The European Medicines Agency set up the (CAT) in 2009. Its main tasks are to:
- review marketing-authorisation applications (MAAs) for (ATMPs);
- provide support to the Agency’s Scientific Advice Working Party for all scientific advice for ATMPs;
- provide scientific recommendations on the classification of medicines as ATMP;
- evaluate early quality and non-clinical data in the framework of an ATMP certification.
In 2013, the CAT expects to review three to four MAAs for ATMPs. This compares to three initial applications for MAA received in 2012, which led to the authorisation of the first gene therapy in Europe for the treatment of lipoprotein lipase (LPL) deficiency, a very rare inherited disorder. “With the approval of the first gene therapy in 2012, the CAT is paving the way for the approval of similarly complex medicines in the future, as more gene therapies for rare diseases, personalised medicines and nanomedicines are on their way”, explains Christian Schneider, Chair of the CAT.
While the number of MAAs for advanced therapies is still limited, the research and development pipeline is large, as shown by an analysis of ATMPs under clinical evaluation published in 2012 in the journal Molecular Therapy. This is confirmed by the number of scientific advice provided to companies by the CAT and the number of classified ATMPs. As a consequence, a high number of MAAs for advanced therapies is expected over the next five to 10 years.
Among the ATMPs under development, three quarters are cell-based medicinal products while one quarter represents gene therapies. Mostly these products are being developed for cancers, cardiovascular diseases and haematology-related conditions. Also of interest, ATMPs are being developed mainly by non-commercial sponsors (60%) and micro, small and medium-sized enterprises (SMEs; 38%)
Clarification on advanced-therapy classification
The CAT delivers scientific recommendations on ATMP classification. In December 2012, the Committee published a reflection paper that clarifies the legal basis for the classification of medicines as advanced therapies and provides information on how these medicines are classified as gene-therapy, somatic-cell-therapy, tissue-engineered or combined medicines. Some borderline cases and areas where scientific knowledge is limited or evolving rapidly are also discussed.
Because ATMPs are complex and science in this field is developing rapidly, there are frequently questions on whether or not a product can be classified as an ATMP, as these products are often at the borderline between medicinal products and other products such as medical devices or cell and tissue transplants. The classification procedure gives companies developing innovative new treatments the opportunity to check whether the products they are developing can be considered as advanced therapy medicinal products, and can therefore benefit from the regulatory pathway and the available scientific guidelines for these types of medicine. This procedure also often constitutes the first contact between the Agency and developers of ATMPs.
In 2012, 17 applications were submitted for a scientific recommendation on advanced-therapy classification. The CAT classified 14 of these as ATMPs, compared with the 12 submitted and 12 adopted in 2011. A similar number is expected in 2013.
Raising awareness of certification procedure for SMEs
“Apart from non-commercial sponsors, SMEs are the main developers of ATMPs. In the current financial and economic environment, many companies would not take the risk of having this kind of product in their pipeline, for which there is limited regulatory experience. We thus need to incentivise the development of these potentially beneficial innovative treatments,” says Christian Schneider.
In 2013, the CAT wants to raise awareness of the certification procedure which offers an incentive for SMEs developing ATMPs. In the certification procedure, the CAT carries out a scientific evaluation of quality data and, when available, non-clinical data on ATMPs. Following this, the Committee may recommend issuing a certificate confirming to what extent the data generated so far comply with the review standards that would be applied for the evaluation of an MAA. The aim of this procedure is to facilitate dialogue with the regulatory authorities early in drug development. In addition, the assurance provided by the certification may help SMEs to attract investors and to raise more capital for the development. “It is the only regulatory procedure that actually provides a scientific assessment prior to a marketing-authorisation application,” says Christian Schneider.
As the Agency has only received a total of two applications for certification since 2009, the CAT conducted a survey among SMEs developing ATMPs and their stakeholders in 2012 to find out why the certification procedure is not more widely used by applicants. The outcome of the survey reveals that the certification procedure is perceived to be a valuable incentive from SMEs developing ATMPs with a majority of companies indicating that they might consider applying at a certain point during the development of the product. However, the understanding of the scope of the certification procedure vis-à-vis other regulatory procedures emerged as a potential issue.
Based on the outcome of this survey, the CAT is planning to conduct initiatives to encourage a greater use of this procedure. In particular, the scope of this certification will be explained in a question-and-answer document. The Agency will further clarify the place of certification within the range of services provided by the Agency to support SMEs. The Agency will also bring the results from this survey to the attention of the European Commission.
Strengthening interactions with stakeholders
As part of its work programme 2010-2015, this year the CAT will continue to hold meetings on specific topics with interested parties such as scientific associations or trade organisations. These will be similar to the focus group on non-clinical development of ATMPs, which took place in October 2012.
On 23 October 2013, a collaborative workshop will be held by the CAT and the Translation Centre for Regenerative Medicines (TRM) in Leipzig called .