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Integrating patient experience into research and clinical medicine: Towards true “personalized medicine”

We advocate science-based medicine (SBM) on this blog. However, from time to time, I feel it necessary to point out that science-based medicine is not the same thing as turning medicine into a science. Rather, we argue that what we do as clinicians should be based in science. This is not a distinction without a difference. If we were practicing pure science, we would be theoretically able to create algorithms and flowcharts telling us how to care for patients with any given condition, and we would never deviate from them. It is true that we do have algorithms and flowcharts suggesting guidelines for care for a wide variety of conditions, but there is wide latitude in them, and often a physician’s “judgment” still ends up trumping the guidelines. While it is also true that sometimes physicians have an overinflated view of the quality of their own “clinical judgment,” sometimes to the point of leading them to reject well-established science, as Dr. Jay Gordon frequently does, what I consider to be physician’s judgment is knowing how to apply existing medical science to individual patients based on their circumstances and, yes, even desires and values.

Indeed, if there’s one area where SBM has all too often fallen short in the past, it’s in taking into account the patient’s experience with various treatments. What got me thinking (again) about this issue was an article by Dr. Pauline Chen in the New York Times last Thursday entitled Listening to Patients Living With Illness. She begins her article with an anecdote:

Wiry, fair-haired and in his 60s, the patient had received a prostate cancer diagnosis a year earlier. When his doctors told him that surgery and radiation therapy were equally effective and that it was up to him to decide, he chose radiation with little hesitation.

But one afternoon a month after completing his treatment, the patient was shocked to see red urine collecting in the urinal. After his doctors performed a series of tests and bladder irrigations through a pencil-size catheter, he learned that the bleeding was a complication of the radiation treatment.

He recalled briefly hearing about this side effect three months earlier, but none of the reports he had been given or collected mentioned it, and once he had recovered from the angst of the emergency room and the doctor’s office visits and the discomfort of the clinical work-up, he didn’t give it more thought — until a few weeks later, when he started bleeding again.

By the time I met him, he was in the middle of his third visit to the hospital. “I feel like I’m tied to this place,” he said. He showed me a plastic jug partly filled with urine the color of fruit punch, and he described a post-treatment life marked by fear of going to the bathroom and discovering blood. “If I had known that my life would be like this after radiation,” he sighed, “I would have chosen the surgery.”

To this, I’ll add a little random bit of personal experience of my own. No, I wasn’t a patient who had to face something like this patient, but I do see something similar in my patients. Back when I was in my surgical oncology fellowship — and before that, in my general surgery fellowship — I was always taught that lumpectomy was preferable to mastectomy because it saves the breast and most women want to save their breasts. After all, lumpectomy plus radiation therapy results in the same chance of survival as mastectomy; so we should offer lumpectomy whenever tumor characteristics (the main one being size relative to the rest of the breast) permit it. Yet this assessment often neglects to acknowledge that, for some women, undergoing six or seven weeks of radiation is horribly inconvenient, and that there are often complications. It also often neglects to acknowledge that there is a price for saving the breast besides having to undergo radiation therapy: there’s the possibility of more surgery to achieve clear surgical margins, not to mention a higher risk of local recurrence in the breast. For some women, this latter possibility is a deal-breaker. Even though they acknowledge that their chances of survival would be the same with lumpectomy or mastectomy, the thought of an approximately 8% local recurrence rate eats at them to the point that they opt for mastectomy.

Then there is the issue of chemotherapy. We frequently recommend cytotoxic chemotherapy for women with relatively early stage breast cancer, even though the addition of chemotherapy in such patients only increases the chance of survival by perhaps 2–3% on an absolute basis, depending upon the tumor. Of course, as I’ve pointed out before, the benefits of chemotherapy are more marked in more advanced operable tumors, but in early stage tumors they are rather modest. This is therapy that causes hair loss, increased risk of infections, and can cause damage to the heart, but it is the standard of care. Most women are willing to undergo this sort of therapy, too; I can’t locate the study, but I’ve seen one survey where women respond that they would be willing to undergo chemotherapy for a 1% increased chance of survival.

