Journal of Market Access & Health Policy

Population-adjusted indirect comparisons (PAICs), including Matching-Adjusted Indirect Comparison and Simulated Treatment Comparison, are increasingly used to inform health technology assessments. These methods offer a pragmatic approach to generating comparative evidence between treatments when head-to-head trial data are unavailable and standard indirect treatment comparison methods are unfeasible. In rare diseases, however, PAICs often face substantial methodological challenges arising from small sample sizes, limited covariate overlap, and the frequent use of unanchored comparisons that rely on unverifiable assumptions. These limitations can lead to unstable estimates, reduced precision, and bias that may undermine the reliability of findings. Methodological refinements—such as optimized weighting, Bayesian approaches, and doubly robust estimators—provide some improvements but do not resolve these fundamental issues. Current European Joint Clinical Assessment guidance recommends that anchored PAICs be applied with great caution, while unanchored PAICs are considered highly problematic, and other methods should be used instead. We argue that PAICs can play a supportive role within a multidimensional and deliberative HTA process, contributing to comparative assessment alongside other evidence sources when available data are limited. However, their results require careful interpretation and transparent communication of uncertainty. Future research should prioritize the further development of formal frameworks to quantify bias and systematically assess robustness, thereby preventing overstatement of the credibility of PAIC-derived evidence in rare disease contexts.

Background: Value-based pricing has the potential to contribute to the appropriate allocation of healthcare expenditures. We developed the “MARIE”, a qualitative scheme that evaluates broad value elements without requiring a comparator to estimate new drug prices. In this study, we updated the conversion table that monetizes points calculated from value elements. Methods: We investigated and calculated the daily drug prices at the time of listing for drugs containing new active ingredients that were included in the National Health Insurance Drug Price List from fiscal year 2015 to 2024, summarizing the data using descriptive statistics. Results: New drug prices are listed annually in Japan, so we updated the conversion table using current drug prices to maintain continuity. This study also observed a trend where the median daily price tended to be higher as the maximum number of patients decreased. Conclusions: The MARIE method, our developed framework to qualitatively evaluate the various values of drugs, monetizing points calculated based on the elements of value via the conversion table, plays a crucial role. Updating the conversion table based on the latest data maintains continuity with the current drug pricing system and is considered to contribute to the social implementation of the MARIE method.

European citizens made it clear in 2024 that healthcare should be the EU’s top priority for shaping the future of Europe. This sentiment reflects the escalating health challenges facing the region, driven by ageing populations, rising chronic disease burdens, and persistent disparities in access to healthcare. Despite these growing needs, the most recent data on health spending as a share of gross domestic product (GDP) is just slightly above the pre-COVID-19 pandemic level, and spending on pharmaceuticals specifically has remained a stable proportion of healthcare spending over the last 20 years. Austerity measures have profoundly impacted the health sector and pharmaceutical industry, more so than any other sectors, despite the wide range of health and socioeconomic benefits medicines bring to patients, the health system, and society. Such trends are not keeping pace with evolving population demographics and disease prevalence. To secure a healthier, more equitable future, Europe must urgently increase health investments, optimise health systems, and address unmet needs by supporting fast uptake of pharmaceutical innovations. Policymakers must work with all stakeholders to ensure stronger and sustained investments in health innovations by (i) adopting a long-term vision, moving away from short-term thinking and valuing health appropriately to drive economic growth; (ii) implementing transformative policies that eliminate ineffective and wasteful spending; (iii) promoting value-based approaches to improve patient access and system sustainability, and (iv) creating incentives that attract greater investments to strengthen Europe’s competitiveness and safeguard against health threats.