[To reimburse or not? Evaluating expensive drugs differently]

Area of Science:

• Health economics
• Pharmaceutical policy
• Clinical trial design

Background:

• Health insurance reimbursement relies on placebo-controlled trials demonstrating significant clinical benefit.
• This evidence standard is challenging for rare and chronic diseases, impacting drug accessibility.
• Rising costs of new rare disease drugs strain health insurance solidarity.

Purpose of the Study:

• To explore challenges in drug reimbursement for rare and chronic diseases.
• To propose a new collaborative model for drug development and reimbursement.
• To address the sustainability of health insurance coverage amidst expensive new therapies.

Main Methods:

• Analysis of current reimbursement practices and their limitations.
• Review of surrogate endpoints and post-marketing studies in drug evaluation.
• Examination of collaborative drug development models and their potential benefits.

Main Results:

• Placebo-controlled trials are often insufficient for rare/chronic diseases.
• Surrogate endpoints and post-marketing studies present expediency solutions but require further validation.
• Collaborative development models offer a framework for shared risk and reward.

Conclusions:

• Current reimbursement models are inadequate for many drug types.
• A shift towards collaborative drug development is needed to balance innovation, cost, and patient access.
• New models can enhance the solidarity of health insurance for all citizens.