To speed progress in treating chronic conditions, engage patients and caregivers as research partners

In my day job, I conduct research for a large international research and consulting firm on affordable housing and the effects of policies to help individuals build assets, increase earned income, and access resource-rich neighborhoods. In my other job, I care for our teenage daughter who has a chronic connective tissue disorder, Ehlers-Danlos Syndrome, and related neurological and immunological complications.  

At work, my research applies a mix of research methods, ranging from random assignment and quasi-experimental methods to different qualitative research techniques. At home, I find it hard to turn off the research mindset, formulating mini-research questions on a daily basis: What is making our daughter nauseous? Why does she have a new kind of headache? How can we improve her brain fog? And while caring for her, I naturally posit and test hypotheses to address these questions.  

The data I gather in these daily observations are obviously anecdotal, but they may have value for research purposes, especially if other patients or caregivers have similar reports. To accelerate progress in treating chronic disorders like those my daughter is experiencing, as well as the newly emergent long covid, I would encourage researchers to tap into this type of observation and learning. By systematically gathering and analyzing patient observations, researchers could identify promising solutions for more rigorous evaluation.

Chronic diseases like Ehlers-Danlos Syndrome, Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS), and Fibromyalgia have long been understudied relative to their prevalence and the extent of their disruption of normal life. The emergence of long covid among a significant share of the millions affected by covid-19 adds to the urgency of developing solutions to these debilitating conditions.  

Given the high degree of variation from one patient to another and symptoms that can shift over time, patient observation is really the only way to cost-effectively gather the longitudinal data needed to thoroughly understand these conditions. One approach would be to provide patients with tools and training to track their symptoms and response to different therapies on a daily basis over a period of several months. The resulting data could be analyzed using sophisticated qualitative research tools to identify patterns and generate hypotheses for rigorous evaluation of possible treatments using more traditional methods. 

The observations we have made during our daughter’s medical journey help to illustrate the kinds of observations that could be generated through self-reports. Among other data, we have collected information on how her symptoms change over time, which medications and supplements appear to generate improvement, which medications and supplements appear to generate adverse effects, and how she responds to different types of physical activity. If aggregated with data from other patients, these observations could help to identify promising therapies for more rigorous evaluation as well as a more complete understanding of her illness.

By participating in Facebook groups for patients with similar conditions, my wife and I have benefitted from numerous similar observations by other patients and caregivers and their hypotheses about causes and mechanisms for their symptoms. While it’s important not to rely too heavily on individual anecdotal experiences, in our case, at least, the benefits of learning about the experiences of patients with similar conditions, and their real-time feedback on challenges we have faced, have outweighed the risks. 

This leads to a second, and potentially more controversial suggestion. I would encourage researchers to see patients not just as data collectors, but as partners for generating hypotheses for future evaluation. I readily acknowledge that it will be difficult to separate the wheat from the chaff.  But amid a field of theories offered by patients and others, I have seen many well-thought-out hypotheses that merit further scientific investigation.  

To some extent, patient thought leadership and co-production of research is already happening. Many of the individuals experiencing lingering symptoms after their initial recovery from covid-19 – the covid long-haulers – have medical or other scientific training, and some are applying their training to build evidence about the characteristics of long covid. However, in order to access and benefit from the thinking of patients and caregivers without the skills to conduct their own formal research, new approaches will be needed.

One approach is to identify patients, caregivers, and patient advocates that are generating thoughtful content and follow them on social media. Work to build relationships with them and ask for their advice on how to tap into patient-generated observations and hypotheses. Much of the most productive discussion and experimentation is happening in private Facebook groups, and they will understandably be resistant to outsiders coming in who might judge and censor their discussions. But open-minded researchers who show a serious and long-term commitment to studying chronic illness will find allies who can help identify and communicate relevant patient experiences and ideas.

There may also be value in creating more formal vehicles for patients to share their hypotheses. Many medical conferences and journals have made progress in incorporating patient perspectives. But additional steps will be needed to more fully benefit from patient perspectives. Consider, for example, how a patient or caregiver might share a formal hypothesis about a promising treatment or a potential mechanism through which a medication works.  It’s not always clear (at least to this caregiver) which journals might accept a patient-written piece and how it would fare in the peer review gauntlet.  

If we want to make faster progress in addressing chronic conditions, we will need to find new ways to encourage and facilitate the sharing of data and hypotheses by patients and caregivers.

Jeffrey Lubell is the parent of a 17-year old with a long-term chronic illness.

Competing interests: none declared

Patient consent obtained