The recent controversy about a new treatment for multiple sclerosis (MS) has raised questions about the role of patients and politicians in determining health research priorities.

In the past, the scientific community has generally determined what research is conducted.

However the public is increasingly demanding that they be involved in setting research priorities – the best ways of doing so are currently being debated.

The case of CCSVI and MS in Canada

In 2009, Dr. Paolo Zamboni, an Italian vascular surgeon, described a new entity in people with MS, which he called chronic cerebrospinal venous insufficiency (CCSVI). CCSVI is diagnosed with an ultrasound test and consists of abnormalities in the anatomy and blood flow in the veins of the brain and back. Zamboni claimed that CCSVI is a cause of MS, and that improving blood flow using a balloon or stent to remove vein blockages (known as endovascular therapy) improves MS symptoms.

Word spread within the international MS community about Zamboni’s theories and treatment, and patients began to report dramatic improvements after treatment for CCSVI. This led to positive media coverage and a growing buzz within the Canadian MS community about CCSVI and endovascular therapy. However, the vast majority of Canadian doctors who treat MS patients do not believe that there is sufficient scientific evidence to support Zamboni’s theories about CCSVI and MS. As well, provincial governments have been unwilling to pay for a procedure that might not be effective, and could potentially be harmful. Therefore, treatment for CCSVI is not available within Canada.

Many Canadians have travelled abroad to undergo an endovascular procedure at their own expense. They and their supporters have put pressure on their political representatives and governments to make this procedure available in Canada.

Initial response by Canada’s researchers

Public pressure focused a great deal of attention on CCSVI. In the spring of 2010, the federal Minister of Health, Leona Aglukkaq, asked the Canadian Institutes of Health Research (CIHR), the largest research funding body in the country, to assess the scientific evidence around CCSVI and make recommendations about conducting further studies. The expert panel’s report, released in August 2010, found that there was not enough evidence to proceed with clinical trials of treatment.

Reaction of the MS community

CCSVI has galvanized the MS community. Patients demanded research into this new theory of MS, and the MS Society of Canada directed their privately raised funds towards CCSVI research, and also set aside one million dollars to fund future clinical trials. The rapid response of the MS Society demonstrates how patients can directly affect the priorities of the organizations that serve their specific disease condition by raising private funds.

However, many MS patients were not satisfied with the position of the MS Society, because the Society did not advocate for an immediate study of endovascular treatment. They established their own groups to aggressively advocate for access to CCSVI treatment through social media, the conventional media and the political process. The impact of these advocacy campaigns was felt widely, with the House of Commons even having a debate about access to endovascular therapy and research funding for CCSVI. Some politicians also disagreed with the CIHR recommendations – for example the government of Saskatchewan issued a request for proposals for a randomized trial to evaluate endovascular treatment of CCSVI.

Who should determine priorities for public funding of health research?

The CCSVI controversy has raised interesting questions about the role of patients and politicians in determining health research priorities.

The concept of ‘equipoise’ has been used to help determine when it is ethical to conduct a randomized trial of a treatment in which some participants receive the treatment and others receive usual care. For equipoise to be established, first there should be good reason to believe that the treatment will work, because participants will be exposed to the risks of the treatment. Second, there should be enough uncertainty about the treatment’s benefits that it is ethical for some of the study participants not to receive the treatment.

Traditionally, decisions about equipoise have been made by experts. In the case of CCSVI, the CIHR Scientific Working Group felt that the risks of treatment, even within a clinical trial, could not be justified because it was not even clear that CCSVI causes MS. On the other hand, many MS patients have travelled outside the country at their own expense to undergo an endovascular procedure, and are demanding clinical trials.

Thus, in the case of the CCSVI controversy, it appears as if patients and experts have very different opinions about whether or not equipoise exists. Who should decide?

Experts and the public

Some say that because of limited research funding, scarce public funds shouldn’t be spent on studying treatments pushed by advocates that have scant scientific supporting evidence.

However, because their tax dollars are supporting publicly funded research, there is a growing movement to consult and involve patients and the public in research. David Sackett, a physician and leader in evidence-based medicine says that “research in general is becoming more responsive to patients” and that researchers are increasingly involving patients “when designing research and dealing with patient-led disease organizations and agencies.”  However, Sackett warns that while patients, the public and researchers all have “a strong legitimate right to make strong proposals about where research ought to go” they also “need to be careful that they are not in a situation where they have decided what the answer [to the research question] is before the answer comes” through the research process.

The James Lind Alliance in the United Kingdom  brings together clinicians, patients and their caregivers to articulate research agendas that are important to patients. They state that “the pharmaceutical and medical technology industries and academia play an essential role in developing new products. However, their priorities are not necessarily the same as those of patients and clinicians. For this reason many areas of potentially valuable research are neglected.” For example, they recently studied research in osteoarthritis, where 80% of the studies are related to pharmaceuticals. They found that patients and clinicians had relatively little interest in the usual studies of drugs for osteoarthritis of the knee, but wanted more rigorous studies of the effects of physiotherapy, surgery and educational and coping strategies.

There is no equivalent to the James Lind Alliance in Canada. Interestingly, a recent external review of the CIHR recommended that it increase methods for “public and patient participation/input in all its processes from prioritization, through advising on appropriate study endpoints and funding decisions to trial steering groups.” Ian Graham, the Vice President of Knowledge Translation and Public Outreach notes that for the CIHR, which is comprised of thirteen institutes, engaging citizens “is not a one size fits all approach, and that different institutes engage citizens in different ways” ranging from including community members on boards and committees, to engaging citizens in strategic planning and priority setting to having integrated knowledge translation funding opportunities that require researchers to engage knowledge users as equal members of the team. Graham notes that “there is sometimes a disconnect between the problems researchers focus on, and the topics or questions that patients want research conducted on” and the CIHR is making efforts to better engage with the public and patients, as there is a sense that “citizens and patients will increasingly be seeking to be engaged and participate in decision making at the clinical and research funding priorities level”.

The Toronto Health Policy Citizen’s Council is a research-led group of citizens who were brought together to deliberate on issues of health care priority setting. They met in November 2010 to consider whether or not treatment trials for CCSVI should be undertaken in Canada. The majority of citizens supported publicly funded trials, but a significant minority felt that public funds should not be spent on such studies until there is better evidence showing that CCSVI is more common in people with MS than those without MS. The full report of their deliberations is available here.

Recent developments

Nearly a year after their initial meeting, the CIHR Scientific Expert Working Group met in June 2011 to review the most recent evidence, including the results of a systematic review of studies evaluating whether CCSVI occurs more frequently in people with MS. This review found eight relevant studies, but the results were inconsistent among the studies. When the results of all studies were combined, CCSVI was more frequently found in people with MS than those without MS. However, because of the variation in results, the researchers (including Andreas Laupacis, one of the founders of healthydebate.ca) concluded that more high quality studies are required before it is conclusively known whether CCSVI is more common in people with MS.

After their meeting, the CIHR modified their earlier position, stating that they would support a “Phase I/II interventional trial”. However, details of the trial have yet to be released.

In late September, the government of Saskatchewan announced that their call for research proposals to study CCSVI treatment “did not result in a suitable research proposal.” Therefore, the government announced a partnership with a clinical study of CCSVI taking place in Albany, New York, and has set aside approximately $2 million to pay for about 90 Saskatchewan residents to participate.

What are your views about patient and public involvement in setting health research priorities?

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