Regulatory Landscape

The regulatory environment for stem cell research is evolving and is governed in the US by the Federal Drug Administration (FDA), in Europe by the European Medicines Agency (EMA) and in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA).

In the UK, the MHRA has published the Interim UK Regulatory Route Map for Stem Cell Research and Manufacture which provides guidance and a flowchart through the legislation, whilst the Department of Health has published the new UK stem cell toolkit, which provides a list of questions to guide you through the legislation for your specific application. These documents provide valuable information on the regulations in the UK and Europe.

SSCN has produced an overview of the various requirements applicable to:

Regulators - what will happen next?

It was announced at the end of July that following the Government's review of arm's length bodies, there will be a reduction in the number of the health regulatory bodies. The aim is to cut costs and reduce duplication so that the work done is by means of the most efficient use of resource. In terms of the impact on those working in the regenerative medicine field, the relevant regulatory bodies include the HFEA (Human Fertilisation and Embryology Authority), the HTA (Human Tissue Authority), the HPA (Health Protection Agency) and the MHRA (Medicines and Healthcare products Regulatory Agency).

One role of the HFEA is in relation to human embryo storage and research including the derivation of human embryonic stem cell lines (hESCs). However, the HFEA's remit stops at the point at which the hESC lines are generated and (as a condition of a licence to conduct such research) deposited in the UK Stem Cell Bank.

The HTA regulates the removal, storage and use of human tissue, including stem cell lines where they are intended for use in human clinical application such as a medicinal product, the HTA also governs the donation, procurement and testing of those cells to ensure that they are in compliance with the EU Tissues and Cells Directive.

The MHRA regulates medical devices and medicines which are to be brought to market. Included in the MHRA's remit are products and therapies derived from tissue engineering whether classified as an Advanced Medicinal Therapy, or not.

The HPA is relevant in this context primarily through the fact that the National Institute of Biological Standards and Control (NIBSC) merged in to the HPA on the 1 April last year. NIBSC hosts the UK Stem Cell Bank which acts as a depository for human stem cell lines of all types, but in particular hESCs derived under licence from the HFEA.

The proposal from the Government's review is no make no change to the MHRA, abolish the HPA but to retain both the HFEA and the HTA for the time being. The functions of the HPA will transfer to the new Public Health Service in due course and the functions of the HFEA and HTA will in time be transferred by the end of this current parliament. One of the key questions is what entity will assume those functions - it is not yet clear whether it will it be to a new regulator, the Care Quality Commission and/or the Health and Social Care Information Centre.

There has been a mixed reaction to this initial government review. There have been proposals before to merge at least the remit of the HFEA and HTA into one regulator. During the pre-legislative review of the Human Tissue and Embryos draft Bill 2007, the Joint Committee made the recommendation against merging the HFEA and HTA into a single authority - RATE (the Regulatory Authority for Tissue and Embryos). There was concern at the time that a combined body would lack the necessary expertise for either regulatory function. However, many thought that this concern could be addressed by way of specialist sub-committees. The benefit for those working in the regenerative medicine field, especially those working with hESCs, could be simplified interaction with a single regulator that would be responsible for the initial licence for research, through to compliance with requirements for cells that are to be used in human clinical application and the onward interaction with the MHRA in taking that to market.

However, there is time for much debate on the pros and cons of who will assume responsibility for the roles that the HFEA and HTA play and how they will inter-relate. Any change to the current set up would need to be by way of primary legislation amending both the Human Embryology and Fertilisation Act 1990 and the Human Tissue Act 1994 which established the HFEA and HTA respectively. However, this could be seen as an opportunity to streamline some of the conflicts or overlap that are perceived to be the case in dealing with the HFEA and HTA as two distinct regulators, particularly from the perspective of a hESC line that has commercial applications as a medicinal product.

Philippa Montgomerie
DLA Piper Scotland LLP
August 2010