Rebecca Lowe: Access to experimental medicine is extensive in the UK, but the fashionable focus on patient choice is only skin deep

Although it might seem obvious that freedom-lovers should be fighting for wider access to experimental medicine — whether by demanding weaker regulations, or by arguing that anyone should be able take any drug they want, whenever they want to — it isn’t. Two key considerations arise: the opportunities we should have to access such medicine, and the extent to which our exercising of those opportunities should be governed by our own decision-making.

The Access to Medical Treatments (Innovation) Act 2016,[1] which was proposed in the Commons by Chris Heaton-Harris, has been incorrectly touted as the enactment of the controversial 2014-15 ‘Saatchi Bill’.[2] Lord Saatchi wanted to try to decrease resistance to the prescription of experimental medicines by affording doctors immunity from litigation — an approach criticised as dangerous (it risked ‘undermining’ evidence-based medicine),[3] illiberal (it could ‘remove’ a patient’s right to sue),[4] and unnecessary (such litigation was apparently ‘vanishingly rare’).[5] After similar accusations were raised against Heaton-Harris, the final Act featured no discussion of such matters, but rather provision for a database of innovative medicines. Greater access to such information sounds promising, but there were already comprehensive databases, run by the NHS, the EU, and various charities. Patients themselves do lack access to much existing data — although complications arise around ownership and privacy, and the need for ‘blind’ participation within testing — but the Act does not explicitly cater to this.

However, while it is accepted that the UK has ‘sometimes lag[ged] behind’ in its approach to medical innovation,[6] there is currently extensive scope for patients both to participate in clinical trials and to access unlicensed drugs when involvement in these trials isn’t possible. Trials aren’t open to all patients (eligibility requirements relate to factors such as health, previous treatment regimes, and life expectancy), but restrictions here should surely remain primarily the domain of drugs manufacturers and testers. It makes sense, therefore, to focus for now on patients who don’t qualify for their desired trials.

The recently-introduced Early Access to Medicines Scheme is aimed at hastening the overall licensing process,[7] but has also upped individual-user access, on top of the provision of ‘specials’.[8] Indeed, the two clearest ongoing restrictions to access aren’t systemic blocks in need of policy liberalisation — although the NHS’s structural problems hamper its responsiveness to innovation — but, rather, take the form of specific doctors and specific manufacturers. The latter is unsurprising: access to a drug depends not only on patients being allowed to take it, but also its supply. Supporters of the recent American ‘Right to Try’ legislation have had to recognise that it doesn’t equate to the right to force drugs companies to comply with patient requests.[9]

Manufacturers are often wary about allowing patients access outside of formal trials, not least owing to the difficulty of choosing to whom they should supply limited products.[10] It’s also often contended that such access risks interfering with the testing process, thereby damaging the common good. This argument is sometimes made on the grounds that access outside of testing can decelerate, or even prevent the approval of promising drugs. Testing, however, should surely be premised on gaining awareness of both the successes and failures of drugs — including evidence from ‘real-world’ cases— rather than hopes that ‘picked winners’ will sail through the process.[11] A stronger argument relates to the need for ‘blind’ participants in trials: if people know they can access drugs outside of trials, why take the chance of being assigned a placebo within one? Various mitigations could include, again, manufacturers taking control by setting their own guidelines for all forms of access, and also the use of additional payments as an allocation tool (unlicensed drugs are currently provided at cost).

It’s also somewhat unsurprising that access to experimental drugs can be blocked by a patient’s doctor. The NHS ‘Constitution’ is heavy on the language of patient choice,[12] but it remains the case that a ‘patient is entitled to drugs that the GP believes are necessary, not those which the patient feels should be prescribed’, and that ‘GPs are responsible for all prescribing decisions they make’.[13] Moreover, doctors often refuse or are unable to prescribe a drug for reasons unrelated to the specific patient’s needs — such as bias, lack of experience, or on cost grounds — including considerations related to their local area’s resources, and, therefore, the needs of others, too.[14] Although patients can technically seek alternative opinions within the NHS, or turn to the private sector, it would seem sensible, at least in extreme cases — where a patient is determined to take a unlicensed drug (and its manufacturers are happy to supply it), yet they cannot find any doctor to agree — to also allow the burden of responsibility to be shifted on to the patient. Criticisms of the Saatchi proposal relating to the ‘removal’ of a patient’s right to sue would be irrelevant here, as it would be the patient themself who had knowingly chosen to take on the risk.[15]

