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AIXIAL Review #10

A long-awaited revision of the HAS’s evaluation doctrine for clinical trials

In order to make France the leading country for clinical trials in Europe, in accordance with the Health Innovation 2030 plan, Olivier Véran has asked the HAS to initiate work on clinical research methods and to modify its evaluation criteria accordingly (see the letter of referral published by NILE[1], accompanied by a note from this agency[2]).

The Minister thus designates the new methods used to develop presumed innovative therapies likely to obtain early access. This request is in response to an increase in the rapid development of therapies increasingly aimed at small target populations, which poses serious difficulties in carrying out randomized controlled trials (RCTs) due to recruitment difficulties (the breakdown of frequent diseases is similar to the problem of rare diseases).

In order to develop its doctrine, the HAS has launched a call for contributions on the development of methodological expertise in new types of clinical trials (it will be open until November 30[3]).

The mission entrusted to the HAS generates significant expectations on the part of industry, practitioners and all clinical research professionals, evaluators and patients. Helping to discover and make available as quickly as possible health products that bring real progress for patients is of undeniable interest. The revision of the HAS doctrine on clinical trials should make it possible to improve the transparency and predictability of its opinions on them.  To this end, the HAS is expected to integrate into its doctrine new so-called unconventional trial methods such as master protocols with fixed or adaptive designs, frequentist or Bayesian statistical analyses, but also trial methods carried out in silico using virtual cohorts.

Recommendations on the use of artificial intelligence (AI) to build and conduct trials would also be welcome. But to be even more useful, the HAS could include in its doctrine rules to make its positions explicit in the face of the inevitable increase in uncertainty surrounding the results of any short development plan: what uncertainties will be acceptable to the HAS? On what criteria will it accept them and under what conditions? What real-life evidence will it be waiting for before it can remove them? If necessary, how will the study requests included in the contracts between the company and the CEPS be elaborated (uncertainty management contract, performance contracts, contracts established when an uncertainty variable may cast doubt on the transposability of the data to real life or when the product’s performance can be optimized by an action provided by the company (see LEEM-CEPS framework agreement of March 5, 2021[4])?

Explicit opinions dissociating the scientific value of the facts from the discursive arguments of the deliberation phase, the latter being stated just as transparently as the facts, in order to arrive at the opinion of the HAS on the sufficiency or otherwise, as well as on the level of an expected service, would undoubtedly constitute the most important step towards legibility and predictability of opinions and why not inspire other agencies which are also working on updating their doctrine.

It remains to be explained how the reworking of the HAS doctrine will contribute to making France the leading European country in clinical trials and will it succeed in finding and developing breakthrough health products? Which would require above all, from our point of view, incentives and support for a certain type of research more than a change in evaluation doctrine (see next post: To each his own DARPA?).

[1] https://www.nile-consulting.eu/drop/21104-Lettre_de_saisine_OV-HAS.pdf

[2] https://www.nile-consulting.eu/notesdenile/211005-notedenile-HAS_Essais_cliniques.pdf

[3] https://www.has-sante.fr/jcms/p_3295941/fr/appel-a-contribution-sur-le-developpement-de-l-expertise-methodologique-dans-de-nouveaux-types-d-essais-cliniques

[4] https://solidarites-sante.gouv.fr/IMG/pdf/accord_cadre_21-24_signe.pdf

— Written by Jacques Massol, MD PhD,  AIXIAL consultant

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