Dr. Ishak Mansi is professor in the department of medicine and clinical sciences and in the division of outcomes and health services research at UT Southwestern. He recently gave Grand Rounds and talked about studies looking at adverse events related to statins. Below is a short Q&A with Dr. Mansi as he talks about why he became interested in outcomes research and what constitutes a good study.
Q: What made you get interested in outcomes research?
I am a clinician, who practiced medicine since my graduation 33 years ago; therefore, as I interact with my patients, listen, and note their perspectives and concerns, I wonder about many of our practices and their actual impact on meaningful patients’ clinical outcomes.
We have to admit that we do “weird stuff” in the site of lay people: think about giving patients a strange looking pill that may cause symptoms and costs money, while they have no specific complaint (e.g. statins in primary prevention).
Q: Why are you interested in statin therapies and their outcomes?
I was exposed in my career to two extremes of statins utilization. The first was in Louisiana State University Health Science Center in Shreveport (LSUHSC), where patients rarely filled statin prescriptions. In LSUHSC, the institute did not have an outpatient pharmacy and our patients were too poor to afford paying for statins.
The second was in the Brooke Army Medical Center, where statins were available for free for all their population. Frequently, patients LDL-cholesterol levels were collected through electronic medical records, and were considered as quality of care measures for primary care providers. Therefore, physicians liberally prescribed statins to avoid being penalized as “poor-performers”. Living these two extremes challenged my thoughts and elicited my interest about the actual benefit/harms from these medications.
Q: What are the characteristics/features of a well designed study that looks at outcomes? (Ex. Large cohort, prospective versus retrospective)
Clinical trials are the gold standard in establishing medication efficacy (does this drug work?); but it should be followed by other studies that establish medication effectiveness (how well this medication actually works in clinical practice for a target population). Additionally, observational studies should guide the design of future clinical trials to ensure appropriate design. In general, prospective cohort studies are better than retrospective studies, but they are also much more expensive, time consuming, and require many years before obtaining results.
A well-designed study is the one that considers all known information about a problem and sincerely attempt to answer the question at-hand, rather than attempting to prove a “premeditated outcome” that serves a private interest.