Structuring and Presenting the Patients’ Perspective at AMIA

PatientsLikeMe members share health data on the site adding their own individual-level health experience to a repository of structured outcome data. The result? An unprecedented data set that informs medical conversation not only within the patient community but also with the larger scientific one.

Organized Patient ExperienceEarlier this fall, the venue for this conversation was the annual meeting of the American Medical Informatics Association (AMIA). Typically, AMIA is a forum for medical researchers to discuss clinical-facing informatics projects like electronic medical records, doctor decision-support systems, and standards. This year, the event agenda included its first-ever panel on patient collaboration, with PatientsLikeMe presenting on how our members use informatics systems to spearhead original research.I presented a paper co-authored with Michael Massagli chronicling the activity on the ALS site regarding the site-based evaluation of Lithium. Questions about the paper were enthusiastic and challenging as medical researchers contemplated the full implications of patients conducting research outside of the healthcare system. The most provocative comment came from Danny Sands of Cisco who introduced the possibility that while he saw the value of PatientsLikeMe, we may also be “polluting clinical trials” – when patients with rare diseases take experimental treatments before being enrolled in (his) randomized clinical trials. My response was that he and others in the clinical trial world may feel differently if someone he cared for was diagnosed with ALS – a disease where patients have limited time to explore treatment options. As medical researchers, we should be proud of our patients who are taking an active role in their health care; I know I am.

The truth is, whether practitioners discuss it or not, people have long experimented with novel, off-label, and alternative treatments (some with and some without their physicians). Barring the few cases that are published in academic journals, these individual efforts only resulted in a tiny group of people learning anything from their experience. By organizing these individual efforts, PatientsLikeMe allows the data to be pooled and recorded systematically for analysis.

Of course, randomized clinical trials (RCTs) are the most rigorous means to systematize experimentation, but they have their draw-backs. They are expensive to run, time consuming (they take years, our study took months), and may be subject to more confounds than their organizers would like to believe.

While coordinated patient-led research on PatientsLikeMe is new, and as such presents novel challenges in its methods and credibility, we have now glimpsed its promise, its potential. Lithium was a first effort to study one treatment in one condition; it is just the beginning. There are many other treatments being used listed on the site by our patients that are not – for a variety of reasons – being studied systematically anywhere else. For example: Low Dose Naltrexone (LDN), Stem Cell Transplants in ALS, 4-Aminopyridine (4-AP), Botox injections to manage excess saliva, etc. These demand our attention.

Regarding the Lithium experiment, one patient described the situation better than I ever could in this forum post: “This is an amazing process.  Instead of sitting on the sidelines searching for promising research we are for the first time ever (from what I can determine) taking a group of people with a particular disease and taking an active role in researching a treatment.”

PatientsLikeMe’s members are not only learning how to best care for themselves, they are contributing their otherwise anecdotal experience to a body of data. Motivated by their own desire for better outcomes, patients are reporting directly to the website and building a body of evidence not being collected anywhere else. Together, with our carefully designed tools to capture, display, discuss and analyze this data, we are creating a repository of patient reported outcomes that will add patient data to evidence-based medicine and advance our knowledge.  Isn’t that what it’s all about?

PatientsLikeMe member jfrost

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13 thoughts on “Structuring and Presenting the Patients’ Perspective at AMIA”

  1. I thought Jeana’s post was very well done. From where I sit, doctors, scientists, researchers, etc., simply don’t seem to understand the urgency that one with an chronic illness feels.

    As an MS patient, I want to be functional and productive as long as I possibly can. I cannot begin to understand the panic that an ALS sufferer must feel. While MS is probably not going to kill me anytime soon, ALS does not have a long shelf life and patients want some help and they want it fast!!

    Jeana was doing a Vulcan Mind Meld on me when she wrote this piece. She put into words what I can’t because research methods and government bureaucracy are things that often turn me into a snarling, raving, crying maniac. And it’s hard to debate an issue when one is hysterical.

    In my opinion, Jeana represented the patient community here in stellar fashion.

  2. “Polluting clinical trials”? What a funny term. I agree with Jeana, having a chronic disease imparts a sense of urgency to your life that means you really don’t want to wait for a few years for a possible treatment, you want it now, or better yet, yesterday.

