OBJECTIVES : ATMPs are medicines for human use that can be classified into gene therapy, somatic-cell therapy and tissue-engineered medicines. If these are medicines with new active substances, ATMPs have to undergo a benefit assessment according to §35a Social Code Book (SGB) V. The objective of this analysis was to determine if ATMPs have the same Health Technology Assessment (HTA) requirements as other drugs in Germany.

METHODS : All §35a assessments for ATMPs publicly available on Federal Joint Committee’s (G-BA) website up to 20.05.2021 were evaluated. Evidence was screened on procedural level and analysed descriptively by two individual reviewers based on pre-defined evaluation criteria including study design, population, comparator, duration, endpoints and results.

RESULTS : For nine ATMPs a G-BA decision on the assessment of the additional benefit has been published until May 2021. Seven were designated an Orphan Drug Designation (ODD), for which the additional benefit is automatically granted as non-quantifiable. Four ATMPs have provided a Randomized Controlled Trial (RCT) and therefore fulfilled the standard G-BA requirements regarding study design. Regarding study comparator, for Orphan Drugs (OD) the additional benefit does not need to be shown against an Appropriate Comparative Therapy (ACT) as determined by G-BA. The analysis showed that ATMPs require studies with a matched study and label population, a duration of at least 12 months and results based on sufficient information on methodology and statistical analyses for validated patient-relevant endpoints. One Orphan ATMP with the presence of a RCT was granted a considerable additional benefit.

CONCLUSIONS : In conclusion, ATMPs have the same HTA requirements as other drugs in Germany. A crucial prerequisite for success in the German HTA is the presence of a RCT with the comparator as defined by G-BA with the adequate population and duration to achieve an additional benefit for non-OD and an additional benefit higher than non-quantifiable for OD.