France – Principles of medicinal products assessment and appraisal for reimbursement purposes

The Transparency Committee’s doctrine is designed to provide benchmarks and visibility with respect to the main criteria of medicinal products assessment and appraisal for reimbursement purposes.

Based on medical and scientific criteria, the doctrine needs to consider the number of dossiers presenting a high level of uncertainty, innovations and changes in clinical trial methodology, taking into account
patient care pathways.

In this context, highlighted by Dominique Polton’s report in 2015 and by the CSIS (French Strategic Council for the Healthcare Industries) in its July 2018 report, the Transparency Committee wanted to detail its methodology and update its doctrine with a view to improving reproducibility, transparency and fairness.

Following discussion by the Transparency Committee members, it was decided this document should be focused on:

 Clarification of the factors determining the CAV score and extension of eligibility conditions for major CAV;

 Assessment of innovation and the management of uncertainty using surrogate endpoint for example;

 Elements related to the predictability of CB and, in particular, an insufficient CB;

 Definition of a public health benefit and of the criteria used to assess it;

 Clarification on how quality-of-life data and real-life data are taking into account;

 Incorporation of the patient perspective.                                                                These notions have thus been included in the doctrine.

This doctrine should be considered as an evolving document, rather than a static text. What was important to me was to clarify – both internally within the HAS and with respect to industry and the public authorities – the necessary evolutions in the criteria guiding our decisions.

It is only by ensuring precise, fair assessment, taking into account the benefit provided by an innovation as well as the potential risks related to a treatment, that patients can benefit the most new medicinal products and that companies can be encouraged to innovate.

We hope that this new version of the doctrine will facilitate patient access to innovation and meet the expectations of all relevant stakeholders…