    Clinical trials are a distant hope, not a real world solution for many people. The ability to try something that may (or may not) work opens the patient’s prerogatives so much more than passively waiting.

  3. I am a patient with MS, and active in the PLM community. My time there invaluable. If it were not for this community, my quality of life would still be horrible, and I mostly house bound. But now, I am looking to get off disability and go back to work, as I have been offered a job. This, a feat I never thought possible. Now my unused Master’s Degree and training will be put to use. To PLM, thank you.

    What I have learned from PLM that has improved my quality of life, is, I now take a better drug for pain, spasticity, and fatigue. All drugs I am currently on for these symptoms, never were offered to me during my six years living with MS after diagnosis. This due in part to my inability to adequately describe my symptoms, and part due to my doctors inability to think outside the box from what he defaulted to using. But with more information, knowledge and understanding, communication on both sides improved and I got the help I needed. This only due to PLM. For it is there I learned to describe my symptoms better, and from there I shared with my doctor other treatments. With a little time, he became more open, and I communicated better.

    I also changed my vitamins, learned stretching exercises to help with pain due to spasticity, etc. I in turn shared experiences of low sugar intake,low intake of bad fats, rest, and managing stress, etc. As we all have shared the ins and outs of our daily routines, we make adjustments from what we learn on PLM. This greatly improves our quality of life as well. Add the support element, and the loneliness of living with a chronic disease lessens. That is priceless.

    If all this can happen in small part, improving ones quality of life, just imagine this process taking flight in the scientific community. The success of the individual then spreads to the whole of the group. We all, patients, doctors, and the whole medical community is lifted up. If there is fear because the process is outside the box, well I say there in lies the hope for progress. For those who are sick, the benefits far out weigh the risk. Because in the end, it is the quality of life and enjoying ones time with loved ones that matters. Medical advancement and all that goes into that, gives us the ability to enjoy our lives and our families.

    Thank you to Jeana, and all of PLM. To the doctors and scientists that join in this effort, I thank you too. I am living my life, and this in part to what all of you give me and those like me. So, to all involved in the process of collective learning, thank you.

  4. The outright stubborn old thinking ways of many researchers and doctors is quite frustrating to many people of chronic diseases, the openess of individuals with what works for them and alternatives available to others is invaluable to me at least.

    I was informed and joined a normal clinical trial, and through that information others did as well, would the trial have recieved as many people in it as it has without PLM? possibly but would have taken much longer to do so in my opinion.

    Many doctors get upset even if told of patients coming to this community, that aspect boggles my mind, most doctors spend 5-15 minutes every few months with patients unless serious issues arise, yet expect patients to sit there miserably in between and manage miserably? that is infuriating to me, I tell my doctors right off the bat I am in this community, if they have issue with that then they are not looking at my best interests imo.

    Many folks here have little to no support outside of online community, that in itself is beyond compare. Doctors and researchers need to quit being so self centered (yes I know with some that is impossible) and think of the best for the patient…you know the one paying the bill…

  5. Prior to joining PLM I considered myself a well-informed MS patient. I read voraciously and had connections with many other MS victims.

    PLM has made me realize how woefully uninformed I was. I have learned so much from other members-not only from their own experiences but from the multitudes of valuable links they provide on hundreds of topics. The contacts with members from other countries has been of irreplaceable value.

    Regarding clinical trials, they have their place in all things medical. However, I suspect that the majority of MS victims live in areas where participation in trials is not a viable option. Additionally, exclusionary rules prevent many others from signing up for a trial.

    PLM has provided us with a means to help ourselves, especially when our doctors are overbooked, overwhelmed, minimally knowledgeable, and firmly in the pockets of the pharmaceutical industry.

    I have watched as PLM members gain confidence, become assertive and proactive with their medical providers, and FIGHT to receive the proper care.

    I have also watched the PLM family members make huge changes in their lives–diet, exercise, vitamins, spiritual——–all because of the knowledge and support offered on this site.

    I believe that as sites such as PLM (none better, by the way) proliferate, the medical community is going to HAVE to take notice and will begin taking patients more seriously. Empowered and knowledgeable people can NOT be blown off!!!

