Europe – Europe urged to adapt faster to gene therapy challenges

Europe’s leading nations should commit to a rapid overhaul of health technology assessment (HTA) and bold initiatives such as new pan-European real world evidence (RWE) frameworks, in order to nurture the nascent cell and gene therapy revolution in the region.

That is the conclusion of a new report from The Alliance for Regenerative Medicine (ARM), the international advocacy organisation representing cell and gene therapy companies and the broader field of advanced therapy medicinal products (ATMPs).

ARM has more than 70 members across 15 countries in Europe, and is working closely with European stakeholders, looking to create a leading commercial and regulatory environment for ATMPs in the region.

The last 12 months have seen the European approval and launch of groundbreaking therapies in the field, most notably the first two CAR-T therapies, Novartis’ Kymriah and Gilead/Kite’s Yescarta, plus Spark/Roche’s gene therapy Luxturna, and most recently, the approval of Bluebird’s gene therapy Zynteglo.

Investment has been flooding into the cell and gene therapy pipeline in that same period, despite the fact that the commercial model for these therapies is far from established…