Regulation and governance

There are several ways in which experimental treatments can be supplied and offered to patients within the UK legal and regulatory framework.9 There is uncertainty about how some medical treatments will be regulated when the UK leaves the EU, although the UK regulatory body for medicines and devices has stated a commitment to continuing close working relationships with European partners.10

Regulation of product supply

Medicines must have marketing authorisation (a licence) before they can be supplied in the UK, from either the UK authority – the Medicines and Healthcare products Regulatory Agency (MHRA) – or the European Commission (EC) after assessment by the European Medicines Agency (EMA).11 The licence is issued following evidence gathering and clinical trials to assess a medicine’s safety, quality, and efficacy, and commits the manufacturer to ongoing drug safety monitoring.12 However, EU Regulation includes a provision for compassionate use of unauthorised medicines.13 In the UK, the specials exemption allows the supply of an unlicensed medicine on request from a healthcare professional in order to treat a patient in their care where no equivalent licensed treatment is available.14 The early access to medicines scheme (EAMS) gives UK manufacturers a route to offering medicines 12-18 months before they have been licensed. Manufacturers can apply if they have promising scientific evidence on efficacy, and there is a clear unmet medical need.15

Medical devices and implants must have a CE mark, which certifies that they meet European safety and performance standards, before they can be supplied in the UK.16 However, clinicians and manufacturers can apply to the MHRA for exceptional use of a medical device that has not been CE-marked if there is no certified device available that meets the needs of an individual patient. The device manufacturer must provide evidence of safety and the clinician must provide justification for its use.17 Under the in-house manufacture exemption, devices that are made in a healthcare establishment can be used for patients within that establishment without certification.18

Advanced therapies, such as stem cell and gene therapies, must have a centralised European marketing authorisation, granted by the EC following assessment by the EMA, before they can be supplied in the UK and Europe.19 However, the hospital exemption allows ‘non-routine’ use of custom-made advanced therapies provided they are manufactured in authorised facilities and used in the same member state. There is no requirement to notify the MHRA of treatments offered under the hospital exemption.20 The specials exemption can apply to advanced therapies as well as medicines, where no equivalent licensed treatment is available.21

Regulation of medical practice

General Medical Council (GMC) guidance states that healthcare professionals must provide effective treatments based on the best available evidence, and that patients must be told whether a proposed treatment is experimental and about any additional risks or uncertainties.22 Beyond this, and within the constraints of available funding, it is up to healthcare professionals to judge what treatment to offer based on their knowledge of the patient. This can involve administering or prescribing unlicensed treatments, or prescribing licensed medicines or CE-marked medical devices ‘off-label’, which means for a use or purpose different to that for which they have been licensed.23 This could include use for a different dosage, a different duration of treatment, or in a different patient group, such as a drug which has only been licensed for adults being prescribed to a child, or a different disease.24

Following a legal challenge mounted by two pharmaceutical companies, a UK Court recently affirmed that the drug Avastin, a licensed cancer treatment, can be prescribed off-label for a common eye condition which it is also known to be effective for.25

Emergency situations

In a rapidly spreading epidemic or other emergency situation with high mortality rates it might not be possible to initiate clinical trials immediately, and national authorities can allow experimental treatments as part of the emergency response.26

References

9 Other relevant regulations exist that cover the management of hospitals and clinics, the use of human tissue, product safety, tort law, and consumer protection. These areas are regulated by various bodies and legislation including the Care Quality Commission, the Human Tissue Authority, and the Consumer Rights Act 2015.
10 MHRA news story (4 October 2018) Making a success of Brexit; HC Deb (22 October 2018) c60; Gov.uk (2018) Guidance: how medicines, medical devices and clinical trials would be regulated if there’s no Brexit deal.
11 Different licensing procedures apply depending on whether the manufacturer intends to market the medicine in the UK and/or in other specific EU countries, or throughout the EU. See: MHRA (2014) Guidance: Apply for a licence to market a medicine in the UK.
12 Human Medicines Regulations 2012 (SI 2012/1916); MHRA (2014) Guidance: Good pharmacovigilance practice (GPvP).
13 Regulation (EC) No 726/2004 of the European Parliament and of the Council of 31
March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency.
14 MHRA (2014) The supply of unlicensed medicinal products ‘specials’, MHRA guidance note 14.
15 See MHRA (2016) Guidance – Early access to medicines scheme (EAMS): how the
scheme works; Strategy& (2016) The early access to medicines scheme (EAMS): an
independent review.
16 In the EU, certification is carried out by independent organisations accredited by the competent authority in each member state. Directive 93/42/EEC of 14 June 1993
concerning medical devices; The Medical Devices Regulations 2002; MHRA (2017)
Guidance: notified bodies for medical devices.
17 EC (2016) Clinical evaluation: a guide for manufacturers and notified bodies under Directives 93/42/EEC and 90/385/EEC; MHRA (2014) Guidance: exceptional use of non-CE marked medical devices; Regulation 12(5) of The Medical Devices Regulations 2002.
18 MHRA (2014) Guidance: In-house manufacture of medical devices.
19 The Human Tissue Authority regulates the donation, procurement and testing of tissues and cells used in the manufacture of ATMPs. The manufacturing process following procurement and removal of tissues and cells from a tissue bank is regulated by the MHRA. MHRA (2015) Guidance: Advanced therapy medicinal products: regulation and licensing.
20 MHRA (2011) Guidance on the UK’s arrangements under the Hospital Exemption
Scheme. Facilities have to comply with Good manufacturing practice, see MHRA (2018) Guidance: good manufacturing practice and good distribution practice.
21 MHRA (2015) Guidance: advanced therapy medicinal products: regulation and
licensing.
22 GMC (2014) Good medical practice; GMC (2008) Consent: patients and doctors
making decisions together.
23 MHRA (2014) Guidance: off-label use of a medical device; see also Sutherland A and Waldek S (2015) It is time to review how unlicensed medicines are used Eur J Clin Pharmacol 71: 1029-35.
24 Nuffield Council on Bioethics (2015) Children and clinical research: ethical issues.
25 General Pharmaceutical Council (2018) GPhC statement on Avastin; [2018] EWHC 2465 (Admin).
26 Article 5 of the Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community code relating to medicinal products for human use; WHO (2016) Guidance for managing ethical issues in infectious disease outbreaks.

Previous work

Contact us

Nuffield Council on Bioethics
28 Bedford Square
London
WC1B 3JS

bioethics@nuffieldbioethics.org

+44 (0)20 7681 9619

Sign up for e-news

Community

Explore by topic