Patient access to experimental treatments

In some circumstances, patients can access medical treatments before they have been subject to rigorous testing and approved by medical regulators, which are often referred to as ‘experimental treatments’. Patients might access these treatments when they are in a desperate situation, have exhausted all other options, or are not able or do not want to wait for the approval process.

This briefing note considers why, and in what contexts, patients might access experimental treatments, how they are regulated in the UK, and ethical questions raised by their use.

What do we mean by ‘experimental treatments’?

We use the term experimental treatments, but terms used elsewhere include innovative, novel, unproven, unvalidated, non-standard, and unlicensed treatments. (We note that there are problems with each of the terms used to describe this phenomenon, for example that they are value laden and have different meanings to different people.) There is a spectrum of treatments that might be considered to be experimental, from those which have never been used in humans, to those which are used routinely but are not licensed for the condition in question. Experimental treatments include medicines, surgery, the use of medical devices and implants, stem cell and gene therapies, and fertility treatments. (This briefing note excludes tests and procedures that are not intended to be therapeutic, such as diagnostic testing.)

This briefing note does not consider treatments that are being tested in clinical trials, as these are subject to specific regulatory frameworks. The use of complementary medicine is explored in a separate paper by the Nuffield Council on Bioethics.5

Why might patients consider experimental treatments?

Patients and their families might consider experimental treatments if proven treatments have not worked or are not available. While some patients can access experimental treatments by participating in clinical trials, not all patients are eligible for or able to take part in trials. In addition, the process of testing and approving new treatments through clinical trials is often complex and lengthy, particularly for medicines.^*

Patients’ decisions about experimental treatments might be influenced by a range of factors including their own values and goals, the views of their family and community, advice from healthcare professionals and others, marketing activities of companies offering treatments, and available funding.^**

* EURODIS (2017) Early access to medicines in Europe: Compassionate use to become a reality; BIO, BioMedTracker, Amplion (2016) Clinical Development Success Rates 2006-2015; ABPI (2012) Time to flourish – Inside innovation: the medicine development process. Shorter times to market is possible, and a number of initiatives by regulators aim to speed up the process of approval, such as the Office for Market Access; MHRA Innovation Office (2018) Read our case studies. See also Gov.uk news story (23 October 2018) Faster access to treatment and new technology for 500,000 patients.

** See, for example, Sleeboom-Faulkner M and Kato M (2017) Motivations for seeking experimental treatment in Japan BioSocieties 13:1, 255-75; Snyder J et al. (2014) “I knew what was going to happen if I did nothing and so I was going to do something”: Faith, hope, and trust in the decisions of Canadians with multiple sclerosis to seek unproven interventions abroad BMC Health Serv Res 14: 445.