With changes to the pricing and access processes such as the new Accord Cadre and the soon-to-be implemented ATU reform likely to impact pricing and access of orphan products in France, Cécile Matthews, Bhavesh Patel, and Owen Male look at how French payers have valued rarity in the past in this article published in PM LiVE.
Pricing and market access in France operates on a dual system, under which health technology assessments (HTAs) are conducted first with the aim of informing subsequent pricing negotiations. To do this, the HTA body, the CT (Commission de la Transparence – Transparency Commission), provides guidance in two main forms: a comparative clinical benefit rating, the ASMR (Amélioration du Service Médical Rendu) and a target patient population.
In the case of rare diseases, however, there seems to be an intrinsic flaw in this system, as comparators are often not available and epidemiology estimates are often unreliable. This leaves CEPS (Comité Economique des Produits de Santé, pricing committee) with many uncertainties in managing pricing negotiations. We set out to understand how French payers manage these uncertainties and assess the value they place on rarity. We analyzed 22 non-oncology orphan drugs (ODs) that received EMA approval between July 2012 to the end of 2019, focusing on chronic, non-curative treatments. Dosing assumptions followed EMA or HAS (Haute Autorité de Santé) guidelines. The number of patients eligible for treatment and ASMR ratings
were taken from CT (Avis) reports. Annual treatment costs (ATCs) were calculated using ex-manufacturer list prices (Tarif de responsabilité HT) in Legifrance, or ATUc prices.