Mary A. M. Rogers - Comparative Effectiveness Research

Comparative Effectiveness Research

Mary A. M. Rogers

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Library of Congress Cataloging-in-Publication Data
Rogers, Mary A. M., author.
Comparative effectiveness research / Mary A.M. Rogers.
p. ; cm.
Includes bibliographical references.
ISBN 9780199986040 (alk. paper)
I. Title.
[DNLM: 1. Comparative Effectiveness Researchmethods. 2. Data Collectionmethods.
3. Evaluation Studies as Topic. 4. Research Design. W 84.3]
R853.C55
610.724dc23
2013029710

9 8 7 6 5 4 3 2 1
Printed in the United States of America
on acid-free paper

To my mother, DeLellis

Mary A. M. Rogers, PhD, MS, is a Clinical Epidemiologist who received her degree at the University of Washington in Seattle. She began teaching integrated science in the Peace Corps and has spent her career teaching and conducting clinical research. Dr. Rogers is a Research Associate Professor in the Department of Internal Medicine at the University of Michigan.

I knew a woman who died suddenly of sepsis. Thats nothing unusual. In the United States and the world as a whole, sepsis is one of the leading causes of death. But after she died, I took a retrospective look at her life, reconstructing the events that may have contributed to her early death. After she had delivered six children she experienced prolapse of her uterus, and therefore, with medical consultation, she made a decision to have a hysterectomy and oophorectomy. Once her uterus and ovaries were removed she received estrogen replacement therapy, which she took for many years. Estrogen replacement therapy slightly increased her platelet count and after several tests showing elevated platelet counts with an unknown cause, she was given the diagnosis of thrombocytosis. Standard treatment for this disorder at the time was hydroxyurea, which is an antineoplastic drug. Hydroxyurea not only decreases platelet counts, but tends to reduce white blood cell counts, placing her at greater risk of infection. So after years of taking hydroxyurea, she developed an infection at home, which spread to the bloodstream and placed her in the hospital, where she died. Throughout her life, she was a relatively healthy womannever had cancer or a stroke or a heart attack. But that initial decision she made and each decision along the way were consequential. The hysterectomy and oophorectomy led to estrogen replacement therapy, which led to elevated platelets, which led to a specific diagnosis, which led to hydroxyurea, which led to the infection, which led to death. The points along the way reflect decisions. Was that initial decision a good one? Well never know. Were there other options along the way that she could have taken? Perhaps. If we had better information from well-conducted studies about the risks and benefits of various therapeutic options and if these options were understandable and accessible at the time, might she be alive today? I think so.

Comparative effectiveness research is a mechanism to provide this needed information. Many people do not know the consequences of various therapies; once diagnosed with a disease, they often rely on professionals for guidance. The ultimate decision is most often made by patients and their families in consultation with physicians and other health care professionals. The choices can be quite dauntingparticularly for those patients with scant medical knowledge. Some rely on the advice of a relative or friend who may have had similar experiences. Some turn to media sources such as the Web to search for relevant information.

How would a patient make a good choice? One could imagine that we would gather the relevant facts, lay them all on the table, discuss, debate, and then decide. But where do you obtain these relevant facts? Asking your physician is sensiblebut where does each physician obtain this information? The basis of decision making, one would expect, would be driven by empirical evidence: Evidence derived from the results of good quality clinical research studies.

One would hope that there was some grand compendium of knowledge gained from all scientific research that answers every question that each patient has. There isnt. There are guidelines and reviews that compile results from scientific studies for specific conditions. There are trials of various treatments and therapies. There are well-respected medical journals filled with original research articles and reviews related to the detection and treatment of various conditions. But putting all the information together and keeping up-to-date with all the latest studies is a formidable task.

One may be surprised to learn how few studies have been conducted regarding specific therapies and their effects on a persons health. There are too few studies that answer common questions regarding available choices, the timing of such choices, and for whom such choices are relevant. An initial glance at the availability of medical information from media sources might lead one to conclude that we have a wealth of data regarding health and disease. This is true. But most often what one sees are narrative summaries and opinion pieces. Underlying such opinions and narrative reviews, how many original studies have actually been conducted to assess the effects of therapies on specific patient outcomes? Are there studies that indicate whether the therapy works in a wide variety of different types of people, or does it show promise for just certain individuals? It only takes one unexpected diagnosis for you, a family member, or a friend to realize that there are many particular issues regarding disease that we do not yet know. Was there a chance that, somewhere along the line, you could have done somethingor avoided somethingwhich could have prevented the diagnosis from occurring? Now that the diagnosis is made, which options are better, knowing that you cannot change your age, your genetic profile, and things that occurred earlier in your life? What is the best decision for me now?

Essentially, this is a call for actionable intelligence