Medicines and Medical Devices Bill

Written evidence submitted by Cancer Research UK (MMDB10)

Public Bill Committee on the Medicines and Medical Devices Bill

Executive summary

1. Cancer Research UK welcomes the Government’s intention to enhance the UK medical research sector and ensure innovation benefits patients as quickly as possible. This Bill lays an important foundation for that ambition, and we look forward to further engagement on how to bring that ambition to life.

2. We also believe that a focus on domestic innovation cannot be a substitute for international collaboration, particularly with our close partners throughout Europe. It is possible to both accelerate the delivery of clinical trials and new medicines, whilst also maintaining our invaluable collaboration with researchers in Europe.

3. Although the Bill enables future changes to the regulation of medicines and clinical trials, the Government has not proposed any specific changes at this stage. Consequently, Cancer Research UK will not be advocating for any amendments to the Bill’s content.

4. Instead, our consultation response focuses on how the Bill’s power should be used, as well as the context in which those future regulatory changes may occur in. The outcome of negotiations on the future UK-EU relationship will significantly affect the limits of these new powers and their context, and as such will influence the Bill’s likely impact on patients.

5. It is crucial that any agreement between the UK and the EU on the future relationship:

a. Does not allow for any reduction in UK patients’ access to new medicines; and

b. Does not close off future opportunities for the UK to participate as fully as possible in
cross-border trials involving EU countries on the basis of the incoming EU Clinical Trial Regulation and associated infrastructure.

About Cancer Research UK

6. Cancer Research UK is the world’s largest charitable funder of cancer research, committing £546 million towards research in 2018/19. We receive no Government funding but do rely on the UK’s thriving research environment, as we seek to support the very best research to drive progress towards our ambition to see 3 in 4 people survive their cancer by 2034.

7. Clinical trials play an essential role in our efforts to achieve this ambition. They determine the effectiveness and safety of new medicines, and in the process provide patients with access to potentially life-saving new treatments still early in development. Cancer Research UK funds nearly 200 clinical trials and recruits around 25,000 participants each year to all trials we support.

8. Cancer Research UK is responding to this consultation because the Medicines and Medical Devices Bill directly affects the Human Medicines Regulations 2012 and the Medicines for Human Use (Clinical Trials) Regulations 2004. These regulatory frameworks directly impact our work, the patients we support through our clinical trials, and the researchers we work with to deliver them.

The Bill and the UK-EU relationship

9. Whilst the Bill could help maintain patient access to new medicines and UK access to pan-EU clinical trials, its capacity to achieve this will be subject to the shape of future relationship between the UK and the EU.

10. On medicines access, the Bill would allow the Government to establish new regulations on marketing authorisations for new medicines. This will likely mean an independent UK marketing authorisation process, along the lines set out in the Medicine and Healthcare products Regulatory Agency’s (MHRA’s) plans for a ‘no deal’ outcome in 2018 and 2019. [1] Alternatively, the UK could choose to unilaterally continue to recognise new European marketing authorisations as valid. However, depending on the outcome of UK-EU negotiations, the MHRA may be unable to participate and contribute its expertise in the European Medicines Agency’s (EMA’s) marketing authorisation process.

11. On clinical trials, the Bill could allow the Government to replicate the EU’s clinical trial application system, thereby reducing the administrative burden on UK-EU collaboration. This would be necessary if the MHRA had to develop a separate clinical trial application system that would operate in parallel to the EU’s.

12. Given the influence the UK-EU future relationship will have on how the Bill’s powers can be used, we recommend the Committee encourage the Government to regularly update and consult the medical research sector as negotiations progress.

13. Such an arrangement would be in line with the ethos of Clause 40 of the Bill, which requires the Government to consult relevant people and organisations when proposing regulatory changes. [2] We welcome this commitment, as input from the medical research sector is vital to help ensure future regulations deliver the greatest possible benefit to patients.

The Bill’s role in maintaining patient access to new treatments

14. The EMA’s centralised marketing authorisation process allows accelerated access to new medicines for patients in the UK and across Europe. It provides a route for companies to access the entire EU pharmaceuticals market based on one regulatory submission, with EU and EEA countries’ national medicines regulators working together to assess these applications.

15. If the UK pursues an independent medicines licencing system, companies will have to submit separate marketing authorisation applications to the MHRA in the UK, and to the EMA in the EU. The EMA covers 25% of global pharmaceutical sales; the UK on its own makes up just 3%. [3] Companies are likely to submit applications for new drugs to the EMA before the MHRA – meaning UK patients risk having slower access to the latest medicines.

