Consumer choice and the regulation of medicine

James Gubb, 6 November 2008

This Tuesday, a forum at Civitas heard the ideas of Bartley J. Madden, an independent researcher in the US. His big idea is a dual-track system that aims to speed up the time from trial to licensing for new medicines. Topical, given the recommendation of Mike Richards’ review on top-up payments this week to consider how PCTs can be better supported to make decisions on funding off-label drugs, Madden argued that drugs that have passed Phase I safety tests should be subject to two regimes…

Alongside the standard testing regime, there would be a second track that would allow terminally ill patients and others to use not-yet-approved drugs with informed consent. Of course, this is already possible in the UK on compassionate and exceptional grounds, but PCTs have proved understandably reluctant to fund them. But, with an established dual-track system, an intriguing question arises if pharma were to do so in their place in order to help assist and speed up the clinical trial process.
Madden’s system would rely on knowledgeable consumers and doctors, primarily using an informed consent liability release and an online “Tradeoff Evaluation Database.” The database would publish real-time, continuous updates about the safety and efficacy of all experimental drugs. His presentation can be viewed here and his ideas read here.
Of course, as many delegates pointed out, there are problems. Safety is one – who would pick up the can in the case of adverse reactions to the drugs? And who would make the decision to discontinue treatment? People may be more existentially secure in themselves these days, but this typically makes them both more consumerist and more safety-conscious.
Another is that such a system could perversely delay or even stand in the way of innovation by big pharma as – not taking the form of a randomised control trial – the observational data it generates may well be excessively negative. Numbers needed to treat are typically high for most drugs; particularly those that may only be effective in very specific cases. To be a useful tool any “Tradeoff Evaluation Database” would have to be sensitive to the individual and accurate. This is likely to be difficult; ineffectiveness can be molecular, down to incompetence on the part of a physician, or down to the patient not taking the drugs properly.
What, also, would such a system do to the doctor/patient relationship? Would we end up with overt consumerism and the phenomena of doctor shopping? Another point worth considering is the reverse, that of tacit coercion. How strong is the patient’s mind at the time of terminal illness? And, if the illness isn’t terminal, should we be exposing people to this level of risk?
Specific to the UK, most, in any case, would say the significant and frustrating source of delay is not in the trial process, but subsequent health technology assessments carried out by NICE. All are points that would have to be addressed.
That said, many people, particularly when faced with death or prolonged pain or disability, may be acting quite rationally in being willing to take major risks to improve their health. So long as they are kept fully informed as new findings emerge, and so long as decisions are taken in accordance with the advice of physicians, is it not still reasonable to suggest they should be free to choose such drugs that have yet to be licensed?