Written evidence submitted by the
Human Fertilisation and Embryology Authority (GEN0021)
1.1. Genomics and gene editing show real promise in the field of assisted reproduction and research involving human embryos. This is an area of research and clinical practice in which the UK excels, and is an element of our vibrant life sciences sector. The existence of a statutory regulator in this field, the HFEA, has greatly helped public acceptance of this contested area of medicine and the regulatory regime has provided a clear set of rules that has allowed innovation to flourish. The evidence strongly suggests that policy making with open public engagement at its heart has been vital to that success.
2.1. The UK has a strong tradition of innovation in the field of assisted reproduction and research involving human embryos. The first ‘test-tube’ baby, Louise Brown, was born in the UK in 1978. The UK was also the first country to establish a dedicated regulator of IVF and embryo research. We started regulating the sector on 1 August 1991.
2.2. The Human Fertilisation and Embryology (HFE) Act 1990 (the Act) was a landmark piece of legislation; the culmination of almost a decade of expert and public debate about assisted reproduction. It has stood the test of time remarkably well, with only one significant legislative amendment since it was enacted. One reason for this longevity is the system of regulation established by the Act; one which established a regulator with policy-making and licensing powers. By law, anyone wishing to provide assisted reproduction services or undertake research involving human embryos in the UK can only do so under a licence from the HFEA.
3.1. Our regulatory remit relates to a narrow part of your enquiry - that which involves the procurement of gametes and use of human embryos in genomics and gene editing research. However, we hope that our submission will provide the Committee with useful information on the following three areas:
4.1. In view of the controversial nature of this aspect of clinical practice and research, the Act sets out a regulatory framework in which clinics and laboratories can operate with relatively few prohibitions but, crucially, close oversight by the regulator. This approach in the legislation represents, in effect, the bargain between science and society which Parliament has decided is appropriate in this area of medicine.
Licensing of genome editing in human embryos
4.2. Research on human embryos has been a central component of the UK regulated landscape since the passing of the Act in 1990. The Act sets out strict conditions under which such research can take place, providing reassurance to the public, some of whom hold principled moral objections to any such research. It is easy to underestimate the success of the regulatory system in promoting research innovation in such a contested area of medicine.
4.3. Many leading edge developments have taken place in the UK, right up to the present day when in 2015 – following extensive public dialogue led by the HFEA - the UK Parliament made lawful for the first time in the World treatments which could avoid the inheritance of serious mitochondrial diseases. And in February 2016 we licensed the Francis Crick Institute in London to undertake research involving the new gene editing technique CRISPR-Cas9 in human embryos for the first time in a regulated environment.
Limitations of genome editing in human embryos
4.4. Although the regulatory approval of this new technique is ground breaking, it is important to bear in mind that there is only one group currently carrying out research on genome editing using human embryos in the UK – so the scale of this type of research is still very small. It is also important to note the limitations on bringing such research into a clinical setting. The Act prevents the clinical use of any techniques that involves genetic modification of gametes and embryos to treat patients (apart from techniques to prevent the transmission of serious mitochondrial disease). This means that Parliament would need to amend current legislation before genome editing on human embryos could be used for clinical treatment purposes.
4.5. Any decision to allow such techniques in clinical treatment, however circumscribed, would represent a very significant development and we would highlight the importance of effective public engagement to explore the ethical, legal, and societal issues beforehand. We draw upon our experience of public engagement ahead of the changes to the law permitting mitochondrial donation in Section 6 of our submission.
Innovations in the field of genomics and gene editing
4.6. These ground-breaking developments have been able to happen because of the legal and regulatory framework in the UK. The Act provides a stable, yet flexible, framework in which UK bio-science and clinical expertise can flourish. Scientists and clinicians have been able to go about their work free of the ‘culture wars’ that has hampered such activity in the USA or the regulatory free-for-all of much of the Far East.
5.1. An increasing focus of modern medicine is the use of genomics information to benefit public health (public health genomics). This has led to more effective, personalised treatments with better specificity, targeted to the genetic makeup of each patient.
5.2. This approach has limited applicability to reproductive medicine, in which the focus is on genetic testing technology, either to avoid inheritance of genetic disease or to avoid repeated miscarriage. The Act sets out a regulatory framework for embryo testing, by either pre-implantation genetic diagnosis (PGD) or pre-implantation genetic screening (PGS). We license both the clinics offering embryo testing and the circumstances in which it can be used.