The point is that these sorts of questions are value judgments that often depend upon what patients consider important. The patient described by Dr. Chen, for instance, would apparently prefer risks of surgery rather than peeing blood all the time and having to go back to the doctor’s office and hospital time and time again for this problem. Science can tell a physician and patient like this that radiation or surgery will produce an equivalent chance of surviving his cancer. It can tell them what the complications of each choice are likely to be, and what the odds are of each complication. That’s part of what I mean when I refer to science-based medicine. What it can’t tell the patient and doctor is which constellation of risks would be more easily bearable by the patient. The same is true for whether to choose mastectomy or radiation or whether to opt for chemotherapy after breast cancer. Science provides the numbers and the “price” of each choice, but it can’t — nor should it — tell the patient what to value. Moreover, what the patient values may not be what the physician values. As Dr. Chen points out:

Whether conducted at a laboratory bench or in clinical trials, medical research has long been driven by a single overriding goal — the need to find a cure. Usually referred to more modestly as a search for “the most effective treatment,” this standard has served as both a barometer of success and a major criterion for funding. Most published studies are marked by a preponderance of data documenting even minor blips in laboratory values or changes in the size of a spot of cancer or area of heart muscle damage on specialized X-rays. Some studies bolster the apparent success of their results with additional data on societal effects like treatment costs or numbers of workdays missed.

Few studies, however, focus on the patient experience.

She then refers to a study by Dr. Albert W. Wu, lead author and a general internist and professor of health policy and management at the Johns Hopkins Bloomberg School of Public Health in Baltimore published in the journal Health Affairs entitled Adding The Patient Perspective To Comparative Effectiveness Research. In this study, Wu et al argue for the inclusion of the patient’s perspective in comparative effectiveness research. What this involves is patient-reported outcomes. To illustrate the concept, Wu et al use this chart for patients with chronic obstructive pulmonary disease (COPD).

These sorts of measures are particularly appropriate for comparative effectiveness research (CER). For the reason, consider what CER is: basically CER compares existing treatment modalities already determined to be effective in prior clinical trials in order to determine which is more effective. Other important measures include cost-effectiveness. However, although some efforts go into assessing patient-reported quality of life outcomes of the sort listed above, all too often it’s hit-or-miss whether these sorts of measurements are included in clinical trials. One initiative that this article describes is the Patient-Centered Outcomes Research Institute, whose mandate is to:

  • Establish an objective research agenda;
  • Develop research methodological standards;
  • Contract with eligible entities to conduct the research;
  • Ensure transparency by requesting public input; and
  • Disseminate the results to patients and healthcare providers.

Wu et al suggest that the PCORI can only realize its potential if it supports initiatives that integrate measures of patient experience into not just research but into routine clinical care. A number of possibilities are suggested, including how to integrate general and disease-specific tools into clinical trials in order to measure patient-reported outcomes. Also suggested are various means of integrating these tools not just into clinical research but into routine clinical care, including using them in administrative claims data, linking this data to electronic medical records, and even promoting the collection of such data as being required for reimbursement.

One problem I can perceive immediately in trying to use the PCORI is that it has no real power. In fact, the health insurance reform bill known as the Patient Protection and Affordable Care Act (PPACA), which mandated the creation of the Patient-Centered Outcomes Research Institute, provides no power to it. Indeed, its main charge is to assess “relative health outcomes, clinical effectiveness, and appropriateness” of different medical treatments, both by evaluating existing studies and conducting its own. Even given that huge mandate, the law also states that the PCORI does not have the power to mandate or even endorse coverage rules or reimbursement for any particular treatment. Indeed, so toothless is the PCORI, at least in its present form, that it has been disdainfully described as being like the UK’s NICE but without any teeth, which is all too true. Basically, the law says that Medicare may take the institute’s research into account when deciding what procedures it will cover, as long as the new research is not the sole justification and the agency allows for public input. Moreover, if the political reaction to the USPSTF’s revision of the guidelines for mammographic screening last year is any indication, if politicians don’t like a PCORI recommendation, you can be quite sure that they’ll behave similarly. After all the ranting about “rationing” that was used to attack the PPACA, it was not politically feasible to make the PCORI a government agency or to imbue it with any real authority.

Politics aside, let’s get back to the sorts of initiatives suggested by Wu et al. One that in particular interests me is the concept of using patient portals to collect this information. Patient portals are websites that offer a variety of services to patients, including secure e-mail communication with the clinician, the ability to schedule appointments and request prescription refills, as well as the opportunity to complete intake and other forms that used to be completed on paper in the office. The authors propose using such portals to collect patient-centered quality of life measurements and give an example of how this might be done in the case of a hypothetical breast cancer patient:

In one possible scenario, a woman with breast cancer is being followed by an oncologist who would like to know how she is doing on the chemotherapy regimen she is receiving. The oncologist logs on to PatientViewpoint.org, enters the patient’s number, and orders the BR-23 Breast Cancer–Specific Quality of Life Questionnaire for her to complete online before her next visit. The patient receives an e-mail notification to do this, logs on to PatientViewpoint.org, and completes the survey.

The patient’s results are automatically calculated and are made available both on the website and within the hospital’s electronic health record alongside all of her other laboratory test results. At the visit, the oncologist pulls up the results and asks the patient about an increase in her depression scores. It would also be possible to aggregate all of the patient’s questionnaire results with those of other patients receiving chemotherapy for similar breast cancer cases and to use these data to help compare the effectiveness of different regimens.

