Monitoring outcomes in patients treated by ATMPs

In a recent Editorial in HemaSphere, an important aspect of advanced therapy medicinal products (ATMPs) produced and used in therapy under the hospital exemption (HE) status is raised by Domanovic et al.1 The European Blood Alliance, together with scientific societies and professional organizations, strongly supports maintaining and strengthening the HE scheme provided that specific criteria are approved by Regulatory Authorities, European and National. As the authors mark, a working system for monitoring outcomes in patients treated with ATMP/HE is lacking in the EU with public reporting, despite the clinical importance of this therapeutic modality. However, we also note that although important scientific and technological advancements were experienced since the establishment of ARMP Regulation in the European Union,2 the scientific community in general is not yet in a position to evaluate the whole results of chimeric antigen receptor T (CAR-T) cell therapies as applied with the commercial products available in the EU. This is mainly due to the lack of any active system gathering cumulative data regarding the short- and long-term outcomes of ATMPs. The data individually kept by each medical center or by marketed CAR-T cell suppliers cannot provide a global insight to this mode of treatment. Besides several parameters involved in evaluating the results (population group applied, disease, age, sex, management history, comorbidities, etc.) before a final argument on the efficacy of this treatment is achieved, the variability of commercial products has also to be accounted for. Different particular products produced by different protocols may also turn to have different therapeutic effects or untoward effects. Therefore, treatment trials with particular commercial products may be non-comparable as far as the outcome is concerned. It is only a cumulative analysis that can permit such a conclusion. It seems that it is now the proper time for establishing a monitoring system for the outcomes in cases treated with ATMPs/CAR-T cell approved centrally by commercial products or via the HE mode in Europe. The authorization of these products was in principle a “conditional” one; the regulatory authorities and scientists must insist on consistent quality, safety, and efficacy standards. Kostas Konstantopoulos: Conceptualization; writing—original draft; writing—review and editing. The author declares no conflicts of interest. This research received no funding. The data that support the findings of this study are available on request from the corresponding author. The data are not publicly available due to privacy or ethical restrictions.