As both a researcher and Charlie’s mom, I am often asked by research teams to help them develop more “patient-centered” research protocols, programs, and advisory groups. A lot of funders are including requirements for the applicant to state how they are working with the patient community. To be clear, I am not referencing any particular funder or activity. Here is some of my best advice. I would love your thoughts and questions and for other advocates to weigh in as well!
Let me start by saying that as a veteran advocate, I don’t really care for “patient-centered,” “patient engagement,” “stakeholder engagement,” or almost any new and buzzy descriptor attempting to upgrade those phrases to make them more inclusive or sound fresher and cooler. I am cynical about this because of the very issues I describe below. I have been tricked into being on “patient-centered” advisory boards only to sit, silenced, at the patient advocate version of the “kid’s table,” while the “grown up” scientists did all the talking. However, a common language facilitates communication, so I use those terms, too. I will use other terms if people ask me to change the words I use, within reason, if it means a lot to them. I think words do matter, maybe not as much to me as they do to others. For example, I changed the name of my blog to remove reference to “special needs” because people told me that it bothered them. Note: I tend to use “patient,” “caregiver,” and “person with lived experience (PWLE)” interchangeably when I write, for efficiency, but they are not interchangeable; I personally prefer PWLE.
But I digress.
For researchers
On the philosophical side, the number one thing you can do to set yourself up for success is to believe, deep down in your soul, that people with lived experience can be true partners in your research endeavors. If you don’t believe that, no matter how many buzz words you use in your application, if the funder is truly serious about patient centered research designs, it will negatively impact your score.
I have plenty of researcher friends who do not get this. Their mistake is in thinking that the great advice that I give them comes from being a researcher AND a mom, and they could not get this from just any parent of a child with a rare disease.
But here’s the thing: my best advice almost never comes from my research training. It comes from something I learned from a more experienced parent thousands of miles away, who does not have a college degree, in the middle of the night in a Facebook group. It comes from years of taking care of a person with rare disease. From arguing with insurance companies, from fighting with the school district, from everything BUT what I do for a living.
I’m never going to convince them, but I still respect their work. If you don’t think PWLE can benefit your work, no hard feelings. Keep scrolling. If you do not believe this deep down, then you should step aside and let others handle the work that involves PWLE. Don’t try to retrofit your very un-patient-centered project for a patient-centered space because you see the dollar signs and/or prestige.
Enough touchy feely philosophical stuff. Let’s get down to business.
On the technical side, the number one thing that you can do to set yourself up for success is make a plan for capturing what outcomes that are important to the stakeholders in your disease area and what evidence gaps they want to see closed.
A big mistake researchers make is selecting outcomes that they assume everyone who is impacted by the diseases will be on board with, but never actually asking a PWLE for their opinion. Not only does this impact funding, but it also affects recruitment. A very good example that I use from my own life is seizure frequency as an outcome measure. Because my son sometimes has hundreds of seizures per month, putting a percentage on seizure reduction isn’t all that meaningful to me. There’s a lot of reasons for this, including the challenges with even accurately counting seizures, but suffice to say that a 25% reduction in seizures still leaves us with a lot of seizures. I’m not sure it would be worth it to me to go out and participate in a study for a 25% seizure reduction, given how difficult it is to get a child like my Charlie out of the house. What would be very meaningful to me would be improving his sleep, allowing us to have more seizure-free family outings, improving his ability to communicate, or reducing the number of rescue meds we need every month. To find this out, and dramatically improve your application, all you have to do is ask!
Do your homework before you apply. Don’t say that you plan to ask the patient community only after you are funded. You have to do this before you submit your funding proposal, because if you really are taking what the patient community is telling you seriously then there’s a chance your study design will change based on what you learn from them. You need to figure this out BEFORE you pitch the study design.
Just yesterday, I advised a group to plan for a simple qualitative study with questions about outcomes and gaps to be conducted during their annual family conference. This will be a quick IRB review and will give them a chance to capture important data long before the panic of a looming grant deadline.
There is no one size fits all patient engagement solution. It should be fit for purpose. Make sure you really understand your stakeholder groups. Hospital based community advisory committees are wonderful, but they are not the sole solution for every research protocol; you may need to add additional advisory teams to meet your needs. If the application is truly patient centered then just saying you’re going to run this by an existing patient advisory board made up of people who might not even have the disease you are researching is not enough. It means making sure there is a balance between patients, caregivers, patient advocacy organizations, and other stakeholders. It’s also important to be specific and diverse. If you are studying a specific variant or phenotype, you should try to identify people from that group as advisors. I am often asked to advise on epilepsy, which is great, but if your study is about the very specific type of epilepsy that my child does not have, you should also try to find someone with that specific condition. Likewise, neurotyical people with epilepsy who live independently are not always the best representatives of my perspectives.
Think about what happens to PWLE if your idea takes off. If your intervention is successful, but will cost a lot of money, or will be difficult to access for another reason, make sure you are identifying barriers to implementation and access, and include groups like insurance companies in your engagement plan. There is nothing less patient-centered than designing an intervention that only wealthy patients can afford.
Have a strong plan for how you are going to incorporate the stakeholder feedback into your project. I have been on every kind of panel of advisors imaginable and many of them have been just an FYI to me. I’m invited to a Zoom meeting, I’m going to watch some doctors talk about what they’re up to in the research project, and then, at the end, the first interaction I have is when I’m asked to post recruiting materials or a link to the manuscript which is behind a paywall and I can’t even read without special credentials. That is not cool, and I won’t return their call next time. There are a lot of great published frameworks for a good patient engagement, which I am happy to share.
Think about the lives of the people you are engaging with before you plan your activities. For example, I need a lot of notice in order to travel because I need to arrange for caregiving for Charlie. A lot of families live paycheck to paycheck because of catastrophic medical expenses. It is a lot to ask of them to pay for the travel expenses up front and reimburse them later.
For funders
If you’re really trying to say that you are patient centered, then you need to take the review process seriously:
You need to make this part of the application important to the overall score.
You should do your homework and use evidence-based engagement frameworks.
You must carefully select and train reviewers with lived experience.
You must give them time and space in live conversations so that the other reviewers don’t talk over them.
You must pay us the same as the “scientific” reviewers.
You should include us in scientific discussions, even if we are not reviewing the sections under discussion.
See my advice above about accommodating travel for people with lived experience.
You must keep an open mind and listen to them if they express that something is wrong with the review process, or they have deep concerns about an aspect of an application that nobody else picked up on. Recently, I pushed back on the review framework of a funder. To their credit they acknowledged that my input educated them, and they were wrong.
I know this is all a lot of work on top of what you’re trying to accomplish with the other aspects of your research, but I promise you that you will never regret including people with lived experience in your research projects!