OBJECTIVES: ATMPs comprise medicines for human use based on gene therapy, somatic cell therapy or bioprocessed tissue products. ATMPs are pharmaceutically manufactured drugs and mostly subject to central authorization requirements. Legally it is an ambiguous situation and more heterogeneously dealt with. ATMPs are assigned to method evaluation as well as to the AMNOG procedure for drugs. The underlying analysis will present and discuss the crucial steps from the start of the assessment to reimbursement.

METHODS: Guidelines (EMA, G-BA, IQWiG), respective legislation, consultations results were reviewed, and analyzed. Decision criteria and reasoning, assessment outcomes and potential impact on price negotiations were the main aspects for comparison.

RESULTS: ATMPs (Regulation (EC) No 1394/2007) are subject to benefit assessment in Germany, with a decision whether to be evaluated as a drug (e.g. Alofisel^®) or a method/device (e.g. Holoclar^®). By 2018, 16 ATMPs have been approved in Germany, seven on the basis of a central European approval. An ATMP is classified as a treatment method, if the correct administration has at least the same significance for a successful therapy outcome as its mode of action. Depending on the respective decision, an evaluation as method follows or it must undergo the AMNOG process. RCTs are the "gold standard" for a benefit assessment of new therapies, including ATMPs. Conduction of RCTs is not always possible for ATMPs which creates a disadvantage in the assessment right from the beginning. Otherwise no distinction is made between drugs and ATMPs in terms of reimbursement modalities. Outcomes based agreements could help overcoming inequalities and lead to quality-oriented reimbursement.

CONCLUSIONS:ATMPs represent a grey zone causing difficulties in classifying them either as method or drug. For individualized therapies evidence beyond RCTs and new reimbursement possibilities should be considered. Until new regulations are in place it is advisable to enter early into respective discussions with authorities.