Advanced therapies are ready to take centre stage: Academia's involvement with regulation needs to raise its game

Abstract

Several CAR-T cells-based therapies have been approved for the treatment of blood cancers. 1 Gene therapies are constantly maturing and are now targeting not only rare diseases but also more common ailments such as beta thalassemia. Although toxicity issues are by no means minor, 2 it marks the beginning of an era where gene therapies will become widely applied.Clinical trials for advanced therapies (ATMPs, which comprise genetic and somatic cells therapies and tissue engineered products) have grown at a rapid pace, with a dramatic global increase in the number of clinical trials, but much less so in Europe than in other

The British Journal of Clinical Pharmacology: The first 50 years

“…[17][18][19][20] Pharmacologists were initially slow to embrace high molecular weight drugs, but there was a satisfying increase in research papers on biopharmaceuticals during my tenure, and papers on the human pharmacology of these agents, especially RNA drugs, with their potential to provide cures for many previously untreatable diseases, have continued to grace our pages in increasing numbers. [21][22][23][24][25] This is perhaps the hottest current research area in our field, but I am pleased too that we also continue to publish studies that improve the use of older drugs, such as allopurinol. 26 Getting the right balance between readable peer-reviewed research articles and systematic reviews and meta-analyses on the one hand, and less formal reviews, editorials, and other material of general interest and educational value was a constant topic of discussion between me and the other senior editors (International Editors plus Reviews Editor), with input from the Society and the publishers.…”

“…Pharmacologists were initially slow to embrace high molecular weight drugs, but there was a satisfying increase in research papers on biopharmaceuticals during my tenure, and papers on the human pharmacology of these agents, especially RNA drugs, with their potential to provide cures for many previously untreatable diseases, have continued to grace our pages in increasing numbers 21–25 . This is perhaps the hottest current research area in our field, but I am pleased too that we also continue to publish studies that improve the use of older drugs, such as allopurinol 26 …”

“…And it is what I intend to do until I pass the baton to my successor. During my tenure we have been making inroads into newer areas, such as Equity in Precision Medicine,43,44 Advanced Therapeutic Medicinal Products,22 Climate Change Pharmacology,45 and Artificial Intelligence,[46][47][48] and I expect the next EiC to lead us further into these interesting new areas, as well as areas that we have not even thought of. I also hope that the next EiC will continue the Issue Highlights, and that there will be less need to address epidemics, or the atrocities of armed conflicts, or other disasters.…”

Pharmaceuticals and Medical Devices Agency's horizon scanning and the Science Board: Cooperation toward extracellular vesicle‐based products

“…We read with interest the recent article by Hidalgo-Simon and Fibbe, 1 who proposed academia's involvement with regulation for promoting advanced therapy development. We appreciated the authors' insights into the fields and would like to share our experience in Japan that deserves readers' attention.…”

Challenging times: Delivering gene therapies and an opportunity for shared learning

“…The regulation surrounding ATMPs classified as GMOs has been identified as an issue for ATMP adoption in Europe. 8 Thankfully, the EMA now provides guidance on a streamlined approach to assess the environment risk of medicines containing GMOs. To meet the EPAs conditions, Zolgensma ® required additional specific protocols to detail processes for its receipt, handling, storage, internal transport and disposal.…”