Barriers for the evaluation of advanced therapy medicines and their translation to clinical practice: Umbrella review
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Show full item recordEditorial
Elsevier
Materia
Advanced therapies medicinal products Gene therapy Cell therapy Tissue engineering Barriers Development cycle
Date
2022-10-14Referencia bibliográfica
Lorena Aguilera-Cobos... [et al.]. Barriers for the evaluation of advanced therapy medicines and their translation to clinical practice: Umbrella review, Health Policy, Volume 126, Issue 12, 2022, Pages 1248-1255, ISSN 0168-8510, [https://doi.org/10.1016/j.healthpol.2022.10.007]
Sponsorship
Spanish Ministry of Science and Innovation/National Research Agency PID2019.105597RA.I00 MCIN/AEI/10.13039/501100011033Abstract
Advanced therapy medicinal products (ATMPs) are a fast-growing field of medicine with wide potential application.
Nevertheless, so far, only 19 have obtained European Union (EU) marketing authorisation and only 13 of
these have translated successfully into clinical practice. This study conducts an umbrella review to identify the
main barriers for the evaluation of ATMPs and their translation into clinical practice across the development
lifecycle. 71 systematic reviews were included, of which 50 dealt primarily with effectiveness and safety, 13 with
translation from pre-clinical to human subjects. Others dealt with economic issues and translation from health
technology assessment to market access. The literature highlights the importance of synergistic research groups
or networks that collaborate across the in-vitro science, preclinical and clinical investigation phases, and the role
of private investor capital and public-private collaborations. Most ATMPs reviewed seem to have a favourable
safety profile although considerable uncertainties remain. Randomised controlled trials are not always feasible in
these patient groups. Greater sharing of data is recommended, both at preclinical and post-marketing real world
evidence. There are considerable variations between EU countries in how they regulate hospital exemption for
ATMPs, and this can lead to inequitable access for patients.