*Since writing this piece my understanding has changed and I now believe it contains fundamental flaws. Anyone who would like further clarification is welcome to e-mail me at hklaxnessat- yahoo.com*
Editorial
Mega-prizes in medicine: Big cash awards may stimulate useful and rapid therapeutic innovation
Bruce G. Charlton
Medical Hypotheses. 2006; 68: 1-3
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Summary
Following Horrobin’s suggestion of 1986, I argue that offering very large prizes (tens of millions of US dollars, or more) for solving specific therapeutic problems, would be an excellent strategy for promoting the rapid development of effective new treatments. The two mainstream ways of paying for medical research are funding the process with grants or funding the outcome via patent protection. When grants are used to fund the process of research the result tends to be ‘pure’ science, guided by intrinsic scientific objectives. Practical results, such as useful therapeutic advances, are a by-product. Patent-seeking research, by contrast, is more focused on technology than science. It seeks practical results; and aims to pay for itself (and make a profit) in the long term by generating a patentable product or procedure. Prize-seeking research is subject to different incentives and applicable to different situations than either process-funded or patent-seeking research. Prize seeking researchers have a strong incentive to solve the specified problem as rapidly as possible, but the problem may be solved using old ideas that are scientifically mundane or unpatentable technologies and methods. Prizes therefore seem to generate solutions which are incremental extensions, new applications or novel combinations of already-existing technologies. The main use of mega-prizes in medicine would be to accelerate therapeutic progress in stagnant fields of research and to address urgent problems. For example, medical charities focused on specific diseases should consider accumulating their resources until they can offer a mega-prize for solving a clinical problem of special concern to their patients. Prize money should be big enough to pay for the research and development, the evaluation of the new treatment in a clinical trial, and with a large profit left-over to compensate for the intrinsic risk of competing. Sufficiently large amounts of money, and the prestige and publicity derived from winning a mega-prize, could rapidly mobilize research efforts to discover a whole range of scientifically un-glamorous but clinically-useful therapeutic breakthroughs.
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In 1986 David Horrobin put forward the idea that large cash prizes should more often be used as an incentive to develop answers to important medical questions [1]. This proposal, still neglected, has much merit for stimulating therapeutic progress. I believe that offering very large prizes (e.g. tens of millions of US dollars) for solving specific clinical problems would be an excellent strategy for developing effective new treatments for medical conditions where mainstream science and commercial research and development (R&D) has so far failed.
There are two mainstream ways of funding science: funding the process with grants, and funding successful products by patent protection.
Research grants are used to fund the process of scientific research. Grant funded research tends to be ‘pure’ science, guided by intrinsic scientific objectives. Practical results, such as useful therapeutic advances, are a by-product rather than the primary goal. The incentive for doing good research is mainly the incentive structure of science: rewards of success include high status as a scientist and from this comes the perks of a triumphant career.
By contrast with pure science, applied research or R&D is supposed to solve problems. But, even when R&D ostensibly aims at useful practical results, if this outcome is neither enforced nor rewarded the ideal R&D strategy is to make enough progress to maintain funding, but never actually to solve the problem because that would put an end to income. In other words, grant funded R&D is implicitly supported to ‘do research’, and not to ‘solve problems’. Such R&D may end-up being merely ‘grant-seeking’ and generative of what amounts to low quality pure science. Arguably, much modern medical research falls into this category [2].
Patent-seeking research, by contrast, is a kind of risky investment – un-funded in the short term but intended to have a good chance of paying for itself and making a profit in the long term. The aim is to generate a patentable product (something novel, non-obvious and useful), then make money from the patent. Patent-seeking research is usually more like applied science or R&D than pure science, and includes most of pharmaceutical and much technological research.
Prizes are different both from process-funded and patent-seeking research. Prizes are essentially a way of funding research to solve specified problems in situations where the process of development is more a matter of R&D than pure science, but when the aimed-at product is unlikely to be patentable. Researchers have a strong incentive to solve the specified problem because receiving the prize is contingent upon achieving a solution. But prize-seeking researchers are free to solve the specified problem using ideas and approaches that may be scientifically mundane, technologies that are un-patentable, or technologies for which patents have expired.
