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This is a long post, but it strikes deep to the core of the transformation underway in medicine, even in the science that drives medicine.

It appears the world is starting to change, in a very good way.

We’ve often written about the changing culture of medicine, as the professions begin to understand the value of the voice of the patient. This trend got a big boost in September, when the Institute of Medicine (IOM) published its new report Best Care at Lower Cost, which said, among other things:

Patient-Clinician Partnerships

Engaged, empowered patients— A learning health care system is anchored on patient needs and perspectives and promotes the inclusion of patients, families, and other caregivers as vital members of the continuously learning care team.

For years our movement has suffered a lack of credibility from people who said that if patient engagement was intellectually valid, academic medicine would be saying so. Well, THAT’s done.[1]

But change filters slowly through some organizations.  For instance SPM member Perry D. Cohen of the Parkinson’s Pipeline Project, about whom I’ve blogged on my own site, says he’s often been disregarded by the specialty that governs his disease (neurology) when he advocates that we should re-focus research efforts to be more what patients want – and to adjust science’s use of the scientific method to be more oriented toward patients’ goals.

Mind you, this critique is not some airheaded Jenny McCarthy anti-vaccine movement. Perry and peers are intellectually astute and rigorously trained – he himself has a doctorate from MIT Sloan, and many of his peers in the Parkinson’s community have had special training in partnering with researchers.

Something seems amiss when the scientists in a field are simply not interested in hearing what such patients have to say.

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But then there are bright spots, signs of real hope that medicine is starting to think like the IOM.

In the email exchange below, Perry had been invited to join the writing committee for a paper that has recently been submitted to a major medical journal on patient-reported outcomes (“PROs”).

In his comments, Perry wrote: [emphasis added]

Thank you for the opportunity to review the PRO article. My general assessment of the article is that it does a good job of distilling a vast amount of material on the subject. The article succeeds at covering the important technical methodology issues that need to be addressed to assure that research findings are derived from valid measures of these important variables.  I have nothing to add to this topic.

What I don’t see is how you intend to incorporate patients’ views and patient centered perspectives into the determination and assessment of PRO’s, especially where the views of activated patients differ from the views of research professionals.  I would like to see more explicit roles for patients as partners in the research process versus patients as research subjects or informants, in the identification of key variables and surrogate markers. By taking a patient centered approach you will be asking additional questions, such as:

  • How do you account for and control bias from using subjective PROs?
  • How do you value the risk to research volunteers (e.g. from placebo surgery or double blind trial designs) versus the benefits of the knowledge gained from research?
  • How do you strike a balance between the risk of false positives (targeted at .05 by default in the experimental model) versus the risk of false negatives (the worst outcome for a seriously ill patient) which are inversely related to each other?

Notice the incisive questions about pivotal details in the careful design of the study. Then he goes deeper into decisions about where to draw the line in study results – what do we decide is a success or failure? Who gets to decide what the priority is – caution, or seeking possible new understandings?

As was detailed in the article, the severity of illness is a key contextual factor in determination of the risk profile of a patient. How does the research process differ for different types of decisions – scientific/regulatory, patient care, business – in addition to individual differences in the acceptable risk and uncertainty about the expected outcome from a medical therapy.

While the scientific standard for risk in outcomes is generally 95% certainty, a good business risk may only require 1 in 3 chances for success. Likewise, individual patient preferences on risk choices also vary widely. So clarity about the type of decision is important to provide actionable information.

You may want to consider adding a section to the paper to explain how partnerships with patients are important for the effective use of PROs, and how partnerships with patient activists may require some adjustments  in the design of clinical research in order to accommodate patients’ interests.

How’s that for relevant, articulate and well thought out? Notice how clearly he points out the specific assumptions and unintentional blind spots in the original paper and study design. Students of the scientific method will see that these are intellectually valid points.

And the authors of this paper agreed: the response from the article’s lead author was heartening to me. He agreed, edited the paper, and acknowledged that American thinking sometimes lags behind the European view on this:  [emphasis added; edited into shorter paragraphs for readability]

Dear Perry and Everyone,

Perry’s comments resonate with me.  I added in language about the importance of patient engagement, activation, and partnership in identifying outcomes that are important and meaningful to people and designing quality assessment programs overall.

