Public has a right to influence research policy

Newly discovered treatments offering miracle-like cures have long held an understandable attraction for desperate patients and those who love them. Whether peddled dishonestly by snake-oil salesmen of years past, or trumpeted with the best of intentions on today’s blogs and news sites, there has always been a hopeful audience of patients desperate for treatments for myriad health problems. Though research often reveals the ineffectiveness of these “cures”, this research takes years to complete, if it happens at all. Meanwhile, patients clamour for these treatments, spending money and hope on these untested – and potentially harmful – approaches.

Thus, there is often a tension between patients desperate for treatment and physicians, researchers, and policy makers who must balance the risks and benefits, as well as allocate limited health and research resources. These issues have most recently come to a head in the debate surrounding angioplasty as a treatment for multiple sclerosis. The benefits of this approach are based on a single study performed by Dr. Zamboni and colleagues, who suggest a novel link between MS and chronic cerebrospinal venous insufficiency (CCSVI), which can be treated with percutaneous angioplasty – a relatively minor procedure. Though Dr. Zamboni’s suggestive and unexpected results have yet to be confirmed in more rigorous trials, MS patients and their supporters are demanding access to the procedure amid breathless media reports. Given the novelty of the idea that CCSVI and MS are linked, and considering the small, uncontrolled cohort of patients in Dr. Zamboni’s study (65 patients), many researchers and policy makers are stressing the need for more evidence before deciding whether to support the procedure.

Today, the Canadian Medical Association Journal (CMAJ) has added to the debate with an editorial urging the creation of a new, more responsive approach to initiating and supporting clinical studies in response to promising therapies (.pdf here, Globe and Mail article here). Arguing that “good health policy decisions should not based on hope and desperation”, the editorial also suggests “scientists and skeptics should avoid dismissing novel ideas prematurely.” The authors despair at the lack of regulation for non-pharmaceutical treatments, noting that rapid and widespread adoption overwhelms the ability to measure and evaluate treatment effectiveness and safety. However, the need for research shouldn’t prohibit access to promising treatments. Instead, the authors argue that innovative treatments be made available to patients, but only within the context of research and evaluation programs that will be able to effectively monitor them.

The authors, though, miss the mark in their exhortations to policy-makers, researchers, and physicians to resist public pressure to fund specific projects. They suggest: “Failure to do so will leave our academic institutions and research community repeatedly at the mercy of advocacy campaigns and decisions based on political expediency and opportunism.” There are two problems with this. First, it is naive to suggest that current research funding decisions are not already based on “political expediency and opportunism”. From breast cancer to alternative energy, lobby groups exert considerable influence on government research funding priorities. Second, though the authors argue that patients should “have their views represented” when deciding where to allocate research funds, they are also patronizingly exhorted to “insist on evidence”, implying that their opinions are based on little more than desperation. The outmoded distinction between dispassionate, ivory tower academics who can see research issues clearly and without self-interest, and desperate, ignorant patients has been abandoned across health research. Patients have a vested interest in supporting promising research with the best likelihood of success. Furthermore, researchers are not immune from self-interested, conservative opinions about where to allocate funds. Finally, those making demands are not simply patients, but taxpayers and charitable contributors whose money literally supports the research. Why shouldn’t they have a strong and loud voice in where that money goes?

Initiating widespread clinical trials for every proposed treatment is clearly impractical, and peer-review should be an essential step in deciding where to allocate scarce funds. A balanced approach is clearly necessary. Physicians may well “have a duty to keep unproven therapies from clinical use”. The corollary, though, is that unproven therapies must therefore be proved or disproved by the research community. It is not enough to stand and block the gate and then urge the public’s confidence in our medical opinions – denying treatment due to a lack of evidence is indefensible unless we are willing to go and get the evidence to decide one way or the other.

I concur with the need to develop the infrastructure to institute clinical trials more rapidly and effectively. Indeed, clinical research is generally underfunded in this country. I think, too, a process for launching fast-track clinical trials for novel therapies is a good one, but should certainly involve patients’ groups. Being responsive to public interest is essential to maintain the public’s support, and should be a crucial part of researchers’ social responsibility. Ultimately, researchers should be working to build bridges between the clinic and the public, not walls.