My Lords, I support Amendment 38, in the name of the noble Baroness, Lady Thornton, to which I have added my name. I shall also speak to Amendment 39. I am grateful to the noble Lord, Lord Hunt, who has already spoken, for lending his support. I have listened carefully, and I
support what has been said about the issues raised, particularly about whether we remain aligned with the EU trials mechanism or whether we are to be part of that mechanism.
During the EU withdrawal debate this issue was discussed at length. In fact, there was an earlier opportunity for the amendment to be put—as noble Lords may remember, it was widely supported—but I withdrew it, because the then Minister, the noble Lord, Lord Callanan, said that at an appropriate time, when legislation was brought in, the Government would address the issue. By that I supposed he meant that they would address the issue of remaining part of the EU clinical trials regime—but this Bill does not do that.
What options are available to the United Kingdom? One of them, of course, is to remain and participate in the EU clinical trials regime, if that is possible. An alternative is silent participation, as in the EEA model. That would mean that we could not vote, we could not lead projects and we could not raise objections. The third option is to be independent and aligned. The noble Lord, Lord Lansley, referred to that, and I agree with him that the important part of the EU clinical trials regime is its portal—a portal that the UK played a major part in developing—through which companies can apply for clinical trials.
The fourth option is to be independent and divergent: the UK would create a new clinical trials system. There is no time to do that by the end of 2020, but over time the UK could create a new system and build alliances. However, the risks need to be clearly understood, and balanced. Where will the companies go? Will they go where they have a bigger market, and a bigger opportunity, with larger numbers of patients for the trials, or will they conduct their trials in the United Kingdom?
There might be novel ways to approach this, and I understand that the MRHA is discussing and trying to develop a novel way of conducting clinical trials, which might be more attractive to companies. But of course, as we do not know what those are and we are not being told what they are, we cannot comment on them.
Currently, what looks like the best option is to be part of the EU clinical trials mechanism. With clinical trials for rare diseases, it is even more important for the UK to remain aligned with, or to be part of, the EU processes for rare diseases in relation to trials, to the data that will be available, and to medicines—for example, treatments developed for muscular dystrophy and metabolic disorders.
About 3.5 million people in the UK suffer at some point from one of the 7,000 or so rare diseases. The number for which treatment is available is small; hence the great need for collaborative research, data collection and the development of medicines, because a larger population is needed for clinical trials. Companies such as Silence Therapeutics, which the noble Baroness, Lady Thornton, mentioned, use gene silencing technologies for developing novel therapies for rare diseases. Others, such as Sarepta, use gene therapy for developing medicines. Companies such as Gilead Sciences are developing CAR T therapy; it was the first to
introduce CAR T therapy for cancers in the United Kingdom. All those companies have said that they would wish to remain in the United Kingdom to do their trials, if the environment was right.
The treatments that will utilise innovative techniques, such as gene silencing, are often used to treat rare diseases that affect a limited number of people, as I said. The number of patients with a rare disease in an individual country such as the UK is likely to be low by definition. However, for clinical trials to work, they require large numbers. Unified and streamlined international processes are essential to ensure that the application authorisation processes of these clinical trials can continue to work both effectively and at pace.
By implementing the clinical trials regulation, the UK can remain eligible for access to the central EU portals and processes for clinical trials, which ensure that clinical trials can recruit enough patients for rare diseases and include submissions, reporting and authorisation requirements and, particularly importantly, inclusion in patient registries. Those were developed as part of the EU-wide MHRA initiative to develop registries for rare diseases.
The UK should also seek to maintain alignment with patient safety and pharmacovigilance standards, as mentioned by the noble Baroness, Lady Thornton, to give patients and clinicians confidence in trials that are conducted in the UK and to support the UK’s ability to host trials that need to take place in multiple countries. Without this level of alignment, it is likely that clinical trials, particularly for innovative treatments such as gene silencing, will not be able to go ahead in the United Kingdom, denying UK patients access to new treatment options at an early stage.
I will end by saying a few words in support of Amendment 125 in the name of the noble Baroness, Lady Jolly. Given the global nature of the Human Medicines Regulations, the UK should be a member of the ICH—the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use. Having recently joined as an observer on Project Orbis and the Access Consortium, the UK can work towards providing a leadership role on global regulatory standards, and it is more likely to do that if it is part of the EU clinical trials mechanism.