OBJECTIVES: In France, only products recognized as innovative by the national HTA body (HAS) are eligible to funding on top of DRG. This may create a gap in patient access for expensive products not recognized as innovative by HAS but considered as irreplaceable by clinicians. In some exceptional cases, the Ministry of Health thus allocates a specific exceptional budget handled at national level on a case by case assumption. No formal regulation exists for this specific funding which is allocated at the discretion of the Minister of Health and eventually of the health care department director. This study objective was to provide a description of innovative medicines benefiting from this funding to ensure patient access to treatment.

METHODS: Based on experts’ knowledge and desk research, we searched for innovative products for which an exceptional funding was granted.

RESULTS: Three products were identified. Vedolizumab in the treatment of Crohn disease, was granted an early-entry program (ATU) prolonged 2,5 years after approval and was not eligible for budget on top of DRG. Exceptional funding was granted only for treatment continuation in patients that already have initiated therapy but not for new patients. Bevacizumab, in triple negative breast cancer, and trabectedine in sarcoma were respectively delisted according to HAS assessment concluding to low innovation. Both are funded for new patients (initiation of treatment). Recently, another product got attention from the Health Ministry to obtain such a funding but is still under evaluation.

CONCLUSIONS: Conditions of access to this exceptional funding are not regulated. The lack of formal process creates inequality in patient access to innovative therapies. While some products may obtain such a funding, other multiple products in the same situation are left behind. There is a need for formal and transparent process to ensure equity among innovative products.