    Thank you PLM, and thank you Jeana, for an accurate and insightful statement.

  6. It’s fantastic to hear from everyone on this issue and hear about your experiences. I still cannot believe how many people are changing the course of their care – getting educated about their condition, finding promising trials, learning how to get the best treatment, and yes finding a doctor that will help them do so. I think a lot of these individual efforts are invisible as they happen one person at a time – it’s great to learn about them here together to know how it can be done.

    And no matter what physicians and researchers do, the tide is not changing. Patients are involved. Providers and researchers can resist patient participation but ultimately, I think knowledge will be best served when researchers engage this growing group of informed and insightful individuals.

  7. Since I was quoted in Jeana’s post, I wanted to respond and provide some context for my remarks (which was not in the post).

    I understand and empathize with Jeana’s statements and the plight of patients with terrible illness. Although I work for Cisco, I am still a primary care physician (part-time)and care for many ill patients. I think they would all tell you that I am not lacking for empathy, and that I always encourage my patients to seek out information and community on the web. One of my patients, e-Patient Dave (, has blogged and spoken widely about how my encouragement for him to do so was a turning point in the course of his metastatic cancer workup and treatment. I am also a founding member of the e-Patient Scholars Working Group ( and am have just been elected to the board of the Center for Information Therapy ( Anyone who knows me knows tha I am truly an advocate of patient engagement.

    I applaud patient communities and am excited at the prospect of patients sharing information about the management of their illnesses to the betterment of all. I am also excited that PLM even has a research staff. My comments at the AMIA meeting do not negate any of that.

    Randomized controlled clinical trials are the best way to arrive at the true efficacy of most interventions. That said, they are expensive and difficult to do and sometimes are even unethical. For this reason, other study designs are used, with expressed limitations and are followed, when possible, to formal RCTs.

    When we skip the RCT, we often get misled about efficacy of an intervention. A recent example was when an RCT disproved the supposed benefits of hormone replacement therapy in post-menopausal women that we learned from cohort studies.

    Although I welcome patient-initiated research, we must be careful about calling the studies done through patient communities “clinical trials” for the following reasons:

    1. There is no objective assessment of clinical endpoints, which can mislead us.

    2. Interventions can cause harm as well as benefit. Sometimes there may be subjective improvement but actual harm being done (due to disease progression or toxicity). A clinical trial tracks both objectively, and safety issues are (usually) identified by a safety monitoring committee without a vested interest in the study outcomes. In patient initiated studies we do not have this safety monitoring.

    3. The reason that RCTs are blinded (so that subjects do not know if they are in the intervention or control group), is to avoid them perceiving a benefit just because they want there to be a benefit (the placebo effect). That will then bias the results towards showing a positive impact when none exists and therefore giving us an inflated impression of the impact of the intervention.

    4. Once patients have been exposed to an intervention, they often do not qualify for inclusion in an RCT. In the case of common conditions, like diabetes or heart disease, this is not a problem. In the case of a more rare disease it can be impossible to conduct an RCT, which would be devastating to the advancement of medical science, and could lead to continued ignorance of the real impact of interventions. Moreover, prohibiting these patients from participating in RCTs would be disadvantageous for them, as well.

    I find the work of patient communities like PLM fascinating and important for hypothesis generation. I also understand that desperate patients want answers and do not want to wait for well designed clinical trials to be performed. I am not suggesting that this work should not be done, I’m only concerned about our being comfortable accepting these trials as strong evidence of efficacy. These studies can lead to important hypothesis generation. These communities can also be tremendous sources of subject recruitment for RCTs.

    The purpose of my probing at AMIA (and of this post) is to educate the community about the importance of formal clinical trials and the role and limitations of patient community run trials. I think PLM and other communities would do a great service to their communities by doing the same.

  8. I’ll heartily verify Danny’s statement about how patient-centered and empowering he is. I experienced it years before I got sick, it played out from the first moment of bad news, and has continued since then.

    At the same time, I have to say I have multiple, complex concerns about the process by which trials are conducted *and reported* to us, and the ensuing questions about whether we the patients can count on them as the best possible pathway to saving our butts. (N.B.: I did not just say I don’t like clinical trials! I’m pointing to limitations of the system, not saying it’s trash.)

