“As you forecast, Curmudge, another week has passed and I am very much affected by my affliction. I don’t understand it. My physician diagnosed my illness and prescribed what she felt was the best evidence-based treatment. Quite honestly, she gave it her best shot.”
“Jaded Julie, it appears to be time for a second opinion. Consider your situation: (1) Your disease is serious and even life-threatening. (2) Your treatment doesn’t seem to be working or leaves you feeling worse than your disease. (3) Your diagnosis was difficult, performed by just one person, or was somewhat uncertain. Any one of these would lead us to consider a second opinion.”
“But won’t my physician be offended when I propose getting a second opinion?”
“If her primary goal is your good health and if she recognizes that no one can know everything, she will support your decision. That’s why there are specialists, subspecialists, and world-renown academic medical centers. If you look hard enough, you have a good chance of finding someone who can make a conclusive diagnosis and has successfully treated people with your disease. As we discussed back on June 18, 2009, errors in diagnosis do occur. When your disease is life-threatening, you want it to be diagnosed and treated by those who know it best. Remember, it’s your life.”
“Okay, Curmudge, I’m convinced. So please step down from your soapbox and tell me how to proceed.”
“Recall, Julie, that the objective of this series of postings was to teach you how to become a more informed and effective member of your health care team. So if you feel the need for a second opinion, begin by discussing it with your physician. Ask for her thoughts and guidance. Ask, ‘If you were in my situation, where would you go and whom would you see?’ Her answer might be someone across the street or across the country. She (or someone in the office) will tell you how to make the appointment and get your records sent to the appropriate destination.”
“A few weeks ago you quoted Meg Gaines as saying that one should ‘become an active patient ready to make your own way through your disease.’ What do I do if I feel that my physician’s suggestion for a second opinion is a bit conservative and I want to arrange the second opinion on my own?”
“Don’t go away, Julie, it’s possible. Let’s assume that you want your case reviewed by the specialists in an academic medical center and are uncertain where to go. Here’s one approach: Start with the U.S. News Best Hospitals rankings. Then select the specialty that fits your problem (you might not know the exact diagnosis, but you certainly know whether it’s Gynecology or Ophthalmology). A list of the best hospitals, in order, for that specialty will appear. Then follow your mouse for more detailed information.”
“I’ve found one whose reputation appears to be world class in my specialty. What do I do next?”
“The hospital’s Web site should tell you how to proceed. Oh, and by the way, you should probably check with your health insurance to learn if your second opinion will be covered. At some point you will need to authorize your physician’s sending your medical records—including labs, radiology, and pathology—to the second-opinion hospital.”
“This sounds like a big undertaking, Curmudge, especially if the medical center is a thousand miles away.”
“Some hospitals will evaluate your case on the basis of your records alone without your going there personally. The Cleveland Clinic (MyConsult) and Johns Hopkins (Remote Second Opinions) do this. Most commonly, their reports go to your personal physician. Partners Online Specialty Consultants is a physician-to-physician online second-opinion service provided by physicians affiliated with Harvard Medical School. The appropriateness of these programs may depend on the nature of your illness.”
“You wrote on May 13, Curmudge, about how we in the Fox Valley are blessed with quality medical care. In addition, there are two academic medical centers within the state and the Mayo Clinic only two hundred miles away. No wonder so many people around here simply die of old age.”
Affinity’s Kaizen Curmudgeon
Note: An observation on the practice of emergency medicine in a primitive location may be accessed via this link.
Thursday, October 21, 2010
Thursday, October 14, 2010
Evidence-Based Medicine--The Patient's Perspective 4
“I’ve forgotten, Jaded Julie, where our story ended last week.”
“I’m not surprised, Curmudge. You tend to forget everything except when it’s time to take a nap. As a patient with a life-threatening disease, I had learned how to learn about my disease by studying review articles, journal articles, and clinical trial results. I had become the local expert on me. So what’s next?”
“In a discussion with your physician you agreed on an evidence-based treatment plan. After following the plan for a couple of months, you were astonished to realize that your condition had not improved. In fact, you even felt worse.”
“What? Not improved! The treatment was evidence based. Clinical trial results indicated that I should have improved. What’s wrong? Did science fail me?”
“No, Julie, science is alive and well. Your problem might be that you are unique and not average. I wrote about this four years ago when I had a different name, Quality Curmudgeon, and you were not yet a member of the team. Readers of Kaizen Curmudgeon probably never saw my original note, so perhaps we should reprint part of the original posting.”
“Do it, Curmudge. By the time I have read it I might not feel cheated by evidence-based medicine.”
