The N=1 Problem of the Patient Representative

THE N=1 PROBLEM OF THE PATIENT REPRESENTATIVE

By Jessie Gruman

April 2, 2014

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?

Original blog post by Jessie Gruman. Updated by the GW Cancer Institute June 2016.