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The N=1 Problem of the Patient Representative


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What can we learn from an experiment conducted on a single person? That is, when the subject population (N) is a single person, aka N=1? How and how much do such findings contribute to knowledge about the experimental intervention? How relevant are results to other patients or populations or diseases? In assessing what is known about a phenomenon, how are these findings treated in comparison to studies with 30 or 200 or 60,000 subjects?

Questions like these are being raised by personalized and precision medicine researchers and clinicians when they aim to customize decisions and treatments to the unique genetic profiles and histories of individuals.

However, the same questions can be asked about the representation of individual patients on advisory, governance, proposal review and priority-setting committees for health care and research institutions.

The vast majority of patients who step forward to serve in these roles represent the equivalent of N=1. We bring to the discussion our own experience, which may be a lifetime of taking care of a child or ourselves with a severe chronic condition; a few months of recovering from an acute illness; an excellent hospital stay; a tragic, messy hospital stay; or a few years of being treated at the same clinic. While most of us encounter a cascade of events throughout each of these scenarios, what we bring to the table in the end is our experience through the lens of our own unique attitudes, beliefs and histories.

For patients, just managing to snag a couple of chairs at these deliberative tables has been a big challenge. We've heard comments like: "The whole enterprise is there for the patients' benefit, right? Clinicians and the health care industry have patients' interests at heart. We can represent them."

With the muscle of the federal government (through regulations of the Centers for Medicare and Medicaid Services and provisions in the Affordable Care Act), PCORI and NIH, and the persuasive efforts of philanthropy (the Robert Wood Johnson Foundation in particular), the demand for patient representation on a variety of health care and health research groups has increased.

But merely occupying those patient-designated chairs is not enough. Now that we have a place at the table, we need to look realistically at the value we add. This N=1 issue is not insignificant in terms of our own expectations as well as the expectations of those who extend the invitation.

The limits of my ability to represent all patients – or even a subset of us – was painfully demonstrated to me a few years ago, after I had completed treatment for my third primary cancer, when I set out to write a book about what to do when you receive a serious diagnosis. With all that cancer and all those chronic conditions the treatments caused, I figured I was a real expert on health care. I thought I'd just interview a couple people for a little added color and then write the book. But after three interviews, I stopped in my tracks. I was shocked at how much I didn't know about the range of knowledge, experience, attitudes and behaviors others have about their health and about how health care works. I was mortified by my easy (lazy?) assumption that because I'm a pretty average person, I could generalize from my own experience.

I ended up interviewing over 200 people – patients, former patients, caregivers – about their experiences with health care during those difficult few weeks following a diagnosis. It completely changed the book I had planned to write and it completely changed my understanding of what it means to be ill and to seek good health care in the U.S. today.

And it has left me with little patience, on one hand, for professionals who expect patient representatives on deliberative bodies to generalize from their own experience, and, on the other, for patients who believe that all patients are like them and think that "If I can challenge my doctor's advice, ferret out a better treatment than he recommends or wear stiletto heels to my chemotherapy session, so can any patient."

With regard to those who invite our participation expecting direction-changing insights: Calm down. You don't have that expectation for any of the professionals on the group. Our experience, regardless of how exotic it may be to you, represents one legitimate angle that adds to the whole discussion, just as the evidence from an N=1 trial adds incrementally to knowledge of a phenomenon.

A few observations:

  • A single patient representative on a group is simply not sufficient. Two or more patients are needed – both for our ease and to capture even a modest range of variation in experience.
  • It is helpful if the question that our experience can illuminate is clear to us.
  • Get over this idea that our participation on deliberative bodies is "inspiring" or "courageous." We are colleagues with legitimate expertise and knowledge which, like you, we have chosen to contribute.

With regard to those patient representatives who assume that their experience and abilities do not differ from other patients: My expertise about my experience – what it took for me to make this decision or undergo that treatment or manage this disease – is unique. Most of us have no idea how widely we vary in every aspect of our health and health care. But our individual experience – assuming it is relevant to the purpose of the group – is sufficient and will add value to any thoughtful discussion.

A few observations:

  • When we volunteer to represent the patient perspective, our own experience and our perspective is the expertise we bring to the discussion, in the same way a nurse brings her experience caring for patients and a researcher brings her experience of conducting studies. Our expertise is no less legitimate and valuable than that of professionals.
  • It is difficult to sit in front of a group of professionals and say "I don't know." But not knowing something can be important information to other group members who may assume that everyone knows this.
  • It is difficult to resist generalizing to all patients from our own experience. Many of us really don't know how unique we are, or we forget that many people who are ill feel really sick and don't have the energy or zip to participate in their care. Listening to others talk about their experiences might give you a sense of this.

Efforts are currently underway by PCORI to develop research skills among patients so that they are able to participate in the grant review process. This work extends the pioneering efforts of the Department of Defense and the National Breast Cancer Coalition that have been successful in building a cadre of potential patient reviewers and patient advocates. While these skills are critical for some forms of patient representation, none of these approaches offer the equal opportunity for patient representatives to gain critical knowledge about the range of experiences we – infants, kids, teens, adults, older people, rich, poor, insured, underinsured, Vietnamese, Puerto Rican, educated, non-literate – face in finding health care and making good use of it.

The current N=1 contributions of patient representatives are probably good enough for this early stage of broad participation in deliberative health care bodies. Are they good enough for the long-term?

More Blog Posts by Jessie Gruman

author bio

Jessie C. Gruman, PhD, was founder and president of the Center for Advancing Health from 1992 until her death in July 2014. Her experiences as a patient — having been diagnosed with five life-threatening illnesses — informed her perspective as an author, advocate and lead contributor to the Prepared Patient Blog. Her book, AfterShock, helps patients and caregivers navigate their way through the health care system following a serious or life-threatening diagnosis. The free app, AfterShock: Facing a Serious Diagnosis, offers a pocket guide based on the book. | More about Jessie Gruman

Tags for this article:
Medical/Hospital Practice   Patient Engagement   Jessie Gruman   Communicate with your Doctors   Participate in your Treatment   Health Care Quality   Inside Healthcare  

Comments on this post
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Carolyn Thomas says
January 1, 2014 at 11:59 PM

"Volunteer" is the operative word here, I think. As a women's health activist, blogger and speaker, I'm frequently asked to participate on advisory boards, focus groups and other meetings organized by our local "Patient Voices" government health care initiative. I don't doubt the sincerity of organizers of these projects in wanting to have a patient at the table (yes, even for the potential N=1 contribution), but I've also been asked to participate by some whose purpose I suspected was to be able to check off the "patients included" tickbox that's part of this trend.

The bigger difference is that the nurses, doctors and hospital administrators sitting around the same table are getting paid to participate. In some cases, their day jobs require them to participate in such projects no matter who's sitting alongside them at that table.

But unlike these health care providers, I and other involved patients - aside from minimal travel reimbursements - are not paid a penny to participate. Many (if they are still able to work) must even take unpaid leave from the workplace to attend volunteer training, unlike the health care providers for whom training is considered part of their paid career function. And those who are too ill to work are still asked to volunteer their time willingly to these projects as if the privilege of being invited is reward enough despite ongoing health issues.

It seems to me that there's a far bigger matter at stake here other than patients merely "snagging a couple of chairs at these deliberative tables." As you wrote so succinctly in your essay "Do Experts Value Patient & Family Input?", patients care less about "the back-office machinations of physician practices or hospitals or the organization of care delivery except insofar as it affects our ability to get what we need in a timely, safe, cost effective way."

And at the same time, most of us certainly do not care about being merely a token patient at the table if our participation is neither valued nor fairly reimbursed.