If you’ve been reading my stuff for a while, you know that that I encourage everyone to get involved in (ethical) medical research, both when it’s pure science (no clear benefit to the volunteers) and when it’s interventional research (where a treatment might benefit the volunteers). I’ve done both kinds, and the researchers and their staff were always incredibly professional and kind. If there are more opportunities to participate in research, I’ll be first in line to enroll (after reading the protocol and consent documents, of course).
Something else you should know before I get to my main point is that one of the hardest things for researchers to do as part of their jobs, especially with rare conditions, is to recruit enough patients for statistically significant data. There aren’t very many of us (for a given condition), so it’s a small pool to draw from, and it’s not like participating in research is fun or financially rewarding. And that’s before we get to all the usual barriers for potential volunteers who may not be able to take time off from work or family responsibilities, and even if we have the time off, we often have physical limitations on our ability to travel.
The good news is that since patient recruitment is the key to successful research, patients have significant power when it comes to human research. It’s the power of “no.” When the pool of potential volunteers is small, even a few patients saying no is going to shut down the research (or undermine the data if it happens mid-research).
Of course, it’s hard to say no to authority figures (doctors), or to give up on an opportunity that holds so much promise for oneself or the community as a whole. But it’s an option, and I think that too often we forget that we do have that power. I also think that exercising that power a little more often might actually benefit the science that comes out of the research.
I’ve been thinking about the power of no a lot recently, after I heard a report of a major research institution (not naming names, because it’s just rumor) doing research into long COVID, which decided to drop the requirement for the staff to wear masks while working with the patient-volunteers. Keep in mind that the patient-volunteers all have long COVID, and a lot of them are immunocompromised. It also appears that for many patients (with or without long COVID) each subsequent infection can do exponentially more damage, so the patients in this study are at extremely high risk of even worse outcomes than they’re already experiencing, including death, if they’re infected again. At least one patient dropped out of the study (said no) when he arrived at the research site and no one was masked, and they refused to mask when requested by the patient. Reportedly, some other patients dropped out too after learning that the masking had stopped. I later heard that the institution changed the policy so that while masking wasn’t automatic, the patients could request that anyone interacting with them wore a mask, and the staff would be required to honor that request.
Personally, I don’t think that’s a perfect solution — masking in health-care settings should be automatic whenever there’s a high-risk patient on-site — but even the halfway measure is better than the original policy where patients couldn’t even request that staff wear a mask. And it wouldn’t have happened if patients hadn’t exercised their power to say no.
The long-COVID volunteers basically went on strike. They withheld their services (being a research volunteer is work!) until they were heard and accommodations were made. Imagine what we could do to shape medical research in more patient-friendly (and potentially more scientifically enlightening) ways if we went on strike more often!
The long-COVID study is a good example of how patients’ best interests and best practices for scientific validity coincide. I can’t help thinking that if the masking was dropped entirely (and possibly even with the compromise solution), it would adversely affect the reliability of the data from the study. In essence, one of the parameters of the study changed midway through: initially patients were protected by way of mandatory masking (reducing the risk of exposure at the research site), but at some point, patients were no longer protected by mandatory masking (increasing the risk of exposure at the research site). Generally, research protocols try very hard to limit the number of factors that could affect the data, and try to be extremely consistent in EVERYTHING throughout the study, so they can say that one thing (like a treatment) and ONLY that one thing, produced the results captured in the data. Masking protocol is clearly a factor that could affect whether the patients in the study contracted COVID again, and thus could affect their long-term health. The change in masking policy would definitely need to go in the section of the research write-up that talks about reasons why the conclusions might not be valid. And no one dong research wants to have to include something in that section that’s their own fault!
I frequently complain about situations where patients don’t have ENOUGH power (like while research protocols are established, during IRB reviews, and during peer review of journal articles), but it’s important to remember that we do have power in other situations, and we need to be prepared to use it in the right circumstances. If we refuse to participate in research that ignores patient concerns or where principal investigators are disrespectful of patients’ concerns, then the research won’t be able to proceed. If research starts out well, but then takes a bad turn (as in the long-COVID study), and patients drop out mid-study, the data will be useless to both the principal investigator and the sponsor, who either ignored patient concerns or enabled those concerns to be ignored. The sooner the research protocols can be fixed, the better the data will be, so it’s better for both science and the patients.
Figuring out when to say no isn’t easy. The long-COVID study is a perfect example of just how hard it can be. The study was interventional, so the decision to drop out meant weighing the possibility that the treatment could have given them back their health, with the possibility that they’d be exposed to COVID at the research site, which could kill them given their already weakened condition. That’s not an easy choice to make!
For the most part, the hypophosphatemia community doesn’t have to make life-or-death decisions when it comes to participation in research. But that doesn’t mean we should meekly accept whatever researchers ask us to do in the name of science! I’ve been fortunate enough to work with excellent, caring principal investigators, but I know not everyone’s been that lucky. So please remember that researchers need patients as much as we need research. Remember that, in the end, you can always say no. And science, as well as patients, will be better if we exercise our power to shut down bad research until the protocols take into account patient concerns.
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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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