In the United States, a rare disease is defined as a condition that affects fewer than 200,000 people. There may be as many as 7,000 rare diseases. The total number of Americans living with a rare disease is estimated at between 25-30 million. That’s around 8% of the population.
Patient participation in rare disease research, both wet and dry (in a lab with benches and with computers) is, frankly, rare. Of course, patients are subjects of research, but that’s not the kind of participation I’m talking about. I’m referring to patient involvement in 1) setting priorities, 2) study leadership and design, 3) improved access to clinical trials, 4) preparation and oversight of the information provided to participants, 5) post-study evaluation of the patient experience, and 6) the dissemination and application of results. Read More
My recurring mind loop these days is non-traditional patient, non-traditional patient, non-traditional patient. I heard it repeatedly while attended a one-day symposium, Putting Patients at the Center of Research: Opportunities for Ethical and Regulatory Oversight at
Harvard Law School’s Petrie-Flom Center. See a great report written by Andy Oram about the symposium here. The symposium showcased a PCORI (Patient-Centered Outcomes Research Institute)-funded study about patient involvement in research in non-traditional roles (not the subject*). The study actually focused almost exclusively on Institutional Review Board (IRB) perceptions of patients in non-subject roles. Interesting focus since the role of the IRB is to protect patient rights in research studies as subjects, not other roles.
*Please note: Subject is a loaded word for some. They say participant rather than subject, a more egalitarian term. I’m sticking with using subject because I’m introducing the label of Participatory Research. I don’t want to confuse the issues.
No one’s ever accused me of being a traditional patient and I’m not defensive. Right:) You know I’m heavily involved with PCORI whose reason for being is to fund research that matters to patients and will benefit patients. It’s odd that a PCORI-sponsored study would label anything a person does who is not an academic and not a subject of a research study as non-traditional. The roles the study refers to as non-traditional are membership in the research team as an investigator, advisor, consultant, recruiter, or disseminator. It seems that the study started with a bias when they called other roles, nontraditional, rather than, say, non-subject roles. It didn’t call researchers who include patients in non-subject roles, non-traditional researchers.
My patient/caregiver activism rests on a foundation that patients and caregivers should have a seat at the table for governance, design, operations, and learning of healthcare policy, planning, delivery, improvement, and research. It makes sense that much of the research industry feels like a fish out of water with patients in their midst. Perhaps labeling (non-traditional) is a reflection of their acute discomfort with other. We call people of the Navaho nation whose ancestors lived in the continental US before the Puritans, American Indians. We call people who emigrated from China during the California Gold Rush, Chinese Americans. Yet, I’m white, first-generation American. I’m not called Dutch/German American, just American. Perhaps when many researchers think patient, they think someone wearing a hospital gown with their butt crack bare, not skilled, insightful, hardworking, curious, passionate people like themselves. Other.
As a reviewer of PCORI funding requests and co-chair of an Advisory Panel, I’m fortunate to be part of a leading edge of culture change in the human research industry: Participatory Research. I have seen research teams with patient/caregiver stakeholder Investigators and Advisors paid on equal footing as the academics. I’ve even seen respite care budgeted for carees of caregivers, so they could free themselves to participate in any role. Culture change seldom occurs by waving a magic wand. Rather it moves in fits and starts as the bulk of researchers follow Participatory Research early adopters. Early adopters see participatory research as a no-brainer. Those that follow feel like they’re putting round pegs in square holes. They question the capacity, skill, and confidentiality of lay people in research team roles. They think patients need to be protected, that they need to become more research literate. A great research team has members with statistics and methods expertise, recruitment expertise, project management expertise. Often with less experience with patient/caregiver life flow and direct care clinician workflow. They seldom require life experience training or statistical training for those without such experience. However, everyone, no matter the role, needs to have documented understanding of the rights of subjects and confidentiality of individual data.
I appreciated the presentations at the symposium of three patients (Jane Permuller, Marty Carney, and Paul McLean) in non-subject roles highlighting the benefits of patient participation in research. I also respect Harvard Law School’s Petrie-Flom Center for scratching the surface and reminding us (me) that the spread of participatory research is in its infancy and we activists have much work to do.
Alice’s blood pressure is 110/50 right now. That’s a data point. Her blood pressure, untreated, lives around 150/90. She’s prescribed medicine for it, but she ran out last week and doesn’t get paid for a few more days. When she stands up, she gets sweaty and feels like she might pass out. That’s a bunch of data and a story. Hopefully, the data and the story are given meaning (processed, analyzed, interpreted) by someone with Alice who has medical experience and skills and leads to information about her safety. Alice might have orthostatic hypotension. She probably should sit down, for now, refill her prescription, and get some medical help.
So, the 110/50 (a single data point) doesn’t mean much by itself. Multiple data points + stories, when processed, can lead to information. Information leads to choices which can result in action. Data and stories about Alice are collected by her, others, and machines. She might be able to interpret data. So can others and machines. Most action taken as a result of information about Alice is done by Alice. She can’t write a prescription, but she can take it. She can sit down and elevate her feet. She can seek medical treatment.
