Medical Innovation Bill [HL]

Baroness O'Neill of Bengarve Excerpts

Friday 27th June 2014 Lords Chamber

[Baroness O'Neill of Bengarve (CB)]

My Lords, I declare an interest in that I am on the board of the Medical Research Council and chair its ethics, regulation and public involvement committee. Nothing that I say is said in the name of the Medical Research Council, but I believe that it will be compatible with the views that it holds.

I have great sympathy with the purpose of the Bill and agree that we need to ensure that, where permitted, those providing innovative treatments under safeguards should be required to complete and to make available the relevant data for the benefit of others with like conditions. I was therefore very encouraged to hear about the initiative for the registration of innovations, which may make a difference. That benefit may be knowledge of cure or improvement, but, equally, it may be knowledge that protects against treatment that would either exacerbate or not benefit—benefits go two ways in this.

However, I want briefly to point to some wider issues that lie in the background of this discussion. The days of the blockbuster drug may be drawing to a close. We are seeing the emergence of many more narrowly targeted drugs that meet the needs of smaller numbers of patients. This is the promise, but also the problem, of stratified medicine. It raises problems for the classical model of the randomised controlled trial. Such trials are expensive and take a long time. Where a drug or innovation may benefit quite small patient populations, it may be difficult to recruit a cohort of trial participants with the relevant condition. Where a market is likely to remain small, it may not be feasible for any pharma company to take the financial risk. We therefore have strong reasons to think about possible ways of making legitimate clinical trials more feasible and more flexible, while protecting patients and always acting within their consent.

I want to point to one particular sort of research for which we need to make room, and that is participant-led research. In participant-led research, those who suffer from a condition agree to make their data available for analysis. That analysis may reveal that some form of treatment benefits or harms. It is marvellously cheap as a way of doing it. As an example, I would cite the ALS lithium study conducted by users of the online platform, PatientsLikeMe. It was initiated by two advanced-stage patients from Brazil and the US who were users of the platform and who died before the conclusion of the study. A group of 149 ALS patients on the platform took lithium in order to test the findings of a small earlier study into its effects on disease progression and symptom alleviation. The PatientsLikeMe ALS study was completed over a period of eight months—note how short that was—and was published in Nature Biotechnology. It found that lithium had no effect, which was subsequently confirmed by standard clinical trials, but the innovative approach to establishing a result was much faster.

Participant-led research faces some difficulties. It may not be eligible for ethical review, not because one cannot consider the ethics of the matter but because there is, believe it or not, no principal investigator—so how does one do the ethics review? It may not be eligible for publication in medical journals; for example, because it has not been subject to ethical review. I do not think that such research should be subjected to less rigorous ethical or scientific review than other research, but nor do I think that it should be obstructed because it does not conform to the precise assumptions of the classical randomised control trial. Is the Department of Health considering a wider review of the fitness-for-purpose of present ways of regulating clinical trials so that they might be extended to cover a greater range of innovative research and innovation proposals?