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Public has a right to influence research policy

June 29, 2010

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.

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11 Comments leave one →
  1. k. hutchinson permalink
    June 29, 2010 18:53

    Kathy Hutchinson ccsvi undoubtably needs to be studied…but while being treated the same as angioplasty was allowed without first doing long trials etc….first do no harm….save people’s lives first/during…Neuros who take this illogical damaging stance lose the respect of people who can think rationally”this is about the health of thousands..not my little brother’s pissing contest…please Drs….Grow up.
    33 minutes

  2. Jim permalink
    June 30, 2010 08:33

    I think you are missing an important point. In the case you cite, there are no other effective treatments for MS and the Zamboni approach has yet to be proven. MS patients are desperate for improvement and will take what many would consider unreasonable risks to achieve this. They are unlikely to be willing to wait for clinical proof and the current trials can only handle a small number of patients. Hence, there is considerable lobbying pressure and impatience. The danger is that this procedure (which is not without its own surgical risks), will become adopted by demand rather than efficacy. You might argue that at least offering something, even if unproven, is better than nothing but this is wrong-headed. Cancer patients are just as desperate and many go to great lengths and expense to seek out miracle cures. There are plenty of Charlatans more than happy to take their money and exploit their plight. Our primary defence against such quackery is insistence on robust proof. In addition, if the unproven procedure is covered by public healthcare, then it will be at the cost of some other proven treatment.

    The CMAJ article does not deny the right of patients to express a voice or that this voice is hopelessly tainted, but it does raise the spectre of healthcare instituted by loudness of voice. I’d also disagree with the idea that since this type of influence is already in place (through patient advocacy groups and differences in funding available) that it’s OK. It clearly is not, unless it is in the common good of society. It is a fact of life that some diseases have greater awareness than others, and consequently more attention and funding. But advocacy should not be confused with democracy. Compassion and emotion are important elements for consideration, but should not be the driving motivations for decisions that influence others. Dispassionate objectivity of physicians is essential in avoiding bias and relying on subjective parameters otherwise our currently precarious healthcare system would be long bankrupt. Our healthcare practice must at least allude to an evidential basis for decisions. Indeed, it is shocking how much evidence for best practice is currently unimplemented by many in the health professions. Finding a balance between providing access to a promising treatment and rigorously testing its efficacy is not only important, but a requisite for efficient and effective medicine. The CMAJ recognized the dilemma and brought it into light. For this they should be commended as discussion of the ethical and practical issues should not be behind closed doors.

    • Rob Annan permalink*
      July 2, 2010 07:59

      Hi Jim, and thanks for the comment, which is stimulating as always.

      I agree with your main points, and also applaud the CMAJ for discussing the dilemma publicly. My main point, which may not have been made clearly enough in my post, is (to quote Spider-Man creator Stan Lee) with great power comes great responsibility. The medical establishment has assumed the role of gatekeeper, deciding for individuals which treatments they should be allowed to access, decisions based on the best available research. The same establishment, however, is responsible for performing the research on which these decisions are based. This creates a particularly thorny problem when treatment is denied due to a lack of evidence. It is, in my opinion, unethical for the medical establishment to deny treatment due to a lack of research unless the same establishment thus dedicates itself to generating, as quickly and effectively as possible, the evidence to justify the decision one way or the other.

      Furthermore, though I am ambivalent on the issue, I’m a bit uncomfortable about using the power of authority to prevent individuals from being duped by charlatans. First of all, it is an incursion on individual rights. Leaving aside the separable question of public funding, I don’t like the idea of telling people what they can and can’t do with their money/self – deciding for others what is in their best interest. Second, I don’t think it works – look at the debacle around the vaccine/autism issue. Despite widespread medical opinion to the contrary, many people became convinced there was a link between the two. Unlike in the CCSVI case where physicians can refuse treatment, doctors couldn’t compel their patients to receive vaccination. The autism-link lobby grew in voice and influence until good research gained the upper hand and turned the tide. Strict authority on the matter wasn’t sufficient in that case, and I don’t think it will be in this case. Research and evidence are, as you say, the best defence against quackery.