16. This already happens in Australia, Switzerland and Canada, nations with standalone regulators, where medicine approvals can take between 5 and 12 months longer than in the EU. [4] This delay cannot simply be mitigated by regulatory reform – companies on average submit new drugs to these regulators between 3 and 4 months after they do to the EMA . [5] For some patients, especially with aggressive cancer types such as lung cancer, their disease can progress sufficiently in this time that they may no longer be eligible for the new treatment, and may miss out on access entirely.

17. To ensure patients see no delays in accessing new treatments, we recommend future regulations made using clause 2(1) of the Bill should be used to facilitate UK recognition of, and participation in, the EMA’s medicines licensing processes.

18. If an independent UK medicines licensing process is put in place, we recommend the MHRA should maintain equivalent documentation requirements for the UK and EMA marketing authorisation processes, as was proposed in the MHRA’s 2018/19 ‘no deal’ planning.

19. We also encourage the Committee to ask the Government how it will assess the availability of human medicines when considering future changes to regulations, as required under Clause 1(1)(2). [6]  

The Bill’s role in protecting UK-EU clinical research collaboration

20. International collaboration is a critical part of Cancer Research UK’s work to improve survival rates for people with cancer. Nearly 50% of all UK cancer research is international [7] and 28% of the trials Cancer Research UK (CRUK) supports take place with at least one other EU Member State. [8]

21. Working collaboratively across borders is particularly vital for rare and childhood cancers because one country alone may not have enough participants to run a trial. The UK participates in the highest number of pan-EU trials for rare and childhood diseases of any Member State. [9] As our understanding of the genomic drivers of disease increases, with patients stratified into ever smaller groups on this basis, the need for international collaboration will only increase.

22. Pan-EU trials are currently regulated by the Clinical Trials Directive (CTD), which will be replaced by the EU Clinical Trial Regulation (CTR) in 2022. The UK played a central role in the development of the CTR, which is widely regarded by researchers as a significant improvement. The CTR will be underpinned by digital infrastructure that should accelerate trial set-up times, improve safety reporting, and facilitate collaborative research.

23. This Bill can help stimulate the UK’s clinical research environment, but that must include ensuring UK-EU trials can continue, as they are hugely beneficial to both the UK’s status as a world-leading research environment, and to patient care here and across Europe.

24. We continue to recommend that the Government prioritise negotiating as close as possible a relationship between the UK and EU on clinical trials, including UK participation in the infrastructure which underpins the forthcoming CTR.

25. This would be in line with the Government’s current position of aligning with the CTR in areas where the alignment "best suits the interests of UK patients, industry, non-commercial researchers and hospitals". [10]

26. If the UK were outside the CTR this would create significant financial barriers for UK researchers collaborating with the EU. For example, UK-based sponsors of trials involving Member States may need to have a legal representative in the EU after the CTR is implemented. Evidence from our research community has shown that the cost of setting up an EU legal representative can range from £20,000 to £300,000 per year for UK non-commercial sponsors (e.g. universities). This could be prohibitively expensive for some non-commercial sponsors, thereby reducing the UK’s overall research output and attractiveness as a research environment.

27. If the UK does find itself outside the EU regulatory framework, we recommend clause 4(1) should be used to ensure the UK’s regulatory environment for clinical trials is coordinated with the EU’s. An example of this coordination would be ensuring the UK and EU use the same definition of a "clinical trial", as even simple divergences in regulations can create legal and financial barriers to collaboration. Such coordination will prevent barriers to collaboration and facilitate the UK’s engagement in useful collaborative frameworks, such as the CTR and its underlying IT infrastructure.

28. We also encourage the Committee to ask the Government how it will measure the UK’s attractiveness as a place to conduct clinical trials when considering future changes to regulations, as required under Clause 1(1)(2). [11]

Appendix: Responses to committee’s indicative questions

29. Cancer Research UK was invited to an oral evidence session scheduled for the 17th March, however this was cancelled. In preparation for this session we received a series of indicative questions the Committee may have asked. We have included our responses to these below:

30. If the EU exit transition period is extended beyond 31 December 2020, then what impact will this have on the effectiveness of the Bill?

a. This would depend on the length of any extension. If the CTR came into force before the end of the transition period, it would automatically apply in UK law, and the UK would have automatic access to its underpinning infrastructure. The Government would then need to decide if it wants to remain in the CTR regulatory framework after the transition period. If so, the UK’s capacity to change domestic clinical trials regulation under the powers contained in the Bill would be affected, though it is Cancer Research UK’s view that participation in the CTR would be a net positive to the UK research environment.

