A no-deal BREXIT impacts medicine, care, research and access to treatment for PSC patients
PSC Support has responded to the Health and Social Care Committee consultation on behalf of the PSC patient community on the impact of a no-deal Brexit.
Martine Walmsley, Chair of Trustees of PSC Support, said, "PSC is a disease with no curative treatment, yet PSC research is flourishing, especially here in the UK where we have superb investigators and research institutions, and actively engaged PSC patients willing to take part in research. We are concerned that a no-deal Brexit will have a negative impact on research relationships between the UK and the EU, and be detrimental to PSC research progress."
Summary of PSC Support's Health and Social Care Committee consultation response
PSC Support is concerned that leaving the European Union without a withdrawal agreement will negatively impact on the lives of people affected by PSC in terms of:
- access to medicines,
- clinical care quality,
- research collaboration,
- access to clinical trials,
- the development of an effective treatment for PSC
- access to potential effective treatments for PSC.
PSC Support is the leading UK patient organisation for people affected by a rare, immune-mediated liver disease that affects the bile ducts and liver called primary sclerosing cholangitis (PSC).1,2 PSC affects only around 80 people per million in the UK 3 and is designated an “orphan” disease. Currently, liver transplantation is the only proven life-extending intervention and PSC is one of the leading indicators for liver transplant in the UK, accounting for 14% of liver transplants in the UK last year 4. To date, medicines developed have been unsuccessful in improving patient survival. Despite the lack of effective medical treatments for PSC5, research is flourishing. Those PSC patients who have had transplants must take life-long anti-rejection medicines.
PSC Support is concerned about the impact of a no deal EU Exit on PSC patients, both long and short term on access to medicines, clinical care quality, medicine regulation and access to and continuing PSC research in the UK.
Access to Medicines
Patients who have had liver transplants for PSC must take immunosuppressive medication for life. It is well recognised that immunosuppressants are critical dose drugs. We understand that companies have been asked by the Government to stockpile drugs in the event of a no deal Brexit in order to manage potential medicine supply chain issues.
In the event of a medicine shortage, we are concerned that patients’ existing immunosuppressants might be substituted with generic formulations. This is of considerable concern because they may not be equivalent to one another and may put patients at risk. While the active ingredient may be the same, the excipients are not and can create a different absorption profile. Blood concentrations are important for critical dose drugs and therefore immunosuppressive drugs cannot be considered interchangeable. These drugs are not trivial and should not be considered substitutable as with most other medications.
Recommendation 1: Immunosuppressants are not substituted for generic brands.
Recommendation 2: Transplant patients have access to their regular brand of immunosuppressant.
Clinical Care Quality
As a Member State of the EU, the UK NHS has taken a leading role in both clinical and patient involvement in the development of the Rare-Liver European Reference Network (ERN). The Rare-Liver ERN is an infrastructure that allows knowledge and clinical expertise about rare liver diseases to be shared between centres and across borders; it facilitates access to diagnosis and transformative care, because that knowledge and expertise can be shared without burden of long distance travel; it facilitates research, by providing access to clinical cohorts of sufficient size to support clinical trials; and it connects researchers and healthcare systems to clinicians and patients, expediting innovation, access to knowledge and the development of new treatments. Membership of the ERNs is currently reserved to EU and European Economic Area countries. A no deal Brexit will result in the immediate exclusion of the UK NHS from the Rare Liver ERN, and all the benefits to patients, clinicians and research it brings.
Recommendation 3: The NHS can continue to lead and be a member of the European Reference Networks.
European Research Collaboration
Research and clinical care are intertwined in rare disease, especially so for PSC patients. Collaboration across European Member States is fundamental to delivering improved outcomes for patients. The UK currently benefits from being able to share information internationally. A no deal Brexit risks access to EU research funding and risks misalignment of clinical trials regulation, meaning that important collaborative opportunities with the EU-27 could be lost. This will impact adversely on volume and quality of research outputs and participation opportunities/our ability to be partners in research. PSC is complex and difficult to research, not least because of the heterogeneous population and variable natural history. International collaboration is critical for PSC research progress. It is important that our researchers can continue to collaborate with other EU countries and lead on PSC research.
Recommendation 4: The benefits of EU research collaboration should be recognised and encouraged by ensuring that related regulatory systems remain aligned.
Health Technology Regulation and Development
The EU regulates health technologies, such as pharmaceuticals and medical devices, and products of human origin such as blood, tissues and cells. Pharmaceutical companies can make new medicines available everywhere in the EU through the single centralised marketing authorisation procedure provided by the European Medicine Agency, instead of having to apply for authorisation in each individual Member State. The UK has always been a beneficiary by being within this framework and offers world class research expertise. Maintaining access to this centralised authorisation procedure must be a priority for the UK.
The EU regulatory framework includes incentives for the development of rare disease medicines, in particular for Orphan Medicinal Products (OMPs). OMPs enjoy cost-effective authorisation and licensing through the centralised process. This means that health technologies can be brought to market faster for the benefit of rare disease patients. While there is no medical treatment for PSC, research is flourishing and for the first time we are seeing Phase 2 trials in progress and Phase 3 trials in the pipeline. In the event of a no deal Brexit, the UK will no longer remain part of the EU regulatory framework and centralised process, and there will be a system where the 27 Member States offer incentives to develop rare disease medicines faster but the UK does not; we would have to secure new incentives for developing and approving rare disease medicines in the UK.
PSC Support is deeply concerned that a no deal Brexit could mean that pharmaceutical companies no longer have the incentive to develop treatments and run clinical trials for PSC in the UK, meaning that PSC patients would be unable to access a potential life-saving treatment through trials, or that PSC patients would experience major delays in accessing an effective treatment without the benefits of a centralised process.
Recommendation 5: Access to the centralised authorisation procedure is maintained.
Recommendation 6: PSC patients can continue to benefit from early access to the wide range of innovative health technologies available on the EU market. Recommendation 7: PSC patients do not miss out on the opportunities offered by participation in EU clinical trials.
1 Hirschfield GM, Karlsen TH, Lindor KD, Adams DH. Primary sclerosing cholangitis. The Lancet. 2013 Nov 9;382(9904):1587-99.
2 Karlsen TH, Folseraas T, Thorburn D, Vesterhus M. Primary sclerosing cholangitis–a comprehensive review. Journal of hepatology. 2017 Dec 1;67(6):1298-323.
3 Webb G, Ryan RP, Marshall T, Hirschfield GM. Autoimmune liver disease in the UK: a national primary care evaluation of disease geo-epidemiology. HEPATOLOGY 2017 Oct 1 (Vol. 66, pp. 186A-186A)
4 NHSBT Annual Report on Liver Transplantation 2017/8 (published Sep 2018) available from www.odt.nhs.uk/statistics-and-reports/organ-specific-reports/ (accessed 15 October 2018)
5 Goode EC, Rushbrook SM. A review of the medical treatment of primary sclerosing cholangitis in the 21st century. Ther Adv Chronic Dis. 2016;7(1):68–85