      • Jim permalink
        July 2, 2010 11:41

        But there needs to be time to accrue the evidence. That is the problem. There will be quacks willing to offer MS patients a cure for a price. The medical profession is taking CCSVI seriously, but it cannot lower its standards in testing the procedure. For one, it will not be a cure-all, and therefore it is essential to determine who, if any, will benefit. MS patients deserve to get the best information and the best treatment, we can all agree upon that.

      • Claire permalink
        July 6, 2010 10:33

        De-lurking to comment here. I agree that the CCSVI hypothesis should be investigated, but I think it is unethical for physicians to offer a treatment/procedure that carries risk in the absence of sufficient evidence. CCSVI may be shown to be important in MS after more research, and it wouldn’t be the first time science has self-corrected. Right now all we have is a small, uncontrolled study that has been well-publicized.

        I’m glad you brought up the vaccine/autism controversy, but I disagree that the tide has turned on this issue. This is a major public health problem and has not gone away in the face of evidence. Meanwhile, science-based autism research is not getting the attention it deserves. I think this example serves as a cautionary tale of the way public pressure can have a negative impact on science and medicine.

        You say that “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 think that the scientific establishment has reacted very quickly to the CCVSI theory! There is a major study underway in Buffalo, at least one in Europe and more funding available for other studies (at the request of the public). The tricky nature of this work is illustrated by the fact that a trial in Stanford had to be stopped after a patient died. I think that scientists have to maintain high standards of evidence before recommending specific interventions, otherwise we are behaving unethically and lowering ourselves to the level of snake oil salesmen. If your argument is that we shouldn’t deny treatment unless we are actively studying that treatment, then I think that that is exactly what is happening in this case.

      • Rob Annan permalink*
        July 6, 2010 11:01

        Hi Claire, and thanks for your comments. You should de-lurk more often…

  3. July 6, 2010 14:41

    Hi! I’m still a little gobsmacked. After reading Claire’s comments I searched for CCSVI treatments and found a page listing a number of treatment centres in various countries, as well as some centres which screen only. I’m definitely in favour of a more flexible, responsive approach to research. While I’m horrified by this rush to offer a procedure presumably for commercial purposes with very little study, I think that people have ceased to believe that the medical establishment will respond dynamically. In short, there’s a failure to trust that the research will be done in a timely fashion which fuels desperation and opens the door to unscrupulous (or even some humane but dangerous) practitioners. I don’t think we’ll ever eliminate these problems but I do think that developing more open and responsive research initiatives will help. As a matter of fact, I found the swift response to run CCSVI trials quite encouraging.

  4. Jim permalink
    July 23, 2010 09:50

    Talking of the publics right, perhaps you could write up your views on the long form census issue Rob? This, to me, smacks of the very worst type of political intervention and the resignation of the head of StatsCan is an incredibly serious and worrying development. He did the honourable thing when faced with the ethical dilemma of accepting a clearly unsupportable contention by the government that abolition of the mandatory long form census could, for statistical purposes, effectively be replaced by a voluntary version. There appears no recognition or understanding of the idea of representation. Perhaps people believe that internet pop polls are meaningful? The Conservative spin doctors are saying that 49% of Canadians are against the intrusive nature of the long form but aside from the poll being heavily weighted by the loaded question (duh), it raises the issue of when should public opinion supplant expert scientific evidence (not one statistician has argued for the merit of a voluntary census and the experiences of such a census in the States showed it was invalid and a waste of resources).

    I’m ashamed of the debacle and the ignorance it suggests of our political masters. What’s next, Intelligent Design teaching made mandatory in schools? If you have no data, why worry about evidence. Bring back witching scales!

    • Rob Annan permalink*
      July 26, 2010 09:17

      Thanks for the comments and the suggestion, Jim. I’m on vacation in Nova Scotia, but took some time to get my thoughts on the matter off my chest. I feel better, and now I can stop nagging my family members about it so much…

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