Published November 2016
An innovative therapy (IT) is a newly introduced or modified therapy with unproven effects. Unlike research, which follows a predetermined course of action set out in a protocol, experimental or innovative therapy involves a more speculative approach to the patient’s care and may be adapted to the individual’s response. However, such innovations may blur the distinction between treatment and research. Innovative medicines (unlicensed or ‘off-label’ use) are used quite commonly, but there are issues to be explored and addressed concerning other innovative treatments and the recording and sharing of IT results (both positive and negative).
Are there recent scientific, legal or social developments?
The use of ITs has emerged as an important and difficult issue in recent Council reports, such as on children and clinical research and novel neurotechnologies. The recent Ebola outbreak also led to discussion surrounding the acceptability of giving experimental drugs or vaccines to sufferers of the disease outside the confines of a traditional research trial. In March 2016 the UK government passed the Access to Medical Treatments (Innovation) Act 2016 (ATMTI Act 2016) which ‘aims to promote access to innovative medical treatments’ by establishing a database of ITs used in the UK, and the results of those treatments.
Are there complex ethical issues?
The use of ITs raises a number of ethical issues because the border between research and treatment is less clearly defined. Physicians involved in IT will need to be aware of the potential conflict that might arise between the goal of furthering medical knowledge and ensuring the welfare of the individual patient, and the potential for this conflict to undermine trust in the doctor-patient relationship. Patients may feel objectified if doctors view their condition as a subject for experimentation or for professional development via publication. Other patients may view the ability to participate in experimental therapies in a positive light if they see the treatment as their only chance of getting better. However, the ability to give informed consent in this context may be compromised by the emotional pressures of suffering from an incurable and potentially life-threatening condition. Furthermore, implicit trust in the medical profession and in the efficacy of modern medicine may cause both patients and doctors to overlook or downplay the risks inherent in a procedure. There is the question of whether clinicians involved in IT should have a duty to record and share any insight gained regarding the benefits or negative outcomes of the intervention, and if so, how this could best be achieved. This information might be of use to enable other patients to benefit from the same treatments or to avoid suffering the same adverse effects, as well as to guide potential future research strategies. However, this may further blur the distinction between what is considered treatment and what is considered research. There may also be concerns regarding equal access to IT. Patients that are well-informed and confident may request ITs which may also put pressure on health care professionals to offer them.
Is there a potential policy impact?
Unlike research, IT is aimed primarily at treating particular individuals, and so any proposed regulation would ideally need to maintain sufficient flexibility to allow for variations in individual circumstances. The biggest challenge for policy makers may be in defining exactly what constitutes treatment and what constitutes research, and how to regulate the spectrum of interventions that may fall under the umbrella term of IT. There are clear policies in law, and government and NHS guidelines concerning the use of unlicensed and ‘off-label’ medicines for either ‘compassionate use’ or on a ‘named-patient’ basis. There is however a lack of clear policy or governance on other ITs (such as innovative surgery). The ATMTI Act 2016 does begin to address the issues of recording the results of ITs. It has yet to be implemented, so there is still scope to influence the way that the database is set up and used, and to question whether existing models used for research governance, such as research ethics committees, are appropriate in this context and whether an international database is also needed.
Is it a subject of public concern?
ITs are often used in the context of a life-threatening condition when all other treatments have failed, and so is likely to be of significant concern for the public. The ATMTI Act 2016 (and the prior version of the Bill) have received numerous criticisms from the public and medical professionals (including the Medical Research Council), and so the implementation and any amendments of it will be of public concern.
Is consideration timely?
As well as the ATMTI Act 2016, there is also an ongoing government Accelerated Access Review (AAR) which aims to “speed up access to innovative drugs, devices and diagnostics for NHS patients” which includes the Early Access to Medicine Scheme (EAMS) which was independently reviewed this year.
Can the Council offer a distinctive contribution?
There is an opportunity for the Council to contribute in this area by conducting an in-depth analysis of the complex ethical issues involved and providing much needed guidance for both policy-makers and physicians.
Possible future work topics
This is one of the topics that have been suggested as possible project areas for further investigation by the Council. These topic summaries do not aim for comprehensiveness; rather, they are intended to sign-post some of the key considerations and to provide a starting point for discussion. Each summary includes links to relevant publications on the topic.
Possible future work topics are selected and/or revised regularly, following discussions among members of the Future Work Sub-Group and the Council. This set of topic summaries was published in November 2016.