By Michael D Rawlins President, Royal Society of Medicine
Published in the BMJ (British Medical Journal) 15th April 2014
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Many doctors, myself included, will have occasionally tried to treat individual patients – where all else has failed – with novel interventions. This may be where there is no recognised form of effective treatment; or when existing one(s) have not produced the desired effect. The legal basis for doing so, at least for pharmaceuticals, has been the so-called “named patient” provisions of Section 9 of the Medicines Act 19682 which permits any doctor to be able “to sell, procure or supply a medicinal product to a patient under his or her care”.
Although my own experience has been disappointing there have been occasions, when intervening in such a manner, have subsequently led to significant advances. Indeed, such observations are arguably a form of n-of-1 trial.
Confidence in using the “named patient” provisions of the Medicines Act has though become eroded. A number of legal authorities have pointed out that departing from what is regarded as “established practice” or “the standard of care” leaves a doctor open to an action for negligence. The definition of “the standard of care” traditionally follows the Bolam principle2 as amended by the Bolitho decision3 but was forcefully criticised by Lady Butler-Schloss4 in her capacity as President of the Family Division of the High Court:
“The Bolam test ought not to be allowed to inhibit medical
progress. And it is clear that if one waited for the Bolam
test to be complied with to its fullest extent, no innovative
work such as the use of penicillin or performing heart
transplant surgery would ever be attempted”.
The Medical Innovation Bill attempts to rectify this situation. It proposes legislation stating that it would not be negligent for a doctor to depart from the existing range of accepted medical treatments, for a condition, in carefully defined circumstances. These circumstances – allowing for responsible innovation – are laid out in later Sections of the Bill. They include a plausible basis for the use proposed treatment, and an assessment of the risks that could be reasonably expected to be associated with it. The Bill also proposes that, before embarking on such a treatment, the doctor should have discussed it with the patient, the multi-disciplinary team responsible for the patient’s care, and with the Institutions Responsible Officer. The Bill emphasises that its provisions are solely concerned with the patient’s best interests.
The Bill as originally introduced into the House of Lords in May 2013, as a Private Members Bill by Lord (Maurice) Saatchi, and has become known colloquially as “The Saatchi Bill”. The Department of Health is currently consulting on a draft version of it5. I was originally sceptical about the need for the Saatchi Bill but I have been persuaded otherwise. First, it is clear from the comments of Lady Butler-Schloss4, as well as other legal authorities Lord Saatchi has consulted, that there are serious legal impediments in civil law to using therapeutic interventions that do not represent the current standard of care. Second, although Lord Saatchi’s original Bill was confined to patients with malignant disease this restriction has, rightly in my view, been removed in the current draft. There are, after all, many other miserable conditions for which we have no, or very limited, remedies. Third, I did not believe that his suggestion for approval by a multi-disciplinary team, alone, provided sufficient safeguards: the inclusion, now, of agreement by a doctor’s Responsible Officer provides me with re-assurance.
Despite my strong support for the Medical Innovation Bill there are important consequences for the professions when (as I hope) it becomes law:
1) Just because a particular intervention appears to have been effective in an individual patient it cannot be assumed that the results are generalisable. Further research in the form of one or more randomised controlled trials, or case series, will be necessary to establish its effectiveness. To take a recent example, a case report6 describing the apparently successful treatment of generalised juvenile pustular psoriasis with etanercept, requires confirmation before it can be regarded as the current standard of care.
2) There may be occasions when Responsible Officers wish to seek other advice before approving the proposed use of an intervention in accordance with the provisions of the Medical Innovation Bill. This will often need to be provided very rapidly especially when a patient has a life-threatening illness. The Academy of Medical Sciences, or some of the specialist associations such as the British Pharmacological Society, could have an important role here in offering a speedy advice service.
3) If the intentions behind the Medical Innovation Bill are to be fulfilled, NHS hospital Trusts, and their Responsible Officers, will need to look on proposals sympathetically. Anecdotal evidence suggests that too many Trusts are fearful of departing from the prevailing standard of care because of the possibility of litigation. The Bill should provide them with adequate reassurance.
4) It is essential that the results of using the Bill’s provisions, in individual patients, are placed in the public domain whether or not they have been successful. This would allow others not only to learn from such experiences but – especially for interventions that appear to have been successful – to undertake formal research.
I believe that the use of the provisions in the draft Medical Innovation Bill offer benefits to patients – especially those with rarer diseases – as well as to the furtherance of medical science. Subject to the responses to consultation it is the government’s intention to have it on the statute book at the earliest opportunity5.
1. Medicines Act (1968). www.legislation.gov.uk/ukpga/1968/67 (accessed 12.04.14)
2. McNair J. Bolam v Friern Hospital Management Committee (1957) 1 WLR 582. www.e-lawresources.co.uk/Bolam-v—Friern-Hospital-Management-Committee.php (accessed 12.04.14)
3. House of Lords. Bolitho v City and Hackney Health Authority (1998) AC 232. www.bailii.org/uk/cases/UKHL/1997/46.html (accessed 12.04.14)
4. Butler-Schloss LJ. Simms v Simms (2002) FAM.83 para 48. www.bailii.org/ew/cases/EWHC/Fam/2002/2734.html (accessed 12.04.14)
5. Department of Health. Legislation to encourage medical innovation. www.gov.uk/government/uploads/system/uploads/attachment_data/file/285272/9959-TSO-2901828-Legislation_to_Encourage_Medical_Innovation.pdf (accessed 12.04.14)
6. Fialová J1, Vojáčková N, Vaňousová D, Hercogová J. Juvenile generalized pustular psoriasis treated with etanercept. 2014;2:105-8. doi:10.1111/dth.12065.
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