The European Medicines Agency’s (EMA) Committee for Advanced Therapeutics (CAT) is implementing a five-year work plan it says should help academia and industry more easily access and understand the regulatory framework underpinning the development of advanced therapeutics including tissue engineered, cell-, and gene-based therapies.
As part of this work program CAT will liaise with SMEs, larger industrial players, and academia to identify areas where there is a lack of clarity and draft a program to address specific bottlenecks and hurdles. Ultimately CAT hopes to generate an environment that encourages the development of advanced-therapy medicinal products (ATMPs) and eases the route to market.
The EU is coming out of a transitional period that in 2011-1012 will mean all ATMPS will have to be centrally authorized, according to provisions of Article 29 of Regulation (EC)1394/2007, CAT explains. As a consequence of this, all products already legally on the EU market will have to be withdrawn if they fail to receive a marketing authorization. Moreover, stakeholders are now faced with a relatively new framework for ATMPS and the related regulatory and procedural guidance documents.
Emerging scientific fields have been identified as an important driver for progress and change by the EMA’s Road Map to 2015 (www.ema.europa.eu/docs/en_GB/document_library/Report/2010/01/WC500067952.pdf), CAT notes. However, it continues, the complex and financially draining regulatory environment represents a major hurdle in terms of progressing promising products such as gene- and cell-based therapies into late-stage development, particularly for SMEs and academia. The negative effects of such hurdles are evident by the very limited number of products heading toward a potential MAA over the next few years.
One of the ways CAT hopes to make the existing regulatory path more accessible to stakeholders is through the combination of easily accessible education and training to help academia and SMEs understand the process and requirements, together with a dedicated assistance program for applicants submitting ATMPs for regulatory review.
The Committee will also be looking into the possibility of extending incentives currently in place for SMEs, academia, trusts, and small research groups. It will in addition look at how best to apply the regulatory framework to tailor-made products falling under ATMP regulation, and how to optimize the "accelerated assessment procedure" for marketing authorization of ATMPs.
Increased dialogue between national and international regulatory bodies, their subcommittees, and the ATMP developers will also be encouraged, CAT stresses.