OBJECTIVES: The approval of Strimvelis from GlaxoSmithKline (GSK) for treatment of “bubble boy syndrome” raised questions in field of gene and cell therapy products (GCT), whether new models of modified HTA assessment methodologies are needed. The objective of our study is to overview HTA reports in GCTs in Europe and United States (US).

METHODS: A scoping review was performed on HTA reports in GCTs from exiting HTA agencies in Europe and US. The assessment reports planned to identify limited within 8 approved advanced therapy medicinal products (ATMPs) from European Medicines Agency (EMA) and 13 approved gene and cellular therapy products (GCTs) from US Food and Drug Administration (US FDA). The Language is limited in English and German.

RESULTS: CONCLUSIONS: The assessment process for GCTs is similar to other medical products but with more flexibility. For safety concerns, a long-term follow-up observation in a registry is recommended from different agencies. To assess cost-effectiveness, make appropriate health economic model, early involving payers and HTA in the development of GCTs is necessary.