The ATMP Regulation which regulates, inter alia, gene therapies, tissues and cell engineering therapies and was originally designed to encourage the production and acceptance of these therapies, but has been criticised for actually hindering its own goal. This consultation can be seen as a first step towards a new, or amending Regulation on advanced therapies
The consultation responses can be said to adequately reflect the views of industry and research stakeholders, who have identified a number of items of concern in the consultation which ran from 20 December 2012 – 31 March 2013 and was part of the five year review of the functioning of the Regulation.
Functioning of the regulation:
- The ATMP Regulation was considered an important framework for ATMP development.
- 1 in 4 contributors had a negative view of the Regulation, which was thought to be overly burdensome and to contribute to discourage innovation and product development.
- The ATMP Regulation was criticised for not taking into account the capabilities of SME’s causing an overuse of the hospital exemption.
- Almost all contributors recommended a revision of the ATMP Regulation to address its problems.
- The definition of active substances was deemed to be two rigid.
Combined ATMPs and Medical Devices:
- The Major concern related to the regulation for combined ATMPs is that CE mark requirement ads an unnecessary burden to development.
- Some stakeholders indicated that the validation of the device part was not appropriately carried out by the EMA.
- Stakeholders called for the introduction of more flexibility for assessing ATMPs. This is particular relevant for Rare Disease, where industry is calling for similar flexibility.
- Almost all stakeholders expressed the view that quality requirements should be adapted to accept the variability of ATMPS materials etc.
- The hospital exemption was regarded by all as an important tool to meet unmet medical needs and to stimulate innovation.
- Industry indicated that hospital exemption should not be permitted where an authorised product is available.
- Stakeholders requested that clearer definitions of quality requirements be introduced for the hospital exemption.
Other topics addressed by the consultation included:
- Incentives for the development of advanced therapy medicinal products
- Scope of the regulation and adaptation to technological progress
On the basis of the responses to the consultation, The European Commission will draw up a report, which is expected to be published before the end of this year. The report will determine whether the Commission will propose to revise the existing ATMP Regulation.
A summary of the responses to the consultation is available here and the individual responses are available here.