Despite new pricing rules, France still offers opportunities for Early Access to Orphan drugs




Since March 2021, there have been some changes in the pricing of pharmaceuticals in France, leading progressively to a somehow improved access to treatment. These changes came after the signature of new contractual agreement between the French Pharmaceutical Companies Association (LEEM) and the Ministry of Health’s (MoH’s) Economic Committee for Health Products (CEPS) and did not exclude orphan drugs.

Accordingly, in the case where the annual cost of a therapy is determined to be more than EUR 50,000 ($52,599), a flat rate payment may be agreed upon in exchange for a price that is in line with the prices that are applied internationally. A positive funding decision may also be linked to a performance-based risk sharing agreement, with pre-defined rebates being determined by the therapeutic performance of the product.

In these regards, orphan and non-orphan drugs with a therapeutic improvement rating (Amélioration du Service Médical Rendu, ASMR) of I to V may also benefit from the new fast track pathway. The ASMR value is key to obtaining a favorable price in France. Together with the pharmaco-economic evaluation performed by the Commission for Economic Evaluation and Public Health (Commission Évaluation Économique et de Santé Publique, CEESP), the level of ASMR has a major impact on companies’s ability to negotiate a drug price.

As for the fast track pathway, it allows a drug to have a first price set within a maximum of 15 days under the following conditions:

  • ASMR I to III drugs must have obtained a favorable pharmaco-economic evaluation.
  • ASMR IV drugs must have obtained a favorable pharmaco-economic evaluation and be likely to have a negative budget impact.
  • ASMR V drugs must have a proposed price below the comparator price.

Of note, 30% of orphan drugs are deemed to bring therapeutic improvement and 4% of orphan drugs are often excluded from reimbursement due to an insufficient clinical value, compared to 11% for non-orphan drugs. The rest are either marketed under an early access scheme or do not have an ASMR rating.

Compared to non-orphan products, orphan drugs are more favorably assessed by the French National Authority for Health (Haute Autorité de Santé, HAS), resulting in a positive impact on price setting. The recent changes to the pricing rules will further streamline the negotiation environment for orphan-drug makers, ensuring early access for orphan patients and a fixed budget for French payers in the case of very expensive orphan drugs.

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