Prizes tend to generate solutions which are incremental extensions, new applications or novel combinations of already existing technologies. The most famous example is the 1714 Royal Society of London 20 000 pounds Sterling prize for measurement of longitude to within five tenths of a degree [3]. This was eventually awarded to John Harrison in 1773, for designing a sufficiently accurate and robust clock. The delay in awarding the prize came from the fact that the Royal Society was looking for a ‘pure science’ answer to the longitude problem, while Harrison’s method was based on old science implemented by vastly-improved technology.
Indeed, this seems to be the usual way in which prizes are won. The general rule seems to be that prizes stimulate technology rather than science, accelerate R&D rather than generate paradigm-shifting breakthroughs. The prize winners for motorized flight, human powered flight, energy-efficient refrigerators and the ‘X prize’ for cheap space flight seem to confirm this pattern [4].
The main use of mega-prizes in medicine would be to accelerate therapeutic progress in stagnant fields of research, to address urgent problems, and to do so even when effective solutions are neither scientifically ‘sexy’ nor necessarily money-making. Useful therapies for a disease may be even duller than Harrison’s clock appeared to the Royal Society, and may not be patent-protect-able, yet nonetheless extremely valuable to suffers from the disease.
My suggestion is that those medical charities who concentrate on helping patients with specific diseases should not be plowing all their assets into research grants to support ‘pure science’ – which may or-may-not have a therapeutic payoff. Funding the process of research produces a measurable output which can, in the short term, be ‘spun’ into an illusion of therapeutic progress, yet progress in much of medical research remains sluggish or non-existent [2]. Such disease-focused charities should instead consider accumulating their resources until they can offer a mega-prize for solving a clinical problem of specific concern to their patients. The prize money should be big enough to pay for the research and development, the evaluation of the new treatment in a clinical trial, and with a large profit left over to compensate for the intrinsic risk of competing for a prize which may not be won. Mega-prizes would therefore need to range from tens of millions of dollars up to billions of dollars, according to the size of the problem.
For example, many specific types of cancer might be suitable for offering prizes. Shortly before he died of lymphoma, David Horrobin wrote an article in the Lancet calling for a more urgent and pragmatic approach to cancer therapy [5]. He speculated that there might already exist a variety of potentially beneficial (and non-toxic) nutritional and pharmaceutical interventions – supported by biochemical and animal experiments or case studies, but not yet refined or checked by clinical trials. Specific types of lifestyle advice and nursing care might also make a difference. Such interventions, if properly combined and sequenced, and tested in small and well-controlled trials, could rapidly and significantly help people with various malignancies. Yet such potentially beneficial treatments would probably not be patentable, and would also be regarded as scientifically mundane, so would be unlikely to be discovered with current mainstream research incentives. Mega-prizes might make the difference.
Perhaps sufficiently large amounts of money, and the prestige and publicity which would derive from winning a mega-prize, could mobilize research efforts to discover a whole range of scientifically un-glamorous but clinically-useful therapeutic breakthroughs. Such a strategy could provide realistic hope of speeding-up therapeutic advances for patients suffering from diseases for which, at present, little can be done.
References
[1] D.F. Horrobin, Glittering prizes for research support, Nature 324 (1986), p. 221. Full Text via CrossRef
[2] B.G. Charlton and P. Andras, Medical research funding may have over-expanded and be due for collapse, QJM 98 (2005), pp. 53–55. Full Text via CrossRef | View Record in Scopus | Cited By in Scopus
[3] Davis LN. Should we consider alternative incentives for basic research? Patents vs prizes. Paper from DRUID Summer Conference 2002. Available from: http://www.druid.dk/conferences.
[4] Davis LN. How effective are prizes as incentive to innovation? Evidence from three 20th century contests. Paper from DRUID Summer Conference 2004. Available from: http://www.druid.dk/conferences.
[5] D.F. Horrobin, Are large clinical trials in rapidly lethal diseases usually unethical?, Lancet 361 (2003), pp. 695–697.
