OBJECTIVES

Since September 2017, patient associations (PAs) have been able to support French HTA assessments conducted by the “Haute Autorité de Santé” (HAS) through contribution of their insights. As PAs are particularly active in the orphan drugs (ODs) field, this research aims to review appraisals of ODs since the beginning of this new initiative.

METHODS

All ODs granted with a European marketing authorisation (MA) and assessed by the HAS between 1^st October 2017 and 31^th May 2019 were identified. Corresponding HAS appraisals were reviewed to extract the following data: request type, appraisal date, indication, medical benefit (SMR), improvement of medical benefit (ASMR), target population, and availability through an Authorisation of Temporary Use (ATU). Related PA contributions and transcripts of the assessment commissions were also considered.

RESULTS

A total of 59 OD appraisals were identified, of which 15 (26%) were associated with a contribution of PAs. The first contribution identified was dated January 2018. Among these appraisals, 86% (13/15), 7% (1/15), and 7% (1/15) received substantial, moderate, and low SMR appraisals respectively. Meanwhile, 75% (33/44), 3% (1/44), 11% (5/44), and 11% (5/44) of OD appraisals without PA contributions received SMR appraisals of substantial, moderate, low, and insufficient. ASMR appraisals for ODs with PA contributions were III in 40% (6/15), IV in 46% (7/15), and V in 14% (2/15), compared to 5% (2/44), 27% (12/44), and 45% (20/44) respectively in ODs without PA contributions. Where available, PA contributions were generally discussed in HAS transcripts.

CONCLUSIONS

Overall, the option to contribute was widely used by PAs to support OD appraisals. Although the direct impact of PA contributions is difficult to assess, results suggest a higher level of SMR and ASMR granted to ODs with associated PA contribution, alongside increasing involvement of associations through this new opportunity.