Nothing about us without us! (I didn’t come up with that slogan, and I’m not sure who did, but it’s brilliant.) It looks like we need to shout it even louder, because it’s not being heard by researchers (and pharma).
I recently told a researcher that there was a logic error in a survey, which made it impossible to complete. It was a digital survey, and close to the end there was a screen that didn’t include all the possible options, and I fell in one of the omitted options. Plus, there was no not-applicable or none-of-the-above option, so there was no way to finish the survey without choosing one of the inapplicable options. The response was, basically, that the survey couldn’t possibly have that big a problem, because it had been reviewed by both the researcher (medical expert) and the survey company (marketing expert). Do you see the big omission, the elephant NOT in the room, in the list of reviewers? No PATIENT, who would have noticed the omission right away, had reviewed the survey before it went live.
I am just so tired of patients being excluded from the research process at stages where we could so obviously contribute in ways beyond being the subjects under the microscope. It’s particularly annoying when the topic of research is our lived experience, where it’s generally acknowledged that we’re the experts!
There seems to be a trend toward more journal articles on the “lived experience” of patients, and I think it’s a GOOD trend in general. (If you put “lived experience” into the pubmed search engine, you get somewhere between 800 and 900 articles with that key word, just from the first three months of 2026!) But what bugs me is that a spot check of the most recent articles suggests that most DO NOT INCLUDE PATIENTS as anything but data. We’re not listed as authors, and we’re not invited to be part of the team that conceptualizes the article or even that reviews the drafts. And that, I believe, is detrimental to the science in ways we can’t even identify, because no one thought outside the sometimes incorrect expectations of the non-patients.
I’m not suggesting that patients should be the ones establishing the protocol or writing the draft or interpreting the biochemistry, but we should be included in the planning and execution of lived-experience research, just as we should be included in the planning of ALL research and new drug applications. The problem is that the researchers/clinicians don’t know what they don’t know. ESPECIALLY when it comes to the lived experience. And the resulting research/article is the poorer for it. I keep coming back to what I observed during the first research that Ultragenyx did into the lived experience of XLHers, and how, even with a really limited survey (which had only very limited input from patients prior to launch), the results seemed to stun everyone except patients by showing that it wasn’t just kids who were severely affected. I can’t help thinking that all such articles produced without significant patient input likely have useful data, but they’re UNlikely to spot something really interesting, simply because only patients would know to look for it.
Fortunately, there are exceptions where patients are more fully involved throughout the research/publication process. One particularly good example (outside the XLH community, but in a related field) is “The importance of partnership in chronic pain – co-creation of tomorrow’s pain care by working together in research, implementation, and knowledge dissemination.” Not only is the article advocating for better recognition of patient’s insights, but it lists patients as co-authors.
A recent article on the XLH lived experience got it at least partly right: “The lived experience of adolescents with X-linked hypophosphataemia treated with burosumab at end of skeletal growth.” They did the right thing in the beginning by engaging “with four adolescents from the patient advocacy group ‘XLH UK’ to inform the study design and selection of outcome measures, and to assess the feasibility and acceptability of the proposed data collection method.” Getting input at the design stage is critical, but I would have liked to see some later followup by knowledgeable XLHers across a range of ages who reviewed the article for issues the researchers might not have spotted, but at least patients were consulted before the research began. It might also have been useful to get some adult patients’ input early on, since it appears that the minor patients over-estimated their willingness to use trackers (either on paper or digitally), and a caregiver might have been able to temper the expectations.
So, what do we do to be heard? It’s challenging, because we’re generally given a binary choice: participate in surveys that were not optimized with patient input in the design, or not participate. I generally lean toward participation, since more data is probably better than less data generally. Still, I mourn the knowledge we’re missing out on, and I speak up whenever I see research design flaws related to the patient’s lived experience.
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Please note that the author is a well-read patient, not a doctor, and is not offering medical or legal advice.
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