Following the November ministerial report on exceptional access schemes and the public consultation on the ‘therapeutic bet’ doctrine, the Haute Autorité de Santé (HAS) has released a practical framework to manage clinical uncertainty. The announcement, (Early access to medicines presumed to be innovative: for a better-informed bet), was issued on 18 December 2025.
While earlier communications established the principle of the ‘therapeutic bet’ – a formal policy of recommending reimbursement despite incomplete clinical data – this latest update provides the Commission de la transparence (Transparency Committee) with a specific operational tool. This takes the form of an analysis grid based on six criteria, including disease severity, treatment rarity, and the strength of preliminary clinical results. This move follows the joint ministerial report by the Inspection générale des affaires sociales and the Inspection générale des finances, which highlighted the need for better fiscal regulation of the accès précoce (early access) scheme and other exceptional access pathways.
Professor Lionel Collet, President of the Haute Autorité de Santé, oversaw the implementation of this grid to ensure that decisions are more predictable. The new methodology requires the committee to explicitly justify the level of risk accepted when granting early access. This provides a clear structure for evaluating whether the absence of alternative treatments justifies an assessment based on potential rather than proven long-term efficacy.
The measures are being applied immediately to all pending and future applications. By standardising how uncertainty is measured, the authorities aim to maintain rapid access for patients while ensuring that public funds are not directed towards treatments with insufficient promise.
The move creates a more demanding environment for clinical submissions. Success will depend on the ability to provide robust evidence that aligns with the new six-point evaluation criteria. Higher levels of transparency regarding clinical gaps will be expected, and the likelihood of securing reimbursement will increasingly hinge on the precision and justification of the initial therapeutic assessment.
Source: Haute Autorité de Santé (HAS)
Link: Early access to medicines presumed to be innovative: for a better-informed bet
Date: 18 December 2025