On 16 December 2016, the European Medicines Agency (EMA), together with the European Biopharmaceutical Enterprises (EBE) held the fifth annual “Optimizing the development of ATMPs to meet patient needs” regulatory conference on the optimization of the development of advanced therapy medicinal products (ATMPs) to meet patient needs in London, UK.
The conference featured a group of international pharmaceutical and regulatory experts from, among others, Novartis, Amgen, GSK and the FDA. Discussions focused around initiatives to improve the access to ATMPs for patients, as well as outlined the needs for their development and commercialization within the EU. Among them, delegates from academia and the public were also welcome to attend.
Earlier in 2016, the EMA identified four key areas of importance for the development of ATMPs:
- support research and development;
- optimize regulatory processes for ATMPs
- shift from hospital exemption to marketing authorisation
- improve funding, investment and patient access
At the December conference, one of the areas identified as being of critical area of concern is the enhancement of manufacturing processes and logistics, including cryopreservation, to catch up with the progress of scientific discoveries.
Presentations from Steven Kili and Steven Howe at GSK highlighted the need for optimization in cost of goods and manufacturing models – decentralized vs centralized – to improve access of therapies to patients. These considerations were mirrored by Dr. Ke Liu at the FDA who discussed the FDA’s Breakthrough Therapy Designation program, established in 2012, which grants early access to drugs that show preliminary clinical benefit. This program is critical to manufacturing entities as it engages CMOs at early clinical stages, and has increased in recent years with more gene and cell therapies being accelerated.
The EMA’s Ana Hidalgo-Simon summarized discussions at the conference by outlining an action plan which includes an opinion paper on a plan with actions to improve the regulatory and scientific environment for ATMPs under development, to come in early 2017.
At the conference, an update was also provided on the quality, non-clinical and clinical aspects of gene therapy medicinal products by Christiane Niederlaender at the Medicines and Pharmaceutical Products Regulatory Agency. The main changes included new considerations on:
- updates in development genetics, production, purification, characterization, quality control and comparability in the quality aspects of gene therapy medicinal products
- general considerations as well as specific considerations in quality, non-clinical and clinical aspects of specific classes of gene therapy medicinal products