Dr. Wu’s site is currently only set up to accommodate breast and prostate cancer patients, but it could be expanded. There now exist a large number of tools like the BR-23 to assess quality of life, and, with what appears to be the nigh inevitable infiltration of the electronic medical record into medicine over the next several years, integrating such tools into routine clinical care should become increasingly easy and inexpensive. On the other hand, one problem with such tools is that clinicians are already buried in “information overload.” Whether they would actually read and use the results of such studies outside the context of clinical trials is not assured, at least not if there is no incentive to do so. If this sort of approach is going to work, the government and insurance companies are going to have to pony up. Another problem is that a lot of doctors don’t like this sort of measurement. They consider it unscientific and “squishy” or they don’t know what to do with the information. Whether these attitudes will change or not as CER becomes increasingly embedded in clinical research is impossible to say.

Dr. Wu’s article leads me to reflect upon two things. First, it’s important to remember that the reason these “softer,” “squishier” measures are becoming more important is precisely because SBM has been so successful. Diseases that were once fatal are now chronic. A prime example is HIV/AIDS. Back when I was in medical school, HIV was invariably fatal. AIDS patients died rapidly — and in most unpleasant ways. Thanks to SBM, which developed the cocktails of antiretroviral drugs, HIV/AIDS has become a chronic disease, so much so that babies born with HIV are now approaching adulthood. What this success means is that, although not completely, by and large mortality is no longer the be-all and end-all of HIV treatment. Now, we are seeing quality of life issues coming to the fore. The same is true for some cancers, and it’s certainly true for diabetes and heart disease. As Wu et al point out:

Patient-reported outcomes directly support the primary goal of much of health care: to improve health-related quality of life, particularly for people with chronic illnesses. No one can judge this better than the patient. For example, the main objective of hip replacement surgery is to reduce pain and improve the capacity to get around. The main goal of cataract extraction is to improve visual functioning—that is, the ability to perform activities that require eyesight, such as reading, walking without falls, and working on a computer.

In addition, there are often trade-offs between the length and quality of life. Important considerations are the side effects of treatment of HIV disease, the temporary diminution of functioning after coronary bypass surgery, or fatigue resulting from cancer chemotherapy. Even for life-saving treatments, this kind of trade-off can influence a patient’s decision making among alternative courses of care.

Once again, these decisions and the trade-offs patients decide to accept should be informed by the science. The options presented to the patient and their cost in terms of potential complications and impact on the patient’s ability to go about his daily activities and in essence live his life must be based on science. However, that does not mean that the final determination will always be purely based on estimates of efficacy. If the patient decides, for instance, that the survival advantage that chemotherapy will provide after her breast cancer surgery is not sufficient to be worth months of hair loss, fatigue, and the risk of heart damage, then that is her choice. The key is that we as clinicians must make sure that she has accurate, science-based information upon which to base that choice. Informed consent must be based on sound, scientifically verified information. Anything else, such as the sorts of “informed consent” advocated by “health freedom” groups is in reality misinformed consent. It is our responsibility as science-based practitioners to do our best to make sure that the treatments we offer our patients are based in science and that the information about the relative benefits, risks, and costs about these treatments is also based in science.

The second thing that comes to my mind is the complete contrast between the sorts of efforts that Wu et al are undertaking and what purveyors of unscientific so-called “complementary and alternative medicine” (CAM) do. SBM is, through CER, undertaking systematic measurements of quality of life measures, and the use of genetic tests that provide information about prognosis and predict response to therapy, making its first real steps towards truly “personalized” medicine. Yes, these steps are halting — stumbling at times, even — but they are steps towards the day when SBM can offer patients treatment options based on science and personalized to the characteristics of the biology of their disease that are unique to them, all while taking the patients’ own values and desires into account. Contrast that to so-called CAM, where “personalized medicine” basically means making it up as the practitioner goes along, and I think you’ll see what I mean. Whatever the deficiencies and faults of SBM (and it’s impossible not to concede that there are many), SBM is far closer to true “personalized medicine” than any CAM, and it is using CER to come even closer still. CAM has nothing to compare.

Posted in: Clinical Trials, Politics and Regulation

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9 thoughts on “Integrating patient experience into research and clinical medicine: Towards true “personalized medicine”

  1. vexorian says:

    This is actually an OT post. But I felt that SBM should get information about an EU directive that – according to altmed people “bans” herbal remedies as of April 2011. I would link to news sites that speak about this directive, but they are all homeopathic / conspiracy theorist places which I do not intend to increase the visit count of. Since information in non-wacko sites is hard to find, all I seem to know is that it is an April 2004 directive that will finally come into place in April 2011. A non-nonsensical analysis of the directive would be nice to have.

