INTRODUCTION: The role of patient advocacy groups as part of European countries health technology assessment (HTA) has steadily increased in recent years. Since 2016, the HAS (Haute Autorité de Santé, France) has begun to include patient opinion as part of the assessment for the reimbursement of drugs and medical devices, and advocacy groups are encouraged to share contributions on their point of view of the disease and the treatment.

OBJECTIVES: The objective of this research is to identify in which therapeutic areas patient groups are most involved in HTAs from HAS.

METHODS: Drugs receiving their TC (Transparency Committee) opinion between January 2018 and December 2018 and for which a contribution had been made by a patient advocacy group were extracted from the HAS website. Therapeutic areas, orphan status and TC opinion dates were extracted from the EMA (European Medicines Agency) and from the open platform for French public data in June 2019.

RESULTS: 27 drugs receiving their TC opinion between January 1, 2018 and December 31, 2018 and that received at least one contribution from a patient advocacy group were identified. 21 different patient advocacy groups submitted their opinion, with some (n=5), providing contributions for multiple drugs, giving a total of 30 contributions. Oncology had the highest number of contributions (n=9) followed by infectious diseases (n=8) and neurology (n=6). A significant number of orphan drugs (n=7) also received contributions (n=9).

CONCLUSIONS: Oncology drugs, particularly those with orphan drug designation, are subject to receiving more contributions than other therapeutic areas. One explanation for this trend can be potentially explained by the severity or life-threatening nature of the disease and the inflated public interest in cancer. Further research is needed to understand exactly how influential these contributions are during the HAS assessment and what impact they have on TC opinion in France.