“Evidence based” is one of today’s health care buzzwords. If a clinician is faced with a patient whose disease may be mitigated by a medication shown to be effective in a large clinical trial, i.e., “evidence based,” he/she can confidently prescribe it and go on to the next patient. This sounds great, but there is more than meets the eye.
Let’s begin with the basics. Assume a pharmaceutical manufacturer’s preliminary studies have, in general, suggested that Compound A at a determined dosage is safe and effective against disease X. The final test is a clinical study in which large numbers of patients with disease X are recruited and divided randomly into two groups. One group is treated with Compound A; the other group is treated with a competing medication or a placebo. At the conclusion of the test, results from both groups are averaged. If the average outcome from the Compound A group is more favorable, the study is written up, peer reviewed and published. Treatment of disease X with Compound A becomes evidence based, and a new “blockbuster” drug is born.
So what’s the problem? The study has shown that the average outcome from a population of hundreds of patients was positive; but it has not answered the clinician’s most important question, “Will it help my patient?” The test population contained some patients with severe symptoms and some with mild symptoms, some with a host of other ailments and some who were otherwise healthy, and some young and some old. Many individual patients were helped by Compound A, but other individuals may have experienced no effect or might even have been harmed. These individual results were obscured by the magnitude of the test population, which on the average demonstrated a favorable outcome.
“I’m sure it won’t make you feel any better, Julie, but if you had been a participant in the trial of this treatment, you would have been part of the population that the treatment didn’t help.”
“Understanding the reason doesn’t make me feel any happier, Curmudge. So what do I do next?”
“I’m afraid that you are going to have to stay sick for another week, Jaded Julie. Then we’ll talk about a possible path forward.”
Affinity’s Kaizen Curmudgeon
Note: An observation on the practice of emergency medicine in a primitive location may be accessed via this link.
“I’m not surprised, Curmudge. You tend to forget everything except when it’s time to take a nap. As a patient with a life-threatening disease, I had learned how to learn about my disease by studying review articles, journal articles, and clinical trial results. I had become the local expert on me. So what’s next?”
“In a discussion with your physician you agreed on an evidence-based treatment plan. After following the plan for a couple of months, you were astonished to realize that your condition had not improved. In fact, you even felt worse.”
“What? Not improved! The treatment was evidence based. Clinical trial results indicated that I should have improved. What’s wrong? Did science fail me?”
“No, Julie, science is alive and well. Your problem might be that you are unique and not average. I wrote about this four years ago when I had a different name, Quality Curmudgeon, and you were not yet a member of the team. Readers of Kaizen Curmudgeon probably never saw my original note, so perhaps we should reprint part of the original posting.”
“Do it, Curmudge. By the time I have read it I might not feel cheated by evidence-based medicine.”
The Evidence Behind Evidence-Based Treatments
“Evidence based” is one of today’s health care buzzwords. If a clinician is faced with a patient whose disease may be mitigated by a medication shown to be effective in a large clinical trial, i.e., “evidence based,” he/she can confidently prescribe it and go on to the next patient. This sounds great, but there is more than meets the eye.
Let’s begin with the basics. Assume a pharmaceutical manufacturer’s preliminary studies have, in general, suggested that Compound A at a determined dosage is safe and effective against disease X. The final test is a clinical study in which large numbers of patients with disease X are recruited and divided randomly into two groups. One group is treated with Compound A; the other group is treated with a competing medication or a placebo. At the conclusion of the test, results from both groups are averaged. If the average outcome from the Compound A group is more favorable, the study is written up, peer reviewed and published. Treatment of disease X with Compound A becomes evidence based, and a new “blockbuster” drug is born.
So what’s the problem? The study has shown that the average outcome from a population of hundreds of patients was positive; but it has not answered the clinician’s most important question, “Will it help my patient?” The test population contained some patients with severe symptoms and some with mild symptoms, some with a host of other ailments and some who were otherwise healthy, and some young and some old. Many individual patients were helped by Compound A, but other individuals may have experienced no effect or might even have been harmed. These individual results were obscured by the magnitude of the test population, which on the average demonstrated a favorable outcome.
“I’m sure it won’t make you feel any better, Julie, but if you had been a participant in the trial of this treatment, you would have been part of the population that the treatment didn’t help.”
“Understanding the reason doesn’t make me feel any happier, Curmudge. So what do I do next?”
“I’m afraid that you are going to have to stay sick for another week, Jaded Julie. Then we’ll talk about a possible path forward.”
