Coverage and Pricing Recommendations of the French National Health Authority for Innovative Drugs: A Retrospective Analysis From 2014 to 2020.

OBJECTIVES: This study provides a retrospective analysis of the recommendations of the French National Health Authority on the reimbursement and pricing of innovative drugs. METHODS: The analysis includes drugs subjected to both economic and clinical evaluations in France from 2014 to 2020. Ordered logistic and quantile regressions are used to estimate the factors associated with the clinical value (SMR), the clinical added value (ASMR), and the incremental cost-utility ratio (ICUR) of innovative drugs. All variables used in the regression analyses are extracted from the Clinical and Economic Opinions for the 146 observations. RESULTS: Regression analyses indicate that 2 of the 5 official criteria, the efficacy-adverse events balance of the drug and its function, are significantly associated with the SMR rating. The ASMR is positively associated with the disease severity, the quality-adjusted life-year (QALY) gain provided by the drug, and the validation of the ICUR in the Economic Opinion. At the first quartile of the ICUR distribution (approximately €50 000/QALY), higher ICUR levels are observed for drugs with a smaller target population and for drugs claimed as more innovative. Higher ICUR levels are also observed for pediatric drugs and for drugs with no therapeutic alternative at the third quartile of the distribution (approximately €240 000/QALY). CONCLUSIONS: Not all official criteria of the SMR are associated with actual ratings obtained. Regarding the ASMR, the results support the idea of a convergence between the 2 independent clinical and economic appraisal processes. Finally, the factors influencing the ICUR level vary across the distribution of ICUR.

[HAS budget impact analysis guidelines: A new decision-making tool]. / Le guide d'analyse d'impact budgétaire de la HAS : un nouvel outil d'aide à la décision.

INTRODUCTION: Budget impact analysis (BIA) provides short and medium-term estimates on changes in budgets and resources resulting from the adoption of new health interventions. OBJECTIVE: The objective of this article is to present the main messages of the newly developed French National Authority for Health (HAS) guidelines on budget impact analysis : issues, recommendations and perspectives. METHODS: The HAS guidelines development process was based on data derived from a literature review on BIA (search dates : January 2000 to June 2016), an HAS retrospective investigation, a public consultation, international expert advice, and approval from the HAS Board and the Economic and Public Health Evaluation Committee. RESULTS: Based on its research findings, HAS developed its first BIA guidelines, which include recommendations on the following topics : BIA definition, perspective, populations, time horizon, compared scenarios, budget impact models, costing, discounting, choice of clinical data, reporting of results and uncertainty analysis. CONCLUSION: The HAS BIA guidelines are expected to enhance the usefulness of BIA as an essential part of a comprehensive economic assessment of healthcare interventions, which itself includes cost-effectiveness analysis and equity of access to healthcare.

The French National Authority for Health (HAS) Guidelines for Conducting Budget Impact Analyses (BIA).

BACKGROUND: Budget impact analysis (BIA) provides short- and medium-term estimates on changes in budgets and health outcomes resulting from the adoption of new health interventions. OBJECTIVE: The purpose of this study is to present the newly developed French National Authority for Health (HAS) guidelines on budget impact analysis as follows: process, literature review, recommendations and comparisons with other guidelines. METHODS: The development process of the HAS guidelines included a literature review (search dates: January 2000 to June 2016), a retrospective investigation of BIA previously submitted to HAS, a public consultation, international expert reviews and approval from the HAS Board and the Economic and Public Health Evaluation Committee of HAS. RESULTS: Documents identified in the literature review included 12 national guidelines, 5 recommendations for good practices developed by national and international society of health economics and 14 methodological publications including recommendations for conducting BIA. Based on its research findings, HAS developed its first BIA guidelines, which include recommendations on the following topics: BIA definition, perspective, populations, time horizon, compared scenarios, budget impact models, costing, discounting, choice of clinical data, reporting of results and uncertainty exploration. CONCLUSION: It is expected that the HAS BIA guidelines will enhance the usefulness, quality and transparency of BIA submitted by drug manufacturers to HAS. BIA is becoming an essential part of a comprehensive economic assessment of healthcare interventions in France, which also includes cost-effectiveness analysis and equity of access to healthcare.