31. Is there a risk that in the future, where there is no requirement to implement EU law, there could be significant regulatory divergence between Northern Ireland and the rest of the UK? Is this problematic?

a. Our understanding is that, were the Northern Ireland Protocol to come into effect, future EU regulations on medicines, clinical trials and medical devices will apply to Northern Ireland but not automatically to the UK under the terms of the Withdrawal Agreement. This would include the CTR, once it comes into effect.

b. However, the situation is very unclear, for example on whether Northern Ireland would have access to EU databases, the MHRA’s role in enforcing regulations (including medicines authorisation) in Northern Ireland, and whether regulatory checks could affect the supply of imported medicines between Great Britain and Northern Ireland. We understand that how this will work is currently being decided by the UK-EU Joint Committee. We recommend the Government ensures the Committee considers how its decision will impact the flow of medicines and clinical research between Northern Ireland, the rest of the UK, and the EU.

c. The challenges that could arise from regulatory divergence between Northern Ireland and the rest of the UK is emblematic of the divergence’s wider issues. Simple divergence – such as different definitions of a clinical trial – can create legal and financial barriers to collaborative clinical research and patient access to new medicines.

32. How will the transition from the Clinical Trials Directive to the Clinical Trials Regulation affect the Bill?

a. The CTR is expected to come into force in 2022 - after the transition period ends – and will therefore not affect the Bill’s powers.

b. However, if the Government decided to align with the CTR in the future then this would have a knock-on effect on the Bill’s power to change regulation. For example, if the UK were formally aligned with the CTR’s regulations regarding clinical trial information transparency, [12] the Government would not be able to use the Bill’s powers to create regulations that restrict public access to this information.

c. Clause 4(1)(a) of the Bill explicitly "provides the means for provision to be made which is corresponding or similar to the EU Clinical Trial Regulations, if the Government were to choose to do so" – CRUK would support this.

33. What impact will this Bill have on research and innovation in medicines and the funding available?

a. The Bill’s direct effect will likely be negligible, since it itself is not making any significant changes to medicines and clinical trials regulation. However, how the UK’s future regulatory approach evolves – using the powers in the Bill – will be closely watched by industry, and so could have a real impact on the UK’s attractiveness as a destination for research and as a market for new medicines. The signals Government sends through this Bill about its appetite for the kind of regulatory harmonisation and cooperation the pharmaceutical industry has vocally supported are therefore important.

b. Other factors will also play a role though, including UK association with programmes like Horizon Europe, immigration reform, and Government investment in infrastructure.

34. The Government’s intention is to ensure that the UK is aligned with the forthcoming EU Clinical Trials Regulations. Would this sufficiently meet the Bill’s requirement for regulations to have regard to "the attractiveness of the relevant part of the United Kingdom as a place in which to conduct clinical trials or supply human medicines"?

a. The Government has committed to aligning with parts of the new EU Clinical Trial Regulation as much as possible, [13] and CRUK welcomes this. Alignment with the CTR would be seen by industry as a positive signal of the Government’s desire to maintain a broadly harmonised regulatory environment with the EU, which would help sustain the UK’s attractiveness as a launch market for new medicines.

b. The Bill’s powers to define the UK’s future regulatory environment is central to achieving this. We recommend the Bill be used to ensure the UK and EU maintain a cooperative regulatory approach, as this is in the best interests of patients and clinical research, particularly in the fields of rare and childhood diseases where international collaboration is already necessary.

c. However, the CTR does not cover all aspects of the research environment, meaning Government domestic policy must play a role in maintaining the UK’s attractiveness. For example, Government financial support for research (e.g. funding for the NIHR) will significantly affect the demand for research, especially demand for emergent fields such as precision medicine which commercial research considers too risky to invest in currently.

35. What opportunities does the Bill provide?

a. The Bill fills a legal need following EU Exit. The power to create new domestic medicines and clinical trials regulations under the Bill may offer some long-term opportunities, for example to ensure future UK clinical trial regulation reflects technological progress and innovative trial design. However, this should not come at the expense of international collaboration in clinical research.

b. As discussed above, the Bill also offers an opportunity to build a cooperative regulatory approach with the EU, as this will facilitate continued collaboration in clinical research. This collaboration will only continue to grow in importance as the Government continues its programme to expand the use of precision (genomic) medicine. Clinical research involving precision medicine relies on having access to patients with specific (and often rare) genetic profiles, making it necessary for these trials to recruit patients across multiple countries.

c. The Bill can support these multi-state trials and maintain the UK’s status as an innovation hub by creating a regulatory framework that enables cooperation with international researchers and their regulators.

36. What is a wish list to add to the Bill?

a. We do not have recommendations to add to the content of the Bill itself, given it is an enabling piece of legislation. Whilst outside the Bill’s direct scope however, we recommend the powers in the Bill are used to safeguard patient access to new medicines and UK-EU collaboration in clinical research, and not in a way that closes off future opportunities for the UK to cooperate closely with the EU on regulation.

