The “Saatchi bill” will allow responsible innovation in treatment

In his discussion of the Medical Innovation Bill, Michael Rawlins [1] refers to Judge Butler-Sloss’s forceful criticism of the Bolam principle while she was president of the High Court’s Family Division because it leaves doctors open to legal action for negligence if they depart from what is regarded as “the standard of care”. We have direct experience of the adverse consequence of an important omission from Judge Butler-Sloss’s judgement.

We co-chaired the MRC Prion-1 trial steering committee at the time (17 Dec 2002) that the father of a young man with variant Creutzfeld-Jakob Disease (vCJD) appealed to the High Court for permission to seek treatment for his son using pentosan polysulphate injected into the brain. The father believed that the drug might slow his son’s decline because it appeared to have done this in an animal model of the disease studied in Japan. Judge Butler-Sloss granted permission for the father to seek treatment for his son [2], and similar judgements were made subsequently in respect of other sufferers of vCJD.

In making her ruling Judge Butler-Sloss suggested that treatment with penicillin might never have been attempted if the Bolam principle had been applied. What she failed to do was to require organised, prospective documentation and publication of the characteristics and progress of patients receiving pentosan polysulphate for vCJD, as had happened with the first series of cases treated with the penicillin [3]. The consequence of this important omission from her judgement has been that patients, their parents, and the people involved in the PRION 1 trial and other related research remained and remain unnecessarily ignorant about the possible benefits and harms of this proposed treatment.

This unsatisfactory state of affairs cannot be allowed to be a feature of any treatments that may become available under the provisions of the Medical Innovation Bill. Rawlins [1] notes that, ‘just because a particular intervention seems to have been effective in an individual patient it cannot be assumed that the results are generalisable’. We regret that he did not go on to call for proper, publicly available documentation to inform decisions made by other patients and those caring for them. Failure to make such evidence accessible was one of the factors that led to the TGN 1412 disaster [4].

Lester Firkins
Iain Chalmers
Co-chairs, MRC Prion 1 trial steering committee

[1] Rawlins MD. “Saatchi Bill” will encourage innovation in treatment. BMJ 2014;348:13.

[2] CJD case: the judgement. BBC News. http://news.bbc.co.uk/1/hi/health/2585049.stm

[3] Abraham EP, Chain E, Fletcher CM, Gardner AD, Heatley NG, Jennings AM, Florey HW. Further observations on penicillin. Lancet 1941;2:177-189.

[4] Chalmers I. TGN1412 and The Lancet’s solicitation of reports of phase 1 trials. Lancet 2006;368:2206-2207.

Saatchi law: Legal hurdles and Clinical irrelevance

The ‘Saatchi law’ is a wonderful legal initiative but it is unlikely to achieve its original aim of promoting innovative treatments. [1].

Firstly, on the legal front, it is likely to run afoul of the EU clinical trials directive, which in turn has been singularly responsible for the significant drop in number of academic clinical trials across Europe [2][3].

Secondly, there is a high failure rate in development of novel, innovative treatments. It has been estimated that only ‘’1 in 1,000 new compounds that enter preclinical testing ever make it to human testing and only 1 in 5 agents that enter human trials receive FDA approval’’. [4]. The high failure rate is not due to any legal or financial hurdles. It is just a sad fact that most novel drugs are not good enough to be used clinically. So it is indeed extremely unlikely that the proposed Saatchi law would promote new treatments. Even worse, it could end up potentially harming lot of patients by promoting use of ineffective new medications. In fact, the law could even be used to promote quackery and unscrupulous people have certainly used the legal loopholes to enrich themselves in the past. [5].

Hence, unlicensed medications, however innovative they are, should be properly tested in clinical trials (phase1, 2, & 3) to ensure scientifically credible information is obtained regarding their safety and efficacy. Clinical trials, with their ethical oversight and peer review process, should remain the gold standard, at present, for assessing novel and innovative treatments.

Thirdly, there is an important distinction between licensed and unlicensed medications. Licensed treatments are likely to have good safety data and hence use of licensed treatments for unlicensed conditions is certainly less risky. Indeed most pediatricians and many oncologists would attest that there is no clinical impediment in using licensed medications for unlicensed conditions, although in practice there is a financial impediment.

Clinically, at best, the proposed Saatchi law is likely be irrelevant but, at worst, the well intentioned legal safe guards proposed in the law would paradoxically end up curtailing the current widespread use of licensed medications for unlicensed conditions.

References:
1 Rawlins MD. The ‘Saatchi bill’ will allow responsible innovation in treatment. BMJ 2014;348:g2771–g2771. doi:10.1136/bmj.g2771

2 Sundar S. The law of unintended consequences. J R Soc Med 2013;106:303. doi:10.1177/0141076813496235

3 Gulland A. Number of global clinical trials done in UK fell by two thirds after EU directive. BMJ 2009;338:b1052.

4 Boklan J. Little patients, losing patience: pediatric cancer drug development. Mol Cancer Ther 2006;5:1905–8. doi:10.1158/1535-7163.MCT-06-0179

5 Holland JF. The krebiozen story. Is cancer quackery dead? Jama J Am Med Assoc 1967;200:213–8.