I should explain that the noble Baroness, Lady Finlay, is required in the main Chamber to speak in a debate on the Bill there. I have put my name to her amendments. Amendment 26 and others in this group would ensure the development of a rapid provisional two-year licensing procedure, so that patients might more quickly access potentially life-saving medicines and medical devices, and that device trial results were consistently registered and published.
In earlier amendments, several noble Lords commented on the avoidable delays in innovation, and the tardy response of the NHS to new and improved medical devices. The current licensing procedure in the UK can be lengthy. Safety and efficacy of course are paramount, but in our debate on Monday, my noble friend Lord Blunkett referred to my late noble friend Lady Jowell and her powerful call for fast-tracking. Many patients wanting to trial novel therapies say, “It may not help me, but it may help others.”
As the Minister said at Second Reading, and all speakers on the second group of amendments last Monday, particularly the noble Lord, Lord Sharkey, we must remain an attractive place to develop new medicines and devices. Amendment 26 supports that aim through the development of a licensing procedure that would speed up making new medicines accessible to the NHS when clinical trials have shown them beneficial and safe for people with the relevant conditions.
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For medicines to gain market authorisation in the UK, the MHRA follows the International Council for Harmonisation. It requires that the drug shows overall benefit for the patient’s condition with any undesirable effects documented. This means that the drug has usually been through several so-called phase 3 clinical trials comparing the active drug against a placebo in a relatively large number of patients. The process of testing drugs and then applying for a licence can sometimes take up more than a decade, even though there is a point at which the drug has been sufficiently tested in large cohorts of patients where the active treatment is compared with a placebo using randomisation and double-blinded to avoid bias. Ideally, once that phase has been completed, the next process involved in the review and acceptance for market authorisation should take only a limited period; it should not be lengthy.
The MHRA is much quicker than it was a decade ago, but there can still be long delays. Luxturna started development in 2007 to treat inherited blindness caused by RPE65 gene mutation in the retina. It was approved by the FDA in the US in December 2017, by the European Medicines Agency in 2018 and finally, this year, as a NICE-approved gene therapy in the UK for patients at four specialised ophthalmology centres. At this stage, I ought to declare my trusteeship of the Royal College of Ophthalmologists.
This rare condition has led to a few hundred children and young people in the UK being registered blind by their teens. There was no approved treatment prior to Luxturna being developed, and the first clinical studies showed that the treatment corrected the gene defect and improved the sight of those treated. But it took nearly five years to complete the additional studies required by the regulatory legislation. A provisional two-year licensing procedure would have allowed patients to access the treatment much earlier, while still allowing additional data to be generated, particularly in relation to ongoing safety and duration of efficacy. There are other examples where a more rapid procedure would be hugely beneficial. During the Covid-19 pandemic, the MHRA and other regulatory agencies have rightly fast-tracked new and promising medicines for reviews and approvals before beginning a phase 3 study.
My noble friend’s amendment would build on that speed by creating a rapid two-year provisional licence without reducing safeguards around the new medicine. As she said at Second Reading, the Bill is an opportunity to make the UK a major player in advancing new medicines for the world market. She hopes that noble Lords will see this amendment as a key step in achieving that aim. She makes it clear that there should be no compromise on safety; safety requirements would still be met for the MHRA to issue a provisional two-year licence, and the two-year process should be used to ensure that safety monitoring occurs. Manufacturers could apply to the MHRA if they thought their drug met the requirements that my noble friend has set out, with supportive evidence on this from the earliest stages of their clinical trials. The decision would be at the MHRA’s discretion, and it would consult on the process. There must be comprehensive data collection on the benefits and undesirable effects of any provisionally
licensed medicine or medical devices. Patients receiving them under this licence should be recorded on a centrally held register, and outcomes must be monitored for up to two years before a formal licence is issued. The market authorisation company must provide an annual update on the product’s characteristics with the benefits and side effects, and the data must be accessible to doctors and patients. That would allow rapid access to the NHS and at the same time protect patient safety.
As the Minister said at Second Reading, it is this balance between patient safety and innovation that is so crucial. If we do not facilitate innovation in the UK, our patients suffer; stifling innovation is a threat to greater patient safety. We want our patients to have access to the latest treatments in as safe a way as possible. This is what the amendment, which is backed by the Royal College of Physicians, the Royal College of Surgeons of England and the Faculty of Pharmaceutical Medicines, seeks to achieve.
The Bill also concerns medical devices. We need to make sure that the arrangements are right for registry, so that post-market monitoring is in place for devices and problems are rapidly picked up as they arise. The principles behind Amendment 26—a two-year provisional licensing system—apply to devices too, as articulated in Amendment 90. A provisional licence system would help to avoid the invidious situation described by the noble Baroness, Lady Finlay, at Second Reading, where start-ups can have a brilliant idea for a device and do the safety studies required by MHRA, but are then unable to fund the utility study in the second phase of licensing. It is clearly wrong for UK businesses, the taxpayer and the NHS that such start-ups are then bought out by overseas manufacturers that market the device back to the health service at great profit. A provisional MHRA licence would allow the start-up to market to the NHS when safety studies have been completed and as the utility data is gathered.
Meanwhile, by using powers under Amendment 84, also in the name of the noble Baroness, Lady Finlay, the Government would ensure that where medical devices are trialled, whether informally, through a clinician using a device, or formally through a study, the results are always available. Greater data transparency is surely to be welcomed.
Amendment 84 would also give the Government the opportunity to review the various thresholds for medical device trials. At present, new devices deemed to be substantially equivalent to something already on the market are certified as safe by a notified body and can go on to be used quite straightforwardly. This contrasts with standards for introducing new pharmaceutical products, as data from robust clinical trials, including comprehensive adverse event reporting, are not mandated. Devices certified in this way are not tested to establish whether they deliver significant patient benefit, rather the system really only establishes that a device is not obviously unsafe and that it performs its intended function. Even where clinical trials are required, because a device is deemed sufficiently different from what is already on the market, the rules around how the research is constructed are looser than those for medicines. In short, the process for medical device trials is at present much more about getting the product to market than robustly testing its efficacy. A provisional licensing
system for devices marketed to the NHS would improve safety monitoring while supporting UK device development, because the current system squeezes out small innovative start-ups.
Taking these amendments together, we seek to make the UK an innovative and safe marketplace for trialling and developing medicines and medical devices. I believe these objectives are entirely consistent with those of the Government as described by the Minister. I hope that the Government will look very carefully at these amendments, which have been drafted with the support of the esteemed medical Royal Colleges that I have mentioned. I beg to move.