Affinity’s Kaizen Curmudgeon
Note: An observation on the practice of emergency medicine in a primitive location may be accessed via this link.
Evidence-Based Medicine--The Patient's Perspective 3
“Here we are again, Curmudge, back in Kaizen Curmudgeon. A week ago we thought our third discussion of evidence-based medicine would be too technical for this blog and would have to go in Curmudgeon’s Wastebasket.”
“Well, Jaded Julie, I asked Mrs. Curmudgeon to review this. If she can understand it, anyone…”
“Stop right there! One more word and you might not get supper tonight. So back to our topic, how I can learn more about my disease, become a more knowledgeable patient, and achieve a better outcome. I guess you’ve been reading the scientific literature for a long time, Curmudge.”
“Reading a lot and writing a bit for about 50 years, Jaded Julie. But clinical research is different from the industrial type, and the layperson needs some guidance in order to learn from it. When we last talked about this, you were playing the role of a patient with a disease who had learned how to use the major secondary sources of information such as Cochrane Reviews, emedicine, and clinicaltrials.gov. Today we are going deeper into the subject. Oh, and one thing further. If anything in your reading leads you to suspect your diagnosis, share it with your physician. There’s not much value in a patient’s studying a disease that she doesn’t have.”
“So here I am, a patient with the full text of what looks like a valuable article in hand or on my monitor. How do I proceed?”
“The sections of articles in most disciplines follow a similar order dictated by the journal: Abstract, Introduction, Experimental Methods, Results and Discussion or Outcomes, Conclusions, and Literature Cited. What you should learn in each section is described in Gaeta and Nagurney’s Evaluating the Literature. Your first objective is to decide if the article is really pertinent to your interest; that should be evident in the Abstract.”
“But, Curmudge, what if the Abstract is a bunch of medical gobbledygook?”
“It shouldn’t be if the article is truly pertinent. Your previous reading of reviews and other secondary sources should have taught you a lot of essential terms. The Introduction will describe what is unknown about the topic and how the proposed research will fill in some of the gaps in our knowledge. From the Methods section you will learn which of the several types of experimental design was used. Common designs include retrospective (looking backward at patient records) and prospective (following subjects forward in time and collecting data as they are generated).”
“My guess is that the design will tell me a lot about the validity of the results.”
“It should, Julie. Let’s assume that you are looking at a prospective study of a treatment that might cure your disease. The findings should be more meaningful if the treatment groups were randomized with everyone equal at the baseline, double-blind with neither patients nor physicians knowing who received the treatment or a placebo, and accounting for all patients.”
“I’m still with your train of thought, Curmudge. The next stop should be Outcomes. All aboard!”
“Look at the figures and tables of results. You might not know all of the words, but an upward or downward trend means something happened; and it’s often a good something. In my earlier life I was sometimes able to interpret figures even if the captions were in a foreign language.”
“So then, how will I know that the study’s findings were not just by chance and that the treatment might really do me some good?”
“Julie, authors usually use statistics to characterize their results. The so-called p-value provides a useful guide for the reader. The smaller the p-value, the more significant the difference between results from the treated group and the control group. Look for p-values less than 0.05, or even better, less than 0.01.”
“Curmudge, you can’t imagine how delighted I am that you didn’t go any further into statistics.”
“Various organizations have adopted formal guidelines for evaluating the literature, including the evidence grading system from the Institute for Clinical Systems Improvement and the Infectious Disease Society of America—US Public Health Service Grading System for ranking recommendations for clinical guidelines. The latter system uses letter grades (A-E) for strength of recommendation and Roman numerals (I-III) for quality of evidence. You may have encountered these grades in your reading of review articles. In general, if you are interested in a specific intervention, look for prospective, randomized, controlled clinical trials.”
“You have made my brain very crowded, Curmudge, but I believe I can proceed with a little more confidence. Do you have any final words of wisdom?”
“Never final, Julie, but I do have two additional comments and one caveat. First, if you see an abstract to a promising article but can’t obtain the full text, see your medical librarian (Margo Lambert at St. Elizabeth Hospital or Michele Matucheski at Mercy Medical Center). Second, many widely used treatments have never been subjected to a controlled clinical trial and are not, strictly speaking, evidence based. They have been used successfully over time and represent the consensus standard of care; one might consider them to be ‘grandfathered.’ The caveat is that there seems to be nothing in the clinical trial literature about the eventual cost of a particular treatment or medication. So don’t allow your hopes to get too high. What does one do after learning that he can gain an extra four months of life at a cost of $93,000 per year?”
“Wow! That’s a problem in medical ethics that neither of us would touch with a ten-foot pole.”
