Challenges raised by the economic evaluation of CAR-T-cell therapies. The review by the French National Authority for Health.

Since 2013, the process of pricing of innovative drugs by the French National Health Insurance has considered both cost-effectiveness and budget impact. CAR-T cell therapies were first subject to economic evaluation from 2019 in France. We aim to describe the process and results of the economic evaluation of tisagenlecleucel and axicabtagene ciloleucel as well as the challenges these evaluations raised.

How much does the hospital stay for infusion of anti-CD19 CAR-T cells cost to the French National Health Insurance?

Chimeric antigen receptor T-cells (CAR-T cells) have the potential to be a major innovation as a new type of cancer treatment, but are associated with extremely high prices and a high level of uncertainty. This study aims to assess the cost of the hospital stay for the administration of anti-CD19 CAR-T cells in France. Data were collected from the French Medical Information Systems Program (PMSI) and all hospital stays associated with an administrated drug encoded 9439938 (tisagenlecleucel, Kymriah®) or 9440456 (axicabtagene ciloleucel, Yescarta®) between January 2019 and December 2020 were included.

Estimating Rebates and Other Discounts Received by Medicare Part D.

Importance: Spending in Medicare Part D continues to increase. Yet, studies of Medicare Part D are plagued by a common limitation: none can fully account for confidential rebates and other discounts that drug manufacturers and pharmacies pay to Medicare Part D plans.

Objectives: To review existing methods and to propose an approach for estimating rebates and other discounts received by Medicare Part D.

Characteristics of Clinical Trials Launched Early in the COVID-19 Pandemic in the US and in France.

Based on hierarchical classification and logistic regression of early US and French COVID-19 clinical trials we show that despite the registration of a large number of trials, only a minority had characteristics usually associated with providing robust and relevant evidence.

Why France Spends Less Than the United States on Drugs: A Comparative Study of Drug Pricing and Pricing Regulation.

Unlabelled: Policy Points  Spending on prescription drugs is much higher per capita in the United States than in most other industrialized nations, including France.  Lower prescription drug spending in France is due to different approaches to managing drug prices, volume of prescribing, and global health budgets.  Linking a drug's price to value both at the launch of the drug and over its lifetime is key to controlling spending.

Was It Worth Introducing Health Economic Evaluation of Innovative Drugs in the French Regulatory Setting? The Case of New Hepatitis C Drugs.

Objective: This paper constitutes the first attempt to draw lessons from the recent uptake of health economic evaluation of innovative drugs in the French regulatory framework.

Study Design: Taking the example of new direct-acting antivirals against hepatitis C virus, the paper asks whether and how the cost-effectiveness (CE) opinions issued by the French National Health Authority improve the information available to support the pricing decisions.

Methods: The analysis compares the assessment of these drugs based on three different sources: CE opinions, clinical opinions, and the published cost-utility analyses (CUA) available in the literature and identified through a systematic review.

ETHICS EVALUATION REVEALING DECISION-MAKER MOTIVES: A CASE OF NEONATAL SCREENING.

Objectives: This paper aims to describe the added value of combining cost-effectiveness and ethical evaluations when the preferences of the decision maker toward cost-effectiveness evaluation outcomes are not known, with the French national neonatal screening of cystic fibrosis (CF) as a case-study.

Methods: A cost-effectiveness analysis comparing four CF neonatal screening strategies, with or without DNA testing, was performed. Ethical positions toward their outcomes were described.

[Haute Autorite de sante opinion on cost-effectiveness of health products : results and perspectives].

Since 3rd October 2013 in France, drug companies applying for reimbursement of an innovative and expensive drug or medical device are required to provide the French National Authority for Health (HAS) with a cost-effectiveness assessment of their product. After a methodological audit of the economic evaluation submitted by the drug company, the Health Economics and Public Health Committee (CEESP) issues an opinion on the expected or observed cost-effectiveness. This opinion is sent to the Pricing Committee (CEPS) which determines the price of the product.

Online dissemination of clinical practice guidelines as narrative texts and structured pathways: a case study with the treatment of type 2 diabetes.

Recently, National agencies in charge of the development of clinical practice guidelines (CPGs) have started to improve the usual narrative CPGs to provide guidance for different clinical pathways. In France, in conjunction with the development of the type 2 diabetes National CPGs, we have developed the system RecosDoc-Diabète which allows to interactively build a patient-centred pathway and get the appropriate recommendations. National narrative CPGs and RecosDoc-Diabète were published and made available online at the same time (February 2013).

HTA agencies facing model biases: the case of type 2 diabetes.

When evaluating new drugs or treatments eligible for reimbursement, health technology assessment (HTA) agencies are repeatedly faced with cost-effectiveness analyses that evidence lack of adequate data and modeling biases. The case of type 2 diabetes illustrates this difficulty. In spite of its high disease burden, type 2 diabetes is poorly documented through existing cost-effectiveness analyses.