Appendix: UK-EU negotiations and clinical research

CRUK recently published a policy paper outlining our recommendations to both UK and EU negotiators for how the Future Relationship can safeguard clinical research. Though outside the immediate scope of the Bill, this policy paper has informed our consultation response and we encourage you to read it. The paper’s executive summary is copied below, and you can read the full paper on our website .

Executive summary

Collaboration between the European Union and the United Kingdom on clinical research is a win-win. It drives progress for patients in Europe and across the world, helps our research environments to prosper and thrive, and delivers significant economic benefits.

Cancer Research UK has been working to safeguard this collaboration since the Referendum result. We are the world’s largest independent funder of cancer research, committing £546m (€620m) to research in 2018/19 alone. And while we are based in the United Kingdom, we are proud to be part of the thriving pan-European research environment. 28% of our clinical trials take place with at least one EU Member State, and at last count our researchers were partnering with over 400 organisations in the EU.

This paper presents our recommendations to negotiators on how UK-EU collaboration on clinical research can be maintained through the end of the Transition Period and into the future. Cancer Research UK have identified these recommendations by drawing on legal analysis prepared by the University of Sheffield and utilising the expertise of our researchers. We have endeavoured to recognise and respect the mandates of both the EU and UK and draw on precedent, while acknowledging that compromise will be needed from all negotiators to secure the best outcome.

While cancer societies and research organisations across Europe have noted that as-close-as-possible a relationship between the UK and EU is to the benefit of patients and research in both jurisdictions, we appreciate that Brexit will necessitate a reduced level of cooperation than exists at present. However, it is our view that it is the responsibility of all negotiators to protect the interests of patients, health and research in the future relationship. In that spirit, we recommend:

1. The Future Relationship Agreement formally recognises that a high level of protection for human health represents a shared value in the UK and EU’s regulatory approaches.

2. UK and EU negotiators commit to reducing barriers to UK-EU collaborative clinical research, including the establishment of a Research & Innovation Committee or Working Group to facilitate future cooperation on research (including clinical trials).

3. Full UK association to Horizon Europe, in order to facilitate cross-border collaborative research.

4. UK and EU negotiators commit to cooperate as far as possible on medicines licensing and regulation, patient safety, and minimising barriers to trade, including a best-in-class Mutual Recognition Agreement and the establishment of a Pharmaceuticals Licensing & Regulation Subcommittee or Working Group.

5. Prioritisation of data adequacy status for the UK, with an agreement to be reached before the end of the Transition Period.

May 2020


[1] Gov.uk (2019). Further guidance note on the regulation of medicines medical devices and clinical trials if there’s no Brexit deal. [Online]. Available at: https://www.gov.uk/government/publications/further-guidance-note-on-the-regulation-of-medicines-medical-devices-and-clinical-trials-if-theres-no-brexit-deal/further-guidance-note-on-the-regulation-of-medicines-medical-devices-and-clinical-trials-if-theres-no-brexit-deal [Accessed 11 March 2020].

[2] Parliament. (2020). Medicines and Medical Devices Bill. House of Commons. (Bill 90 2019-2021). London: The Stationary Office., p. 22.

[3] Cancer Research UK. (2020). The UK and EU: What people affected by cancer need from the future relationship. [Online]. Available at: https://www.cancerresearchuk.org/sites/default/files/whatpatientsneedfinal.pdf [Accessed 10 March 2020]., p. 8.

[4] Cancer Research UK. (2020). The UK and EU., pp. 8-9.

[5] Cancer Research UK. (2020). The UK and EU., pp. 8-9.

[6] Parliament. (2020). Medicines and Medical Devices Bill., p. 1.

[7] Cancer Research UK. (2014). Exploring the Interdependencies of Research Funders in the UK. [Online]. Available at: https://www.cancerresearchuk.org/sites/default/files/interdependencies_of_funders_in_the_uk_march2014.pdf [Accessed 10 March 2020]., p. 26.

[8] Cancer Research UK. (2020). The UK and EU., p. 4.

[9] Brexit Health Alliance. (2018). The impact of Brexit: Patient access to medical research. [Online]. Available at: https://www.nhsconfed.org/resources/2018/02/brexit-impact-patient-access-to-medical-research [Accessed 11 March 2020]., p. 2.

[10] House of Commons. (2020). Written question - 3773. [Online]. 2019 Session. Available at: https://www.parliament.uk/business/publications/written-questions-answers-statements/written-question/Commons/2020-01-15/3773/ [Accessed 1 April 2020].

[11] Parliament. (2020). Medicines and Medical Devices Bill., p. 1.

[12] European Medicines Agency. (2020). Clinical Trial Regulation. [Online]. Available at: https://www.ema.europa.eu/en/human-regulatory/research-development/clinical-trials/clinical-trial-regulation [Accessed 16 March 2020].

[13] House of Commons. (2020). Written question - 3773.

 

Prepared 10th June 2020