It is predicted that by 2022 there will be about 16 "Advanced Therapies Medicinal Products" (ATMPs) on the market, about 2-4 new therapies per year. There are currently 11 ATMPs approved. According to the annual report of the "Alliance of Regenerative Medicine (ARM)", by the end of 2019 more than 1,000 clinical trials worldwide were investigating regenerative/ advanced therapies for various diseases, 94 of which are in phase 3, 591 in phase 2, 60% in oncological indications alone. Among the best known already approved are the CAR-T cell therapies (2 approved in Europe, 1 in the approval process) and gene therapies for spinal muscular atrophy (also 2 approved in Europe, 1 in the approval process). Two Horizon Scanning reports –a cooperation project of AIHTA and Tirol Kliniken- have dealt with a look into the near future of further gene therapies.

The European Medicines Agency (EMA) classifies ATMPs into three main types: Gene therapeutics and somatic cell therapeutic drugs and tissue engineering products. The two reports should address the questions for which indications CAR-T cell therapies and other ATMPs/gene therapies are in development and by when a marketing authorisation is expected. To answer the questions, a systematic search of study registries was performed to identify gene therapies in development and ATMPs, followed by the extraction of data on the identified ongoing clinical trials, complemented by a search of the EMA database of drugs under evaluation to identify those therapies that are expected to be approved soon. Finally, published information on the products, their indication and patient population was collected. The search identified 32 ATMPs in 8 broad indication areas and 13 CAR-T cell therapies in haematological malignancies at an advanced stage of development.