That’s partnering with thoughtful patients. He continues:

I think this is probably as far as it can go in the context of this paper, but I think these issues go way beyond that. In the U.S., these are areas that PCORI and AHRQ are quite focused on, and we are in some ways behind many of our European peers.  Some of these issues are truly methodological, but I think that the broader cultural issues and entrenched styles of conducting research are the real barriers. 

As far as heterogeneity and bias, these are issues addressed generally in both PRO and performance evaluation work, but are clearly challenges that merit better understanding and standard analytic methods, particularly as we work to broaden representation of patient perspectives in various initiatives.

Thank you very much for the comments, and I suspect I have missed some of the nuances.  I would welcome anybody else’s thoughts.

Notice that he acknowledges that the big issues aren’t pure science, they’re culture and “entrenched styles,” aka “We do it that way because that’s how we’ve always done it.”

Another author added:

Perry, I also thought you brought up some great points and am glad we were able to add some relevant language to our paper on this topic. I also agree with Ethan that it is important to explore other avenues to address the terrific patient centered questions you have raised.

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Even laggards are making steps in the same direction. 

That sequence of events stands in sharp contrast to the response (as an example) from neuroscientists in a conference where patient Perry played a key role.

The event as the 2010 international conference on the ethics and efficacy of the use of sham brain surgery as placebo control groups in the design of clinical trials for regulatory decisions. (“Sham surgery” is the practice of drilling holes in the heads of some Parkinson’s patients but not giving them any treatment. The idea is to control for placebo effect – but as Perry says, “The idea of placebos is that a sugar pill is inert and harmless. Brain surgery isn’t.” Talk about an ethical question! In all other countries, sham surgery is deemed unethical, and American patients agree, but American scientists don’t. Who gets to say?)

The initial request to sponsor the conference had come from Perry, to the Chair of the Recombinant DNA Advisory Committee of the National Institutes of Health.  So Perry was invited to speak to the conference, to present the patients’ perspective.  That was notable, great news, because despite being the ultimate stakeholder in all research, patients have usually been explicitly excluded from such professional, scientific deliberations. The grass roots patient advocate community came out in force to support Perry’s testimony and speak up in the Public Comment segment.

Patients were happy that they finally got a seat at the table. But: when it came time to report on the meeting – to write up what had transpired, for history to read – there were no actual patients on the writing team. Over a dozen authors, but no room for even a single patient.

Not even the patient who had proposed the conference in the first place.

I call this Paternal Quasi-Engagement: patients were deemed worthy of proposing the topic, and being heard, but not worthy of drawing any conclusions. Ahem.

An international group of patient advocate leaders responded with a letter to the journal (Lancet Neurology) to point out this important omission and to describe what was missed by ignoring this vital perspective. Excerpts:

Patients were represented at the  2010 meeting,  but the subsequent report has no patient author and the paper’s content thus fails to  represent the full breadth of opinion on this emotive issue. The views of  patients propounded at the meeting, when expressed on our behalf by the  scientific and medical authorship,  lack the attention to experience that can only be articulated by those living  with an illness such as Parkinson’s  disease. Of course, we understand  that scientific rigour should reflect the views of academia, but this approach should not be at the expense of making assumptions or assessments  that are not based on the reality of  living with Parkinson’s.

… Patients, as the ultimate stakeholders, are in many ways best positioned to discuss the ethical issues surrounding sham surgery and how the interface of risk, benefit, and development of techniques can be best assessed, in relation not only to science, but also to their lives. … the value of the conclusions drawn from the paper is not what it might have been had there been direct input from prospective recipients of sham surgery.

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In the discussion of the PROs article, the authors exhibit leadership and exemplify partnering with patients. My personal thanks to them, and I imagine most members of the Society for Participatory Medicine will be thrilled to see this sign of medicine shifting to be, as the IOM put it, ” anchored on patient needs and perspectives.”

Anchored on the patient point of view.

My personal thanks, too, to Perry for his years and years of advocacy. And my deep appreciation to the IOM, and the people involved in this article, for advancing the shift to participatory medicine – in which patients shift from mere passengers to responsible drivers of their health.

[1] Anyone want to argue with whether the IOM knows what it’s talking about? Here’s the team that worked on the report.


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