Regulating advanced therapies in the UK: an interview with Michael Rawlins

In this interview with Professor Sir Michael Rawlins, chairman, Medicines and Healthcare products Regulatory Agency, learn more about the future of regenerative medicine in the UK.

In this interview with Professor Sir Michael Rawlins, chairman, Medicines and Healthcare products Regulatory Agency (MHRA), learn more about the future of regenerative medicine in the UK.

Michael Rawlins

Sir Michael is chairman of the Medicines and Healthcare products Regulatory Agency (since December 2014). He is a clinical pharmacologist and specialist in internal medicine. He was professor of clinical pharmacology in Newcastle, and physician at the Newcastle Hospitals, from 1999-2006. He was chairman of the Committee on Safety of Medicines (1992-1998), chairman of the Advisory Council on the Misuse of Drugs (1998-2008) and founding chairman of the National Institute for Clinical Excellence (1999-2013). He is recent past president of the Royal Society of Medicine (2012-2014). Currently Sir Michael is chairman of UK Biobank, honorary professor at the London School of Hygiene and Tropical Medicine, and emeritus professor at the University of Newcastle upon Tyne. Sir Michael Rawlins was appointed Knight Grand Cross of the Order of the British Empire (GBE) in 2017 for services to the safety of medicines, healthcare and innovation.

Please introduce yourself and outline the role of MHRA?

I am, by training, a clinical pharmacologist and specialist in internal medicine. I spent 33 years (1973-2006) at Newcastle of the professor of clinical pharmacology and physician at the Newcastle Hospitals (UK).  MHRA is responsible for ensuring that medicines and devices available to patients are appropriate quality, effective for the conditions indicated and safe in relation to their efficacy.  In addition, MHRA has responsibility for authorizing clinical trials carried out in the UK. MHRA also includes the National Institute for Biological Standards and Control (NIBSC) and the Clinical Practice Research Datalink (CPRD).

What have been some of your highlights as Chair of the MHRA over the last 3 years?

Unquestionably the result of the referendum last year was a monumental decision for the country. The UK’s regulatory system is intrinsically tied up the EU and Brexit will inevitably have a massive impact on the Agency.

What are your predictions for the next 3 years?

In the area of regenerative medicines and advanced therapies, we are already seeing an increase in gene therapy products and this is set to continue. This includes therapies where modified viral vectors are used directly in the patient to treat a disease, but importantly also those where patient cells are genetically modified outside the human body and then re-introduced to cure a specific disease. The latter includes the already approved drug Strimvelis (GlaxoSmithKline, Ireland), which treats a genetically inherited immunodeficiency. In the near future we will also be seeing CAR-T cell therapies looking to enter the market as licensed therapies. These therapies are part of the rapidly expanding sector of immunotherapies.

As can be seen from the examples above, another area where we will see a continuing expansion is that of personalized medicines. In the area of advance therapies, this will in the main consist of patient cells being modified and expanded to provide treatment for a variety of conditions.

How are regenerative medicines and cell therapies regulated in the UK?

Cell-based therapies that are medicines are overall regulated in a very similar way to other medicines, but additional provisions ensure that the specific requirements for these medicines are given due attention. At the moment they are regulated as Advanced Therapy Medicinal Products (ATMPs). At the clinical trial stage there is very little difference of the regulatory framework, but MHRA has assessors and inspectors with appropriate specialisms to ensure that the specific requirements of these medicines are fully taken into account for any regulatory decision that we make. MHRA also offers both regulatory and scientific tailormade advice for all stages of development of regenerative medicines.

When it comes to the actual Marketing Authorization, ATMPs, like other Biotechnological products, are currently regulated at European level and assessed via the centralized procedure, which is under European Medicines Agency administration and involves input from all Member States. The ATMP Regulation (1394/2007) makes additional provisions to ensure that the specific requirements of ATMPs are fully taken into account during any assessments. This includes the establishment of a specialized committee, the Committee for Advanced Therapies (CAT), comprised of experts from different member states with specific expertise covering all aspects of ATMPs. This committee is key to any marketing authorization approval for ATMPs.

In the US, unapproved treatments are increasingly being made available through ‘right-to try’ — is there a mechanism for UK patients to access similar treatments in similar ways?

In the UK medicines that are not licensed are available in the course of clinical trials and additionally either under the specials scheme and, for ATMPs, potentially also under the ATMP specific hospital exemption (HE). Both the Specials scheme and the HE are under the discretion of the treating clinician. Medicines given under these schemes do need to be manufactured to the appropriate GMP quality standards.

MHRA also has the Early Access to Medicines scheme which is open to any medicine and which aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need. Manufacturers will have to apply for their product to be considered for the scheme. Find out more on our website.

What do you expect for the future of regenerative and cell therapies in the UK?

The regenerative medicines field is very active in the UK and we hope this will continue in the future. MHRA is working to ensure that we continue to provide a wide range of regulatory and scientific advice to the field.

Further reading

• UK Government, Office for Life Sciences: Early access to medicines scheme (EAMS): how the scheme works