A dispute between some researchers and the Canadian Institutes of Health Research (CIHR – Canada’s largest health research granting agency) about how many large clinical trials should be funded by the CIHR has recently gone public. The scientists believe that more large clinical trials should be funded in Canada, and they should decide which ones.
I agree with them on the first point but not the second. The purpose of this opinion piece is to argue that the public should have a major say in which large clinical trials we invest in.
What are large clinical trials, why are they important and why should Canadians care? The short answer is that they change health care for the better. For a description of large clinical trials and how they benefit people, please click here.
I totally agree that Canada needs to fund more large clinical studies from the public purse – these studies change the way patients are managed for the better, and we currently fund too few. The pharmaceutical industry conducts excellent studies that have appropriately changed practice, but their financial interest means we can’t leave the playing field solely to them.
However, the number of really important large studies that could be done will always be greater than the resources available to pay for them, even if there is a considerable increase in funding from the federal government or the provinces. Who should make the decision about which studies should get preference?
The current answer to that question is “we the researchers”. The argument is that designing and conducting studies that will give answers that can be trusted is complicated, and only researchers can choose the best ones from among different studies. They are also experts in the disease being studied, and some are doctors or nurses who treat patients with the disease. Therefore, the argument goes, they know what is important to these patients.
However, I am not so sure. In addition to the upsides mentioned above, there are downsides to leaving this decision with researchers.
Many researchers are likely to consider the disease they are interested in as the most important. This isn’t a knock on researchers; it is a fact of life that all of us value the things that we are closest to the most highly. Researchers can sometimes get so focused on their research that they fail to recognize that it isn’t as important as they think it is. For example, one of the reasons some studies are very large is that researchers are trying to detect very small differences between two treatments, sometimes for outcomes that aren’t nearly as important as stroke. Those differences are sometimes so small that one can legitimately ask whether they are really worth detecting. Also, some large research studies don’t end up changing practice nearly as much as the researchers expected – could this be because they are too close to their particular scientific question to design their studies in ways that will maximally influence health care delivery and health policy?
An important part of deciding which large studies to fund is weighing the likely importance of the study results to patients and the health care system. This is incredibly difficult to do, but once studies are above an acceptable bar of scientific excellence, I would argue that this choice has little to do with science and much to do with values, fairness and legitimacy. The decision requires consideration of the current impact of the disease on patients, how many effective treatments for that disease already exist, the likely impact of the new treatment (if it works and doesn’t have many side effects) upon the lives of patients and their families, whether the disease has been “neglected” or studied a great deal, whether the treatment will help decrease the existing socioeconomic disparities in health, whether the health care system will be able to afford and accommodate the treatment, and many other factors. (By “treatment” I include preventive, health system and social interventions, not simply drugs and devices).
I think it is time to change the way we decide which large studies to fund. We should separate the assessment of scientific excellence from the assessment of the research’s importance to patients and the health care system. Scientists should continue to play the lead role in the former, and the public should take the lead in the latter.
I would suggest that all large studies go through a two stage evaluation process. They would first be evaluated by independent scientists to make sure they exceed a high bar of scientific excellence. Those that pass would then be evaluated by a committee consisting largely of members of the public whose main role would be to rank the various studies in order of importance to patients and the health care system. They would have access to independent scientific and clinical expertise if they have questions about the science of the studies and the diseases being studied.
This is a lot to ask of members of the public. However, they provide the funds for the CIHR through their taxes. They, their family and friends use the health care system. And if carefully selected, they would provide a broad and fair perspective of what is important to the public.
Lots of details would need to be sorted out for this idea to become reality. How would the public members be selected, should they be supplemented by some clinicians (nurses, doctors, other professionals) and individuals who manage our health systems (I think yes), should the committee be able to question the researchers applying for the funds in a face-to-face meeting (I think they should), and many others.
There will be many critics of this proposal. Some will argue the science is just too complicated for members of the public to understand. However, by ensuring that all research proposals considered by the committee are above a high bar of scientific excellence, assessing excellence will be less of an issue (although there will definitely be a learning curve for committee members). Others will argue that no matter how carefully they are chosen, committee members will always have biases in favour or against certain disorders. This is true of any member of any committee, and I am confident that fair minded members of the public can be found who will evaluate the proposals as impartially as possible. Some will worry that this is a huge burden to put on members of the public. While this is true, I am sure some members of the public will see this as an important contribution to society and one they are keen to take on. And some will argue this proposal will be expensive and will add yet another layer of review to an already onerous process. But given the expense of each large study and the importance of the decision, I think this extra step is worth it.
The CIHR is committed to a Strategy for Patient-Oriented Research that “…is about ensuring that the right patient receives the right intervention at the right time.” What better way to demonstrate its commitment than to involve members of the public in some of its most important decisions. It may be that the public should be involved in other decisions, such as the proportion of research funding that should be devoted to discovery or basic science research versus health services research versus the large clinical studies I have discussed here. Or, how much of the provincial and federal health care budgets in Canada should be allocated towards research to make the system better. However, because large clinical studies are meant to be of direct relevance to patients and the health care system, I see this as a very good place to start.