France to update French National Authority for Health medicinal product doctrine!

In an article published in the British Medical Journal, the HAS reaffirms the importance of randomized clinical trials (RCTs), while allowing adaptations to the traditional model to overcome uncertainties or impossible evidence. The randomized clinical trial therefore remains the standard… except in "exceptional" circumstances.

The HAS presents external comparisons as reasonable alternatives in exceptional conditions, permitting real-life studies and emerging methods to be used as a complement… but NOT as a substitute, adding uncertainty to the regulatory standard.

The improvements are therefore fairly minor and more or less redundant with the developments observed in recent evaluations.

However, the methodological changes that industry has sought were intended to overcome the impossibility of providing comparative studies for orphan medicinal products or for rare diseases without comparators, or to enable access to the market for innovations in oncology following the negative evaluations noted, which impede patient access to these treatments with long-standing, albeit inexpensive, alternatives.

The dual objective – of guaranteeing rapid access to medicinal products for patients while also respecting the duty to propose treatments whose efficacy has been clearly demonstrated – is laudable yet disappointing considering industry expectations regarding the challenge of market access for therapeutic innovations.

Link to BMJ article below:

Authored by Charlotte Damiano