360 VIEW of the French EARLY Access Program
Author(s)
Cardon SC, Kachaner I, Bougeois M, Grand H, Cogez F
Pfizer SAS, Paris, France
OBJECTIVES : This descriptive analysis of French early-access program (“ATU de cohorte” - cATU) aims to provide a 360° view of ATU attractivity from every users (patients, Health Authorities and Industrials). METHODS : The analysis gathers several documents coming from various sources: ATU Publication, Annual Report coming from the French Health Authority (ANSM), Official Journal publications, Transparency Commission (TC, French Health Technology Assessment body) advices. The analysis includes all cATU obtained from January 1st 2016 until April 20th 2020 and is based on data of 55 substances. RESULTS : An average of 10 new cATU are authorized each year. The number of new ATU is stable over the time but we notice an exceptional demand in 2018 (20) which corresponds to development of Advanced Therapy Medicinal Products (ATMP). Between 5 642 (2018) and 11 909 (2016) patients benefit from this early access program each year. The number of patients tends to decrease as the proportion of ATMP with smaller target population is increasing. Market Authorization (MA) requests were submitted for all medicines and only one submission of out the 55 was withdrawn which supposed that Benefit/Risk ratio is well-estimated for most of the medicines. ATU enable patients to get access to the medicine on an average of 3.3 months prior MA approval. This access is even earlier for cATU in Oncology which enables patient to get access to the drug on an average of 4.8 months prior MA approval. Considering the full market access process in France, an average of 22 months is observed between ATU obtention and the Conventional Market Authorization (Price publication). CONCLUSIONS : cATU usage demonstrates its attractivity by giving patient an early access to innovative treatments with strong efficacy presumptions on unmet needs. ATU usage satisfied the main prerequisites of the law with regards to B/R ratio, ATU period duration and MA approval submission.
Conference/Value in Health Info
2020-11, ISPOR Europe 2020, Milan, Italy
Value in Health, Volume 23, Issue S2 (December 2020)
Code
PNS87
Topic
Health Policy & Regulatory
Topic Subcategory
Reimbursement & Access Policy
Disease
No Specific Disease