Affinity’s Kaizen Curmudgeon
Note: An observation on the practice of emergency medicine in a primitive location may be accessed via this link.
“Well, Jaded Julie, I asked Mrs. Curmudgeon to review this. If she can understand it, anyone…”
“Stop right there! One more word and you might not get supper tonight. So back to our topic, how I can learn more about my disease, become a more knowledgeable patient, and achieve a better outcome. I guess you’ve been reading the scientific literature for a long time, Curmudge.”
“Reading a lot and writing a bit for about 50 years, Jaded Julie. But clinical research is different from the industrial type, and the layperson needs some guidance in order to learn from it. When we last talked about this, you were playing the role of a patient with a disease who had learned how to use the major secondary sources of information such as Cochrane Reviews, emedicine, and clinicaltrials.gov. Today we are going deeper into the subject. Oh, and one thing further. If anything in your reading leads you to suspect your diagnosis, share it with your physician. There’s not much value in a patient’s studying a disease that she doesn’t have.”
“So here I am, a patient with the full text of what looks like a valuable article in hand or on my monitor. How do I proceed?”
“The sections of articles in most disciplines follow a similar order dictated by the journal: Abstract, Introduction, Experimental Methods, Results and Discussion or Outcomes, Conclusions, and Literature Cited. What you should learn in each section is described in Gaeta and Nagurney’s Evaluating the Literature. Your first objective is to decide if the article is really pertinent to your interest; that should be evident in the Abstract.”
“But, Curmudge, what if the Abstract is a bunch of medical gobbledygook?”
“It shouldn’t be if the article is truly pertinent. Your previous reading of reviews and other secondary sources should have taught you a lot of essential terms. The Introduction will describe what is unknown about the topic and how the proposed research will fill in some of the gaps in our knowledge. From the Methods section you will learn which of the several types of experimental design was used. Common designs include retrospective (looking backward at patient records) and prospective (following subjects forward in time and collecting data as they are generated).”
“My guess is that the design will tell me a lot about the validity of the results.”
“It should, Julie. Let’s assume that you are looking at a prospective study of a treatment that might cure your disease. The findings should be more meaningful if the treatment groups were randomized with everyone equal at the baseline, double-blind with neither patients nor physicians knowing who received the treatment or a placebo, and accounting for all patients.”
“I’m still with your train of thought, Curmudge. The next stop should be Outcomes. All aboard!”
“Look at the figures and tables of results. You might not know all of the words, but an upward or downward trend means something happened; and it’s often a good something. In my earlier life I was sometimes able to interpret figures even if the captions were in a foreign language.”
“So then, how will I know that the study’s findings were not just by chance and that the treatment might really do me some good?”
“Julie, authors usually use statistics to characterize their results. The so-called p-value provides a useful guide for the reader. The smaller the p-value, the more significant the difference between results from the treated group and the control group. Look for p-values less than 0.05, or even better, less than 0.01.”
“Curmudge, you can’t imagine how delighted I am that you didn’t go any further into statistics.”
“Various organizations have adopted formal guidelines for evaluating the literature, including the evidence grading system from the Institute for Clinical Systems Improvement and the Infectious Disease Society of America—US Public Health Service Grading System for ranking recommendations for clinical guidelines. The latter system uses letter grades (A-E) for strength of recommendation and Roman numerals (I-III) for quality of evidence. You may have encountered these grades in your reading of review articles. In general, if you are interested in a specific intervention, look for prospective, randomized, controlled clinical trials.”
“You have made my brain very crowded, Curmudge, but I believe I can proceed with a little more confidence. Do you have any final words of wisdom?”
“Never final, Julie, but I do have two additional comments and one caveat. First, if you see an abstract to a promising article but can’t obtain the full text, see your medical librarian (Margo Lambert at St. Elizabeth Hospital or Michele Matucheski at Mercy Medical Center). Second, many widely used treatments have never been subjected to a controlled clinical trial and are not, strictly speaking, evidence based. They have been used successfully over time and represent the consensus standard of care; one might consider them to be ‘grandfathered.’ The caveat is that there seems to be nothing in the clinical trial literature about the eventual cost of a particular treatment or medication. So don’t allow your hopes to get too high. What does one do after learning that he can gain an extra four months of life at a cost of $93,000 per year?”
“Wow! That’s a problem in medical ethics that neither of us would touch with a ten-foot pole.”
Affinity’s Kaizen Curmudgeon
Note: An observation on the practice of emergency medicine in a primitive location may be accessed via this link.
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