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The Haute Autorité de Santé (HAS) released a policy paper on 24 November 2025, entitled ‘Doctrine for taking the therapeutic bet into account during the evaluation of medicines’ (Doctrine de prise en compte du pari thérapeutique lors de l’évaluation des médicaments – Consultation publique). This document aims to formalise how the Commission de la transparence (Transparency Committee) handles drugs for which clinical data are incomplete or show high levels of uncertainty at the time of market entry.
Professor Lionel Collet, President of the HAS, oversaw the release of these guidelines. Previously, decisions involving a ‘therapeutic bet’ – where a drug is recommended for reimbursement despite uncertainty – were made on a case-by-case basis without a consolidated public framework. The new measures establish specific criteria under which the committee may accept a gamble on a drug’s efficacy, particularly for rare diseases or life-threatening conditions where traditional large-scale trials are not feasible.
A public consultation window is open from 24 November 2025 – 24 January 2026. Stakeholders are invited to provide feedback via an online questionnaire. The final doctrine is expected to be adopted by the HAS Board in the first half of 2026. It will apply to all subsequent assessments of innovative treatments seeking inclusion on the list of medicines reimbursed by national insurance.
The framework will provide more predictable, yet stringent, conditions for market access when clinical evidence is limited. Clearer requirements for post-authorisation data collection will be established. This ensures that early access does not compromise the rigour of long-term evaluation, as the HAS will systematically review whether the initial bet was justified once further data are available.
Source: Haute Autorité de Santé (HAS)
Link: Doctrine de prise en compte du pari thérapeutique lors de l’évaluation des médicaments – Consultation publique / Doctrine on taking account of therapeutic ‘betting’ when assessing medicines – Public consultation
Date: 24 November 2025