Table 2.

Mutual challenges and possible next steps for ATMPs.

Translational aspects	Challenge	Possible next steps
PoC/MoA	Absence of relevant animal model	Call for generic studies, e.g., for H2030?
PK/PD	Limited to predominantly distribution studies	Comprehensive approaches should be developed e.g., via BMQ?
Dose translation	Alloscaling absent	Systematic generic analyses needed?
Chronic SD/MD toxicity	Exemplar RBA cases are limited	Periodic updates? Encourage public development of relevant toxicity animal models?
GLP environment	Not always followed	New procedure at NCA/EMA SA level?

BMQ, Biomarker qualification; H2030, Horizon 2030; MD, multiple dose; MoA, Mode of Action, PD, Pharmacodynamics; PK, Pharmacokinetics; PoC, Proof-of-Concept; RBA, Risk Based Approach; SD, single dose.