Regulatory Focus™ > News Articles > EMA Wants to Clarify Process for Developing Gene- and Cellular-Based Products

EMA Wants to Clarify Process for Developing Gene- and Cellular-Based Products

Posted 14 February 2013 | By Alexander Gaffney, RAC

The European Medicines Agency (EMA) is touting the ongoing efforts of its Committee for Advanced Therapies (CAT), a panel of expert advisors that EMA said expects to review between three and four marketing authorization applications (MAAs) in 2013. Of particular interest to the committee in the coming year: clarifying for small businesses the process by which they can get scientific advice to support regulatory approvals for advanced therapies derived from gene- and cellular-based products.


The CAT is a relatively recent addition to EMA's arsenal of committees, having been established in 2009 under Regulation (EC) No 1394/2007 on advanced therapy medical products (ATMPs). The committee is comprised of five experts appointed by the Committee for Medicinal Products for Human Use (CHMP), one member from each EU member state, two clinicians and two people representing patient associations.

In April 2012, EMA published guidance on ATMPs-therapies made from genes and cells-which it said it hoped would provide a more stable pathway for sponsors looking to bring products to market.

In September 2012, EMA announced that the CAT was to assume more responsibility under a restructuring plan that would transform two of its working parties, the Cell-based Products Working Party (CPWP) and the Gene Therapy Working Party (GTWP), into ad hoc drafting groups.

The change was intended to free up review resources to allow the new groups to review products, leaving the drafting and reviewing of guidelines to the CAT itself. The groups will also be smaller, with only five standing experts instead of the current 10.

"This is intended to strengthen the role of the CAT as the reference body dealing with all aspects of the development of ATMPs in Europe," explained EMA at the time of the change.

Clarity for SMEs

Those changes are slated to bear fruit in 2013, EMA said in a statement. The CAT is expected to review as many as four ATMP MAAs in 2013, one more than it did in 2012, when it approved the first gene therapy product in the EU. Officials said they hoped that initial approval would clear the way for other complex medicines to be approved in the future.

EMA's 13 February 2012 statement on the committee's planned work in 2013 notes that its review output is limited due to the small number of MAAs submitted to the agency each year. That could change in the near future, however, as EMA said it was aware of a large and growing pipeline of ATMP products and research. "As a consequence, a high number of MAAs for advanced therapies is expected over the next five to 10 years," it wrote.

At least 75% of those ATMPs will be cell-based products, it said, while the other 25% will be gene therapy products. Of those products, 60% are being developed outside of industry-what EMA called "non-commercial sponsors," and 38% by small- and medium-sized enterprises (SMEs).

EMA said it is particularly interested in increasing awareness and utilization of its certification procedure among SMEs. The procedure is essentially an incentive for companies to develop ATMPs, allowing them to obtain certified scientific advice that allows the company to have greater assurances for the requirements of the review process.

Since 2009, EMA said it has only used the procedure twice with SMEs-a number it hopes to increase in the coming years by encouraging its use and providing clarity through a question-and-answer document. EMA said it conducted a survey that showed SME sponsors saw value in its use, but lacked understanding about its scope in relation to other regulatory procedures.


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