A recent change of legal attitude with positive implications for increased patient choice stems from Montgomery v Lanarkshire [2015], in which the Supreme Court stated that ‘[a]n adult person of sound mind is entitled to decide which, if any, of the available forms of treatment to undergo and her consent must be obtained before treatment interfering with her bodily integrity is undertaken’.[16] Responses to this have focused on consent — although the statement of entitlement regarding decision-making is noteworthy — as it counters the standard ‘Bolam test’. Bolam arose from medical negligence cases, holding that a doctor’s standard of care is acceptable as long as it reaches that of a ‘responsible body of medical opinion’, rather than requiring them to involve the patient in risk assessment.

A further complication arises, however, when a patient is incapable of consent — particularly in the case of children.[17] Until people reach the age of majority, it is their parents’ or carers’ responsibility to make certain important decisions for them, including many related to healthcare.[18] Recent high-profile cases have highlighted the role courts currently play when parents disagree with doctors about appropriate, often experimental, medical treatments for terminally-ill children.[19] Although this essay focuses on consenting adults’ access to such treatments, these court cases remind us of the unanswered ethical questions related to the use of new technology in healthcare, as well as the need constantly to assess whether our laws are fair and just. When cases show the law to be in question, they add to a wider process of deliberation, taking into account the common good, as well as exceptional individual circumstances. There are outstanding questions about the societal costs of the rise of legal activism, however, not least in terms of its effect on our political institutions.

But lobbying through the courts often seems easier, quicker, and cheaper — with recourse to crowd-funding — than the traditional route of gradual democratic change. The legal route will surely be pursued increasingly within the area of access to experimental medicine, involving cases against doctors, manufacturers, and even parents. This is not least because although access to these drugs is relatively extensive in the UK, big questions remain about a patient’s lack of ability to take responsibility for their own risk. It seems clear that the fashionable focus on patient choice is still, in many ways, skin deep.

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[1] Access to Medical Treatments (Innovation) Act 2016.

[2] Medical Innovation Bill [HL] 2014-15.

[3] Editorial ‘Undermining the Hippocratic Oath: The Medical Innovation Bill’, The Lancet Oncology, 2014.

[4] Attributed to the NHS Litigation Authority, in Hillen, M. ‘The Road to Hell Is Paved with Good Intentions’, The Opthamologist, 2014.

[5] Lewis, A. ‘The Saatchi Bill: When Only the Fig Leaf Remains’, The Quackometer, 2016.

[6] Taylor, H. et al Accelerated Access Review: Final Report,UK Government, 2016.

[7] See, for instance, Office for Life Sciences ‘The Early Access to Medicines Scheme (EAMS): Operational Guidance, UK Government, 2016.

[8] See, for instance, MHRA ‘The supply of unlicensed medicinal products (“specials”): MHRA Guidance Note 14’, UK Government, 2014.

[9] Wallis, C.‘The So-Called Right to Try Law Gives Patients False Hope’, Scientific American, 2018.

[10] This interesting paper discusses one company’s outsourcing of such decisions to an NYU-based ethics board: Arthur Caplan et al ‘Fair, just and compassionate: A pilot for making allocation decisions for patients requesting experimental drugs outside of clinical trials’, Journal of Medical Ethics, 2018.

[11] The Accelerated Access Reviewreport cited above is, for instance, heavy in its ‘industrial strategy’-style approach. There are also many well-known concerns about the testing process, not least relating to unpublished data.

[12] NHS ‘The NHS Constitution for England’, UK Government, 2015.

[13] BMA ‘Prescribing in general practice’, 2018.

[14] The ‘post-code lottery’ is a misunderstood issue: in a freer system, local competition could drive up standards within the NHS; it is already the case that the NHS ‘Constitution’ purports to give people the opportunity to ‘choose where to receive treatment’.

[15] i.e. volenti non fit injuria.

[16] Montgomery v Lanarkshire [2015] UKSC 11.

[17] Adults who are incapable of consent clearly wouldn’t be suitable candidates to take over the burden of responsibility as proposed above.

[18] This is not to suggest that parents should be able to make choices about non-medically-necessary changes to their children’s bodies.

[19] See, for instance, Re Ashya King [2014] EWHC 2964 (Fam), or Great Ormond Street Hospital v Yates [2017] EWHC 1909 (Fam).

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