Some people and their clinicians are drowning in data and can’t breathe, let alone learn from that data. My OpenNotes record from my neurologist is full of data. Unfortunately, even as a nurse, I understand very little of the note. I want some simple information from the note. How am I doing? I have a progressive disease that will get worse. Am I getting worse? Five pages of data in a note and I can’t tell. I asked my neurologist to explain it to me. He did. Took about three minutes. Turns out the Expanded Disability Status Scale buried on page 4 was the key. I have moved from 5.0 to 5.5 on the scale in the last two years. He used it to support my claim for disability payments. But wait, that’s not right, I can’t walk 100m without my cane. Oh, he says, then you’re a 6.0. Worse than he thought, but now we know. That was an example of missing information (for me) and erroneous information (for him). Let’s not forget biased information. That’s a subject for another day.
The person-clinician relationship feeds on a two-way loop of data, stories, and action about the person receiving care and support. The art for my clinician team members is to help find, share, and interpret data about me and about groups of people like me (old, affluent, white men with Multiple Sclerosis, high cholesterol, food on the table, who have insurance, a home and family), combine them with stories about me, to help me make sense of it all. So I can do something with the information that makes sense to both of us.
So, I still want my DaM Data (Data about Me). But it’s no good without transmogrification (great word!) into information that I can use.
I’ve told my teams over the years, if we don’t fail several times a week we’re not pushing the envelope and not doing our jobs. We weren’t tightrope walkers, pushing IV meds, or manufacturing artificial joints. We were innovators, learners, and leaders. Failure as a virtue is a hard sell – to almost anyone. My teams, my colleagues in leadership, editorial review boards always start by thinking I’m crazy. Sometimes they eventually get it, sometimes not. Leadership usually wants to get A’s. In one health system I worked for, I reported that we successfully completed medication reconciliation in 40% of admissions. OMG, that’s awful! They said. No, I said, that’s great! We’re failing. Let’s succeed. In 18 months we completed medication reconciliation 70% of the time. It’s a lot harder to go from 70% to 80% than 40% to 70%. In research, we don’t publish when the study doesn’t prove the hypothesis. Yet, not proving is as important, if not more important, than proving. I was on an Editorial Review Board once that decided to solicit articles where the hypothesis wasn’t proven and something was learned. Over a 10-year span, we solicited exactly 0 such articles. Zero!
A definition of failure to some is the opposite of success. Not necessarily. Especially when it comes to learning and getting healthier. We don’t tell kids they fail when they fall learning to walk. They keep trying. Same with learning to talk. As an adult, I find failure a motivator to try again. As a thinker and a catalyst for change, I’m delighted when I succeed with 30% of what I try. It’s been the rare boss that’s accepted that. They’ve been the best bosses and we’ve done the best work together in my career.
For health, embrace failure. I did eye exercises twice a day for 8 months before my brain rewired and my crippling double vision cleared 80%. That’s 360 failures and one success! It’s taken years of trial and error to land on a balance, stretching, and strengthening routine that works for me. I stumble a lot, fall infrequently, and sustain only minor injuries when I do. I get frustrated when I see failure and stuck in the muck. Fail and try something else, that’s the ticket.
Failure flavors humility and empathy. My best stories are of failure – my failures. People laugh with me. We can all relate to failure. It’s the warp of our lives. Hearing about a failure, we naturally ask, and then? What happened next? What did you learn? What did you try? What eventually worked?
So, failure, persistence, and humor are inseparable cronies. Keep trying and chuckle at the absurdity. That‘s life, health, music – anything worth doing well. Persist and laugh. Eventually, who knows?
At the #PCORI2017 Annual Meeting, Alan Alda showed us a simple mirror improv exercise (remember Groucho and Chico Marx in Duck Soup?). Alan first showed us him mirroring an audience member, then the audience member mirroring him, and finally, them mirroring each other at the same time. It was an exercise in empathy. Afterwards, someone at my table said,
From the outside looking in, it’s hard to understand. From the inside looking out, it’s hard to explain.
I first heard these words many years ago from a peer support professional describing the experience of depression and addiction. I understand this better now that I’m a person with a chronic illness. I work hard to explain what’s inside to my family and other members of my health team. Often I don’t know or I don’t have words. Mindful meditation helps tremendously – deciding to become friends with what ails me. It’s all me and I love me. I’m not sure if it helps me explain, but it helps me know myself. And for sure, it increases my empathy when I’m on the outside looking in. Thanks, Alan, for reminding us.
See also other posts about Improv and
I care about what works for people on their health journey. How do lay people make choices for themselves in partnership with their clinician partners? So much affects our health choices, not just our medical decisions, but our behavior, our communities, the environment and the systems we use to survive and live well. I’m very interested in research, but I’m also a skeptic: How does this study help me? How does it help my family? How does it help my clinician partners? How does it help the people who support and care for us? We are the people at the center of care. Just because we found out that something might work in a lab, does that mean will it work for us? Read More
It’s hard to reach personal health goals or solve medical problems without a plan. Plans require decisions. Never-ending decisions (choices) in the health journey. Clinicians, researchers, and insurance companies study and use Clinical Decision Support (CDS) to help with the decision-making process. It’s a shortcut for using research (evidence) in the decision-making. Some talk about patient-centered decision support (see a definition at the bottom of this post). They’re trying to figure out how to help people to make decisions in two minutes of ten-minute visits. Yet, few patients or caregivers I’ve met ever talk about CDS. So how can people understand the value and limitations of CDS? Read More