    In my recent post about evidence-based medicine I said “On the fringes of medical knowledge, lives are at stake and medicine doesn’t have the answers yet. What do you do?”

    The e-patients white paper mentions Danny’s work. It also mentions “the lethal lag time” – the lengthy period (more than ten years!) from conception of a research project through its execution and publication. One source said it’s 17 years from conception until findings are actually in use in doctors’ practices. Many many people can die while waiting for that.

    That’s also reflected in the new book Anticancer, which I read recently. A patient at a breast cancer conference was reported to have stood up at a microphone and said “If we wait for you epidemiologists to decide what’s what, we’ll all be dead!” In context she meant that we need to know what we can do now, and frankly if the establishment doesn’t have answers for us, we’re not going to sit around doing nothing.

    Same for me. As I’ve discussed with Dr. Sands, having responded well to an extreme treatment (in a clinical trial), I’m now upgraded to where I only have a 50/50 chance of relapse in my lethal disease. So am I sitting passively, waiting? No, I’m doing what I can to improve my odds, through diet and anything else I can find that even MIGHT improve my odds.

    And then there’s the reality that clinical trials are extremely costly so a trial never even happens on something that doesn’t have big money behind it. (This is not a complaint against companies in general – I’m just talking about a limitation of clinical trials.) As Anticancer points out, raspberries have clearly been shown to have anticancer properties, but try to find RCTs about their effects. (That’s especially ironic because unlike most new potent treatments, raspberries aren’t likely to lead to long-term side effects.)

    The importance of that issue is shown by the very existence of PLM. If commerce could be counted on to produce things like PLM, it wouldn’t have needed to arise on its own, out of the patients.

    Finally, Chapter 5 of the e-patient white paper is titled e-Patients as Medical Researchers and talks about groups of patients collecting their own data. I presume that if that paper were written today, PLM would be a prime specimen. (And remember, it’s Danny’s team who wrote it.)

    All in all, while I fully agree with the value of a good RCT, I myself would not hold myself back from something I believed was good for me, just so I might be a purer specimen a few years down the road.

    —Mind you, this is never an issue with a good clinical team. At one point I was asked if I was *willing* to participate in a different trial, but I was clearly informed that doing so would limit my later options in x, y, and z, and was completely given the choice. Similarly, long ago Dr. Sands told me, before I ever got sick, that a particular screening test was an option for me but it really wasn’t very accurate and could lead to unnecessary treatments, so it was up to me.

    Yet just last night someone on my ACOR (patient community) mailing list said that she felt that her husband’s [famous] cancer center had been more interested in keeping him as a clean specimen than doing anything possible to keep him alive. I’m glad to say I never felt that way. (Mine is Boston’s Beth Israel Deaconess, where Danny practices.)

    All I can say is, power to the people – the patient people. Let’s just do everything in our power to educate ourselves and be proactive in creating the best possible odds for ourselves. To me that includes understanding both the value and the limitation of clinical trials – and open conversations like this.

  9. Thank you for your comments Danny and Dave. First off, I want to say I respect Danny’s work as a patient advocate and thoughtful physician. So, I just wanted to clarify a couple of things. I didn’t understand the comment of “polluting clinical trials” as a reflection of the empathy of a physician to individual patients or a reticence towards patients locating information or support online. Nor, would we describe the investigation on PatientsLikeMe as a RCT or say that RCT’s are not valuable. I heard this comment as well, I think a little tongue and cheek but reflective of a larger concern coming out of the clinical trial research world. Danny just happened to be testing that larger view to a patient-centered panel.

    I heard solely Danny’s last point from his comment above – a hint of a deeply felt and understandable anxiety that patients, if they begin to take off-label drugs of their own initiative, will not be available as test subjects.

    And I do understand that concern. Facilitated by the Internet, an increasing number of people with the interest/resources/wherewithal to find new treatments and with the willingness accept risk, will experiment on themselves – especially when there is no trial available. But, I also think this concern is problematic.

    Can we really argue for patients to hold off treatment in order to remain eligible for a future possible clinical trial? Instead of viewing this phenomenon as a threat, I think we at PatientsLikeMe see it as an opportunity – a challenge to revisit and revise medical research methods as patient-centered.

