February 2014 – by Dr. Muhammad Mamdani
While evidence-based medicine has revolutionized clinical practice by demanding the highest quality evidence to guide everyday practice, knowledge translation into clinical practice is often limited by the generalizability of evidence from typically highly restrictive and controlled ‘test environments’ to ‘real-world’ uncontrolled practice, often challenging implementation logistics (e.g. establishing complex stroke units across regions identical to those studied in clinical trials), and the costs of innovative technologies and practices. A critical issue drives this ‘disconnect’ between evidence-based medicine and knowledge translation – while highly trained clinician scientists are often generating scientifically rigorous studies without a clear understanding of principles in health policy, healthcare policy-makers are often generating policies without a clear understanding of scientific principles in clinical research. Increasingly, the scientific and health policy communities are working together to optimize the integration of best evidence into clinical practice through health policy. Buzz words such as ‘real world evaluations’ and ‘comparative effectiveness studies’ are commonplace in this often confusing intersection. In reality, the effective integration of clinical research into healthcare policy relies on a few fundamental principles: relationships, ‘practical’ scientific rigour, timeliness, and infrastructure.
Relationships: Understanding the perspectives of researchers and policy-makers is critical to effective integration of research into policy. Policy-makers are entrusted with the very difficult task of deciding between competing strategies to maximize societal welfare with a relatively fixed pool of resources. They are often approached by numerous groups with competing self-interests and seek unbiased, high quality information on which they can base their decisions. Further, they are under considerable pressure to manage very limited resources and make decisions in a timely manner. Since many policy-makers are not formally trained in scientific research principles, they often look to expert committees and individuals who they trust for advice. However, in generating policy-relevant research, both policy-makers and researchers need to invest considerable time in jointly developing research questions, agreeing to simple yet effective and understandable research designs, interpreting the output of the research, and developing policies based on the research conducted. Dialogue between the two groups must be brutally frank and honest to be most productive and must occur frequently and regularly. Through this relationship develops a fundamental requirement of an effective relationship: trust.
Practical Scientific Rigour: The two broad categories of clinical research are experimental studies (e.g. randomized clinical trials) and non-experimental studies (e.g. observational epidemiology). While experimental studies typically exclude the vast majority of patients typically seen in clinical practice in order to maximize internal validity, observational studies are far more inclusive but often criticized for being subject to considerable selection bias that threatens their validity. ‘Pragmatic trials’ have been emerging as a means of being inclusive through minimal exclusion criteria while maintaining randomization to minimize selection bias, however their implementation and interpretation can be challenging. No study design is perfect and multiple studies and study design types are often desirable for policy-makers. Further, policy-makers are deeply concerned about the financial and public health implications of their decisions. Ideally, the research portfolio for policy-makers should focus on their specific questions (as opposed to those of the researcher) and span clinical trials, observational epidemiology, health economics, and knowledge translation. An effective strategy is to develop a very brief (1-2 pages) lay-language study design overview for each study proposed and expected output in the form of mock tables and figures for policy-makers to review prior to the conduct of the research. Each study proposed must be scientifically rigorous, as policy-makers value the ability to refer to research published in reputable peer-reviewed journals as the basis for their decisions.
Timeliness: While many research studies take years to design and implement, the timeframe for policy-makers is often considerably shorter- often days to weeks. While these timeframes may be impossible for most clinical trials, it may be possible to conduct observational research and rapid reviews using a disciplined approach to research applied to readily available data sources. For example, an existing rapid response research group – the Ontario Drug Policy Research Network (www.odprn.ca) – typically turns around rapid response requests to decision-makers within 10 business days.
Infrastructure: Enabling infrastructure is just as critical as relationships and scientific knowledge in conducting policy-relevant research. The integration of sound science with highly efficient operations involves leading scientists who understand the needs of policy-makers working with high quality, service-oriented operations staff (e.g. analysts, health economists, epidemiologists, statisticians) and easily accessible informatics infrastructure. Often, establishing such infrastructure can be a costly undertaking. However, if properly managed, it can provide invaluable information for policy decisions that can translate into clinical and economic benefits that far outweigh its costs.
Conducting policy-relevant research can be extremely rewarding and have considerable impact on society if implemented effectively. Researchers interested in policy-relevant research can leverage the breadth of scientific expertise and world-class infrastructure at the HUB to advance the integration of clinical research into health policy – we’re here to help!
About the Author
Dr. Mamdani is the director of the Applied Health Research Centre (AHRC) at the HUB of St. Michael’s Hospital. He is also Professor in the Leslie Dan Faculty of Pharmacy and in the Dept of Health Policy, Management and Evaluation of the Faculty of Medicine at the University of Toronto where he supervises graduate students and an adjunct scientist at the Institute for Clinical Evaluative Sciences (ICES). Dr. Mamdani’s research interests include pharmacoepidemiology, pharmacoeconomics, and drug policy and he has published over 250 research studies in peer-reviewed medical journals.
View Dr. Mamdani’s publications on PubMed.