessment of evidence. There is a need to ensure scientifically sound HTA expertise across all sectors in Japan, and therefore enhancement of HTA literacy and capability among healthcare professionals, academia, government, and industry should be a priority.

Practical implications of using real-world evidence (RWE) in comparative effectiveness research: learnings from IMI-GetReal.

In light of increasing attention towards the use of real-world evidence (RWE) in decision making in recent years, this commentary aims to reflect on the experiences gained in accessing and using RWE for comparative effectiveness research as a part of the Innovative Medicines Initiative GetReal Consortium and discuss their implications for RWE use in decision-making.

Designing and incorporating a real world data approach to international drug development and use: what the UK offers.

Assessments of the safety, efficacy and appropriate use of new medicines lie at the heart of treatment development and subsequent adoption in clinical practice. Highly controlled randomised clinical trials routinely inform decisions on the approval, coverage and use of a medicine. Researchers and decision makers have become increasingly aware that these experimental data alone are insufficient to address those decisions fully. Real world data recorded from routine healthcare delivery by healthcare professionals and patients help provide a more complete picture of care. The UK, with its connectivity and rich longitudinal patient records, accumulated research and informatics experience and National Health Service, provides an exemplar of how real world data address a wide range of challenges across drug development.

An observational study of the effect of two thiazolidinediones on blood lipid levels: Rosiglitazone and pioglitazone in routine clinical practice.

BACKGROUND: Rosiglitazone maleate and pioglitazone hydrochloride are established antihyperglycemic agents that are effective when used as monotherapy or in combination with other medications. However, the data regarding the effects of these agents on blood lipid levels are contradictory. OBJECTIVE: The aim of this study was to determine whether the use of rosiglitazone and pioglitazone in clinical practice is associated with any changes in blood lipid levels. METHODS: A retrospective chart review using electronic medical record data was conducted of patients with type 2 diabetes mellitus who were newly treated with either rosiglitazone or pioglitazone and had 1 lipid measurement within 6 months prior to and 12 months following initial thiazolidinedione (TZD) therapy. Outcome measures were mean changes in low- and high-density lipoprotein cholesterol (LDL-C and HDL-C, respectively). To control for differences in baseline characteristics and/or selection bias, the treatment cohorts were compared using multivariate statistical techniques. RESULTS: A total of 371 patients were included in the study; the pioglitazone cohort comprised 148 patients (82 women, 66 men; mean [SD] age, 64.9 [10.8] years) and the rosiglitazone cohort comprised 223 patients (113 men, 110 women; mean [SD] age, 66.1 [11.9] years). Pioglitazone-treated patients had a statistically higher mean baseline LDL-C compared with rosiglitazone-treated patients (125.0 mg/dL vs 116.6 mg/dL; P = 0.04). On average, LDL-C levels decreased over the study period, with no significant differences between the 2 cohorts (9.9 mg/dL vs 4.3 mg/dL for pioglitazone and rosiglitazone, respectively), although changes in both cohorts were statistically significant (P < 0.001). CONCLUSIONS: TZD therapy appears to be associated with a small decrease in LDL-C within the first 6 months after initiation. No differences in changes in LDL-C or HDL-C could be discerned between patients treated with rosiglitazone compared with pioglitazone.