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AIXIAL Review #11

Compassionate access to presumed innovative medicines: To avoid turning hope into despair

The new French exemption system of access to medicines (early/compassionate access) which replaces the ATU and RTU since last July, is welcome. It simplifies a system which, although innovative at the time, had become highly complex and was even considered by some to be a deterrent. 

The reduction of exemption access modes to three categories instead of seven or eight, a one-stop shop, a link between ANSM and HAS, the setting of maximum response times by the authorities, and pre-filing consultations are all facilitating elements that suggest a simpler, more predictable, more responsive system, with the main expected consequence of faster access to the medicinal products concerned. We also welcome the dematerialisation of application procedures and the support processes put in place, particularly for manufacturers[1]. 1] The collection of the data required for such access is certainly not without its practical difficulties[2] but we are counting on the goodwill of all concerned to find ways of facilitating it without overburdening practitioners, who will thus be more available to their patients.

According to the Ministry of Health, „this reform makes it possible to simplify and harmonise procedures and to guarantee immediate access and care for patients“[3].

However, such a reform should not be seen as merely simplifying and designed to please the authorities and the industry „at the same time“. Let us not confuse the ends with the means. Its purpose is to improve as much as possible the coverage of urgent vital needs, needs that usually concern patients who have reached the final stage of their illnesses and for whom it is a last hope. This reform will only be a success if it does so.

In the context of compassionate access requests, the hope raised by this type of drug, once it is considered as a serious option by the therapist with his patient, generates a last hope that could turn into despair if the manufacturer refuses to give his agreement for fear of adverse effects.
Patients and prescribers therefore expect speed and transparency, but also humanity and ethical responsibility from both industry and the authorities.

[1] Among the documents intended to facilitate the process is a guide to accompanying laboratories https://www.has-sante.fr/upload/docs/application/pdf/2021-06/acces_precoces_-_guide_accompagnement_des_laboratoires.pdf.

[2] the exhaustive collection of data in accordance with the PUT-RD established with the HAS will require resources to be distributed throughout the territory so as not to create a geographical inequality of access as well as fair compensation for the personnel concerned by the collection and the elaboration of a „framework agreement“ linking collection operators to funding pharmaceutical companies…

[3] https://solidarites-sante.gouv.fr/soins-et-maladies/medicaments/professionnels-de-sante/autorisation-de-mise-sur-le-marche/article/autorisation-d-acces-precoce-autorisation-d-acces-compassionnel-et-cadre-de

— Written by Jacques Massol, MD, PhD,  AIXIAL consultant

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