    The question for me/us is where do we go from here? How can patients and researchers take advantage of the efficiency of patient-led work to create new knowledge? How can patients and medical researchers best coordinate efforts, analyze the data, and interpret the results? What are the possibilities and the limitations of this work? These are open questions.

    While we are at the beginning of this effort, we have promising early results and the next generation will be better. The goal is to design new experimental patient-centered research methods such that the resulting data will be informative – as well as patient-centered, nimble, efficient, ecologically valid, and inclusive.

  10. Great dialogue and an important issue for patients (I know if I had a life-changing illness I’d be looking up every trial in the world to see if I was eligible!). Danny is a well-known patient advocate and his reputation speaks for itself; that said, if a physician with his appreciation for the value of online communities has reservations then we obviously still have further to go in engaging people in the debate.

    Something that hasn’t been mentioned yet is just how few patients, even at specialist centers, are eligible for traditional trials. A recent study of the leading ALS centers showed that only 1 in 4 patients were ever enrolled for a clinical trial (Bedlack et al 2008). There are a number of studies cited within that paper that point to an enrollment rate of less then 5% in cancer patients.

    So if you are not eligible for a trial, what are you to do? Remain a “clean specimen” for a small chance of getting on to an RCT (after which you’ve got a 50/50 of being on placebo of course!)?

    We fully appreciate the difficulty, the complexity, the passion and the energy that goes into setting up and executing a clinical trial, and perhaps if we can do a better job of referring on those patients who we know will be eligible, we can avoid “polluting” these trials. But we also have an equal if not greater level of responsibility to offer opportunities to the vast majority of patients who will never be asked to take part in research studies. I firmly believe that research participation is an intervention in itself, particularly in serious conditions like ALS, and it’s something we should be able to offer more patients.

  11. Paul brings up an excellent point. Although some of the low enrollment is due to knowledge and availability (knowing a study exists and having access to it) it’s also due to stringent enrollment criteria, so that we know we are studying subjects that are as similar to one another as possible. This makes the results more valid (but at the expense of generalizability to broader populations).

    So we certainly need a way to reconcile this dilemma. I don’t have the answers. One thing is certain: in addition to providing information to people with serious illness, we must teach them how to find and enroll in relevant clinical trials, and help them learn about the advantages and disadvantages of all forms of clinical research.

    Every clinical research methodology has it’s advantages and disadvantages (validity, generalizability, bias, cost-effectiveness of conducting trial, etc.). I need to consider study design when deciding whether a particular study influences my care of patients, and patients should consider this when deciding whether to start an unproven treatment (as well as the risks and benefits of participating in a trial). This requires a fair amount of sophistication, but it is incumbent on us to help educate patients–as physicians, support communities, or patient website creators.

  12. I originally posted this on PLM, but it has come to my attention that maybe it needs to be here as well.
    The article was wonderful. It stated many of my thoughts about the value of PLM very well.

    In response to people like Mr. Sands, I would say that while rigorous clinical trials are necessary, they also remove options and control from patients. For example the Fingolimod trial has restrictive controls on what patients can have taken in the past and currently and also on patient profiles. This removes many people from the pool of subjects and removes this as a treatment for many many more. I was not eligible for a study because I do not have a complete family medical history.

    While I understand that this is how trials work, it is hard to be removed from a treatment option entirely. If I had a rapidly progressive disease, I would be more than willing to flout conventional studies and try just about anything. The establish research methodology (and its practitioners) need to understand that for some people there is not enough time to wait for the establishment to get together a study and recruit people (which may not include them).

    There is also the fact that the established research community is not interested in many of the treatments (like Lithium for ALS) because the drug is old and off patent. There is no one willing to spend the thousands on a study that at best (if it revolutionized treatment and everyone ended us using it) could net them pennies. They also tend to think very narrowly and pass many treatment ideas.

    I think PLM allows people to find others like them who are willing to advocate for themselves and butt heads with the medical community if necessary. It has made me far more willing to take an idea to my doctors and at least have them consider it seriously. I can’t force them to do anything, but because PLM has made me my own health advocate, neither can they force me to do anything.

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