Authors' objectives: This report is an update of an earlier Horizon Scanning report from 2020, in which 32 ATMPs were identified. Since August 2020 six of these new ATMPs have been approved by EMA and another five will be decided upon until 2023. According to the European Medicines Agency (EMA) ATMPs can be classified into three main types: Gene therapy and somatic-cell therapy medicinal products as well as tissue-engineered products. According to market analyses, in 2022 535 ATMPs are investigated in Phase 1 to 3 trials, another 1451 are in preclinical stages. Coagulation factor VIII (haemophilia A) remains the most common target for non-oncology indications and coagulation factor IX (haemophilia B) has risen to second most common indication. This report aims to address the questions, for which indications gene therapies and ATMPs are under development and by when an approval can be expected.

Authors' results and conclusions: Results: The search in ClinicalTrials.gov yielded 58 hits (Phase 2/3, 3), of which 34 were new studies. After deduplication and clustering of the same therapies, 34 different ATMPs (without CAR-T) were identified. The areas of indications are a diversity of genetic diseases and encompass eight broad indication groups (haemophilia, metabolic -, ophthalmologic -, musculoskeletal -, vascular -, nephrologic -, dermatologic - and neurologic disorders). Since August 2020 six new ATMPs have been approved by EMA (2020: 3, 2021: 1, 2022: 2) and another five will be decided upon until 2023. Conclusion: There are many challenges in evaluating these therapies. They have received advance praise and are often referred to as "curative" or "disruptive" technologies, although there is little long-term data for the few therapies already approved.

Authors' recommendations: The challenge now is that the potential promise of gene therapies must live up to its expectations, and it is the role of HTA to closely monitor the actual effectiveness of the respective therapies. This HS report only represents a "snapshot" and is not as reliable as international initiatives and their systematic and permanent activities, as horizon scanning is time-consuming. In 2022, Austria will join the International Horizon Scanning Initiative (IHSI), which signifies a major step forward in the efforts to identify new therapies at an early stage that could have an impact on health and budgets in the future.

Authors' methods: A systematic search in trial registries was conducted to identify gene therapies and ATMPs under development, followed by the extraction of data on the identified ongoing clinical trials (restricted to phase 2/3 and 3), complemented by a search in the EMA-database on medicines under evaluation to identify those therapies closest to approval. Finally, published information on the products and the current stage of development/regulation was collected and subsequently transformed into short "vignettes".

Project Status: Completed

URL for project: https://aihta.at/page/horizon-scanning-zu-gentherapien-car-t-zelltherapien-und-atmps-update-2022/de

Year Published: 2022

URL for published report: https://eprints.aihta.at/1383/1/Policy%20Brief_006b_1st%20Update.pdf

URL for additional information: https://eprints.aihta.at/1383/

English language abstract: An English language summary is available

Publication Type: Rapid Review

Country: Austria

Organisation Name: Austrian Institute for Health Technology Assessment

Contact Address: Garnisongasse 7/20, A-1090 Vienna, Austria

Contact Name: office@aihta.at

Contact Email: office@aihta.at

Copyright: HTA Austria - AIHTA GmbH