Genetic Alliance UK - Views & Policy:

Regulation

Explanation of What Interest We Have and What Work We Do:

Research, treatment development [link to treatment development page] and care provision all require proportionate and rational regulation. Just as we involve ourselves in promoting and facilitating research, we believe it is important that the voice of patients affected by inherited conditions is heard in the regulatory arena.

Here we describe Regulatory Authorities relevant to Genetic Alliance UK and our Members.

Click the links below to reveal/hide more about our views and policies on the following:

Regulation of Gene Therapy, Tissue Therapy and Cell Therapy
in Europe

Since the passing of the Advanced Medicinal Therapies Regulation in 2007, these cutting edge therapies have been regulated by the Committee for Advanced Therapies (CAT) at the European Medicines Agency (EMA). As you can read here in the Policy Archive 2007, Genetic Alliance UK campaigned strongly for this regulation to pass, as we believe these therapies are best regulated by experts, that the European market for these treatments would benefit from a united regulatory framework, and because the legislation provides many incentives for small organisations to bring advanced therapies to market.

In line with the other recently founded committees of the EMA, patient representation on the CAT is required by the regulation. There are two patient members on the CAT, and two alternate members. Genetic Alliance UK’s Director, Alastair Kent, is one of the members, and our Policy Analyst, Nick Meade is one of the alternate members. The other two patient representatives on the committee are from Eurordis.

Patient representatives are full members of the committee and contribute just as other members of the committee (composed mainly of representatives of EU national medicine regulatory agencies such as the UK’s MHRA, see below) but patient members are expected to involve patients and patient groups with the committee while bringing extra expertise to the committee and assisting with the committee’s understanding of those directly affected by regulatory decisions.

You can find out the UK’s MHRA links with the CAT and the EMA in this area here.

Medicines and Healthcare Products Regulatory Agency (MHRA)

The Medicines and Healthcare products Regulatory Agency (MHRA) is the UK regulator responsible for medicines and medical devices, like other regulators relevant to Genetic Alliance UK, the MHRA is an executive agency of the Department of Health.

The MHRA regulate some advanced medicinal products in the UK if they are exempt from the European regulation.

Human Tissue Authority (HTA)

The Human Tissue Authority (HTA) is the UK regulator responsible the use and storage of human tissue for research, and teaching. The HTA also regulates some cell therapies in the UK, when the therapy is exempt from the European regulation, and the cells being used as a therapy are the patient’s own (an autologous cell therapy).

Alongside regulation of autologous cell therapies, Genetic Alliance UK is interested in the regulation of the storage of cord blood for transplant. This therapy is important for treating some life-limiting blood disorders and has potential to treat a wider range of conditions. Genetic Alliance UK has produced a leaflet explaining the issues surround cord blood collection and use, and the HTA has some leaflets describing the law surrounding the collection and storage of cord blood here.

Human Fertilisation and Embryology Authority (HFEA)

The Human Fertilisation and Embryology Authority (HFEA) is the UK regulator responsible for regulating the delivery of treatments using human eggs and sperm, and treatment and research involving human embryos.

As you can see within the Policy Research section, Genetic Alliance UK is a strong supporter of research into the potential of therapies involving the human embryo, and campaigned hard (see: Policy Archive 2008) for the laws in the UK to permit research whilst keeping it well regulated.

The HFEA also regulate the provision and providers of Preimplantation Genetic Diagnosis (PGD) and Preimplantation Tissue Typing (PTT) [link to PGD and PTT section on Diagnosis and Screening page]. PGD and PTT can only be offered to couples if the HFEA has granted a licence for the condition to be screened for before implantation. For PGD, once the condition has been considered by the HFEA’s licensing committee, any couple can use PGD to screen for the condition they could pass on to their child. For PTT, the HFEA considers each case individually.

You can see the list of conditions that have been licensed here and you can see the list of conditions awaiting approval here; Genetic Alliance UK submits its opinion during the licensing process for most of the conditions considered for licensing.

The HFEA also has more information on where, when, and how you can access PGD and PTT on its website.