5.3. The Act sets out strict conditions that must be met before PGD can be carried out for a heritable condition. There must be a particular risk that the embryo to be tested may have a genetic, mitochondrial or chromosomal abnormality, and that a person with the abnormality will have or develop a serious disability, illness or medical condition. PGD is not allowed for sex selection except where there is also a particular risk that any resulting child will have or develop a gender related serious disability, illness or medical condition. PGS may be used to screen for a range of chromosomal abnormalities which might affect an embryo’s capacity to result in a live birth. Whilst there is no specific authorisation process for the use of PGS in individual cases; clinics which are licensed for embryo testing validate the use of PGS for each category of patients to which it is offered.
5.4. Embryo testing has developed rapidly in the UK - to date, the HFEA has licensed PGD for around 400 serious conditions, providing new treatment options for many desperate families. Developments in testing technology are only likely to increase the number of conditions available for treatment and in the last business year alone, the HFEA has licensed about 50 new conditions, many of them very rare. The licensing process is both rigorous and relatively quick, offering reassurance to the public and reducing the time that anxious patients have to wait for new treatment options.
New technologies in genetic testing
5.5. There have been a number of developments in genetic testing, particularly in the methods used for testing, which are now cheaper, more accurate and can identify more than one mutation at a time. It is possible, therefore, that with advances in technology, embryo testing could be extended to a wider audience. Alongside this, the growing use of genome sequencing amongst the general population may lead a person to seek reproductive treatment to avoid passing on a disease, identified through their personalised genome data, to their unborn child. No longer would that person need to have had an affected child or relative in advance for them to be aware of their significant risk of disease transmission.
5.6. Looking ahead, it is possible that genetic testing might be viewed as a distinct component of a wider preventative health agenda. However, at present the strict conditions set out in the Act prevents the widespread use of PGD (for example, it is not possible to test an embryo for a genetic condition unless there is a known significant risk that the embryo could be affected; and that the condition will lead to a serious disability, illness or medical condition), and Parliament would need to amend the legislation if change along these lines was thought to be desirable.
Monitoring research and horizon scanning
5.7. Scientific developments in this field move at a fast pace. Our Scientific and Clinical Advances Advisory Committee frequently consider scientific developments which may impact upon clinical practice. This includes consideration of short term and long term developments in research.
5.8. For example, subject to scientific developments and a change in the law, genome editing of human embryos could be used to prevent inheritance of serious genetic conditions. Further in the future, it may be possible to carry out genome editing in somatic cells (eg, cells derived from an adult skin cell) and then use those cells to create gametes (ie, in vitro-derived gametes). In theory, this would remove the need to carry out genome editing in embryos because the editing would already have taken place before the gametes were created. Another potential future application of genome editing could be for refinement of other techniques, such as mitochondrial donation. Although potentially decades away, we will continually monitor advances like these as they happen and consider any scientific, ethical, legal or societal impacts. We will also continue to actively carry out long term follow up on techniques which have already been permitted in clinical practice, in order to assess their safety and efficacy.
6.1. The HFEA is a public body with substantial expertise in public dialogue and consultation often on contentious ethical and scientific issues. That experience is central to our success as a regulator and policy maker in such difficult areas. The importance of effective public engagement when it comes to advancements in complex medical science can be illustrated in our work on mitochondrial donation.
Public engagement on mitochondrial donation
6.2. In October 2015, mitochondrial donation became legal in the UK. The technique was developed in Newcastle and in the USA and allows the donation of healthy mitochondria from a donor egg or embryo to a woman whose mitochondria carries a serious inherited disease. Such diseases are often fatal or life-limiting and until now there were no treatment options available that allowed such women to have a genetically related child. The HFE Act 2008 was amended to allow the possibility of regulations to allow such treatments, and after extensive consideration of the safety and efficacy of these treatments and a consultation with the public about the acceptability of the proposed treatments, Parliament approved a change in the law – a World first.
6.3. This ground-breaking step taken by Parliament was in a large part a result of four years of work carried out by the HFEA – both in terms of assessing the safety and efficacy of the techniques and extensive public engagement to seek the public’s view. The latter took many forms, including a public consultation to gather views on the social and ethical impact of making these techniques available to patients; and a multi-method research and engagement project, using deliberative workshops with members of the public, a representative survey, public meetings and a focus group with patients.
6.4. Taken together, the evidence allowed us to identify the process of deliberation people used to form views on mitochondria donation, the differences between informed and uninformed public views on these techniques, interested stakeholders’ arguments for and against the use of the techniques, and an analysis of the ethical and regulatory issues involved.
6.5. Without such public engagement, Parliament would have found the task of changing legislation much more difficult - with the increased potential for more opposition from politicians, the media and public, as well as the increased likelihood of misleading and inaccurate information to surface. And without the existence of the HFEA, as a well-respected regulator, Parliament would not have felt sufficiently reassured that mitochondrial donation would in fact be used in the circumstances they had envisaged when making it lawful.
January 2017