  2. Thanks, this is a nice post.

    When I first started taking medication I wanted to get information about what it was like to take that particular medication. Patient inserts and medical drug reference books gave lists of side effects, which didn’t tell me anything. After a while I stopped caring what medication I was taking because it didn’t matter. Whatever it was, it was safe enough. Knowing more about the medication would not tell me more about the subjective experience of being a patient taking it. That would be something I’d have to discover for myself. I became one of those irritating patients who say “I used to take a white pill, but now I take two yellow ones instead. No, I don‘t know their generic or brand names. No, I have no idea what the doses are.”

    The internet is great. Crazymeds.us has been up since 2002 and gives exactly the kind of information I would have wanted back in 1996. Because I can look stuff up for myself, it now makes sense to know what my prescription is (and I do).

    I think patient experience has always been important to doctors, at least to the good ones. It’s just that now we have the tools to quantify it to some degree.

  3. “Patient portals are websites that offer a variety of services to patients, including secure e-mail communication with the clinician, the ability to schedule appointments and request prescription refills, as well as the opportunity to complete intake and other forms that used to be completed on paper in the office.”

    Yes! I just want to say how much easier this would make our (patient and parent’s of patients) lives. For people who have a lot of appointments, keeping track of appointments and treatment plans (meaning plans that I have hard copies of from various doctors) becomes quite a cumbersome task. To have access to it all and be able to fill out forms for upcoming visits would be quite helpful.

    And just think, no one would need to decipher my awful handwriting to enter my answers into a digital record.

  4. Charon says:

    If we were practicing pure science, we would be theoretically able to create algorithms and flowcharts telling us how to care for patients with any given condition, and we would never deviate from them.

    What this statement implies to me is that you’ve never done pure science. Oh, you may be correct that you’d theoretically (i.e., with arbitrarily advanced knowledge/technology) be able to do such things, but I’ve never seen any scientist do this in practice. Certainly not in my field (physics/astrophysics), which generally deals with much simpler things than people. We have no algorithm for exactly how every quasar works. Sure, there are similarities, but every damn individual one is different! Hey, just like people!

    I fully believe you that medicine is not pure science. But this is the worst explanation of that I’ve ever heard.

  5. David Gorski says:

    What this statement implies to me is that you’ve never done pure science.

    And you would be quite mistaken.

    But I’ll give you the benefit of the doubt and admit that I probably should have said something like a “mature applied science,” like engineering.

  6. windriven says:

    @Dr. Gorski

    “I probably should have said something like a “mature applied science,” like engineering.”

    I presume that it is the ‘mature’ that medicine is not. An applied science it most certainly is. And SBM is the indisputable path to maturity.

    @ Charon

    “But this is the worst explanation of that I’ve ever heard.”

    _|_

  7. David Gorski “But I’ll give you the benefit of the doubt and admit that I probably should have said something like a “mature applied science,” like engineering.”

    I’m just giving you a hard time, but, as a automotive engineer my husband is inclined to remind me that he is not a scientist, he is an engineer (although he does use a lot of algorithms). I believe his point is that the process often just comes down to ‘just try it and see if it runs.’

    He is very mature, though. :)

  8. anoopbal says:

    Hi David,

    I hope you are aware that evidence based medicine DO take into accountpatient preference, values and concerns. That’s part of the definition of EBM.

    SBM probably needs to include it in the definiton too.

  9. JMB says:

    @Charon

    I fully believe you that medicine is not pure science. But this is the worst explanation of that I’ve ever heard.

    Dr Gorski described how scientific knowledge would be incorporated into decisions and actions in the practice of medicine with his statement. He was not describing pure science. Algorithms and flow charts are technologies for translating results of medical science into medical practice.

    @anoopbal

    SBM probably needs to include it in the definition too.

    That’s included in the definition of medicine, at least for those that take the Hippocratic Oath. It doesn’t matter if it’s an EBM dogma, or SBM dogma, or don’t care about underlying scientific principles dogma. Medical school has already drummed it in your head as much as it can be drummed in. Differences in practitioners that persist reflect differences in their personalities, not whether they believe in EBM or SBM, or don’t care about the difference between EBM and SBM. But you are correct in that SBMer’s do have to reassure people that they are not advocating cold calculated treatment without empathy.

    I am concerned if an author makes a recommendation incorporating values in a decision analysis and passes it off as science. Science cannot tell you what your individual values are (even with fMRI). Science can determine the distribution and average of values of a selected population. Why would a patient accept a decision based on a group average of values, rather than relying on their own values?

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