The Promise of Patient-Led Research Integration into Clinical Registries and Research

Project Summary

Background: While patients are increasingly engaged in clinical research and clinical registries in an advisory capacity, collaboration and patient leadership in clinical effectiveness research remains limited. Newer patient-led participatory outcomes research for Long COVID-19 has demonstrated the potential of patient-led outcomes research to assess treatment options for the clinical and functional outcomes that matter most to patients. The Patient-Led Research Collaborative (PLRC) was established to drive research to capture and share a bigger picture of the experiences of patients suffering from COVID-19 with prolonged symptoms using a data-driven approach. In this new research paradigm, patients move from research contributors to leaders of survey and research design.

Proposed Solution to the Problem: The project team will develop tools and resources for traditional research entities and patient-led participatory research communities to overcome challenges in the organization and sustainability of patient-led outcomes research teams. The project team will develop innovative strategies to support capacity building and sustainability models for patient-led patient-centered outcomes research/comparative clinical effectiveness research (PCOR/CER), build bridges to the medical research community, and foster new models of collaborative PCOR/CER.

Objectives:
• Develop innovative strategies to support capacity building and sustainability models for patient-led outcomes research
• Build bridges to the medical research community, and foster new models of collaborative PCOR/CER
• Develop a model for community-driven patient-led outcomes research for replication in different clinical areas based on successes and lessons of the PLRC
• Foster collaborative models of clinical outcomes research and PCOR/CER across diverse medical and patient communities

Activities: The project team will conduct perception surveys and key informant interviews to understand the needs, challenges, barriers, and opportunities for the integration of patient-led outcomes research from the medical and patient communities. A multi-stakeholder advisory panel will be convened to promote collaboration of research experts and patient communities. The team will develop playbooks and training programs for patient communities and clinical research organizations.

Projected Outcomes: The team will develop effective strategies to support capacity building and sustainability models for patient-led PCOR/CER research, build bridges to the medical CER community, and foster new models of collaborative PCOR/CER. The team will develop the tools and resources necessary for institutions and organizations to replicate the inclusion of patient-led and patient-owned data into existing research platforms.

Patient and Stakeholder Engagement Plan: The project is designed to be a partnership between the clinical, research, and patient communities. The project team is a partnership between the Council of Medical Specialty Societies (CMSS) and Patient-Led Research Collaborative (PLRC). The PLRC team of patient researchers, as well as an expanded panel of experts and patient partners, will be involved at all stages of the project.

Project Collaborators: Council of Medical Specialty Societies (CMSS), Patient-Led Research Collaborative

https://www.pcori.org/research-resu...-integration-clinical-registries-and-research

 

From Patient-Led Research Collaborative Twitter thread

"Attention all those who value patient-led research! In partnership with @CMSSmed & funded by @PCORI, we have developed scorecards that evaluate how effective a patient group and research partner collaboration will be at conducting truly patient-led research"

"The goal of these scorecards is to demonstrate how patient groups and research partners can improve their inclusion of patients in the research process so that the research centers patients and leads to improved outcomes for all."

"We're hosting 3 webinars that will explain the scorecards & teach you how they can be used to promote patient-led research. The scorecards were developed based off our experience partnering w/ research organizations, as well as from interviews w/ patients, researchers, & funders"

"Target audience is patients/patient orgs interested in collaborating on research, researchers and research orgs who want to better incorporate patients in their study design process, and funders who want to fund research that meaningfully involves patients"

"The first webinar is this Thursday, January 19th at 3pm ET and will cover Research Organization Readiness and Integration into the Research Process. Register here: us06web.zoom.us/meeting/regist…"

"The second webinar is Thursday, January 26th at 3pm ET and will cover Patient/Partner Governance and Patient Burden. Register here: https://us06web.zoom.us/meeting/register/tZMoce-trTMtHtWCqtRUdghfT7BoXGgtIVsT"

"The third and final webinar is Thursday, February 2nd at 3pm ET and will cover How to Improve Your Score and serve as a Q&A session. Register here: https://us06web.zoom.us/meeting/register/tZwocuCprD8oE9fNF1PpZ944wq8cQwsJsh74"

"The scorecards themselves will be posted publicly soon for all to use - stay tuned!"

"And here they are!!! Scorecards that evaluate Research Organization Readiness, Patient Burden, Patient-Partner Governance, and Integration into the Research Process are available at this link: cmss.org/wp-content/upl…"

Scorecards in original PDF format, https://cmss.org/wp-content/uploads/2023/01/11231_CMSS_Plybk_Scorecards_V3.pdf

and each page individually (click to view)

 

Thanks for posting, @Andy! I saw this info yesterday and didn't have the energy to do a summary or figure out where to put it.

 

I really hope they take this seriously. So far I can't say I have seen any good comments about patient engagement, and a lot of frustration that the same old is happening, patients not being listened to and serving basically as photo op background to the usual politicking and everything substantial happening in secret.

Even Koroshetz had some comment about how they're not used to work like this, which really puts into a bad light the idea that patient engagement is growing. The AIDS initiative remains the only actual process where this played out, the concept of "nothing about us without us" is strictly a patient perspective, it is rejected by the traditions of medicine.

The patients have fully bought in and put in the effort. The institutions of medicine continue to reject the premise that it has any value, the very hint of patient involvement in an outcome is basically seen as an affront to the idea that experts know better, nevermind that when it comes to illness, medical professionals have no scientific theory so the patients are the only valid experts.

 

One of the requirements perhaps ought to be evidence of significant interaction with colleagues who're working on the clinical side seeing patients.

And for any researcher who is working on the clinical side themselves, evidence of interaction with a patient group outside of the confines of time-limited appointments, since there's rarely time to discuss all of the symptoms or their cumulative effect on patients' lives. The symptoms focused on during appointments might also be skewed towards those most amenable to currently-available treatments or that suggest the need to exclude another diagnosis, rather than the most characteristic of ME.

 

Well, the simplest way forward is usually to pay people for the time they spend working on something. When I commissioned artists, they estimated how long a particular commission would take, based on the briefing given and their previous experience of delivering work. Sometimes they'd gain slightly because they over-estimated, other times they'd lose slightly when it took longer than they thought. But on the whole it was a fair and acceptable arrangement—unless the company made decisions that took events out of the artist's control and caused them significant extra work. Then (quite reasonably) they might ask for further compensation.

That principle could be applied to ME patients. Someone with moderate ME, for instance, who travels to a half day meeting, takes part, and then travels home again may need to spend the next couple of days recovering. Therefore, they've committed three days' time to it.

But if a key contributor to the meeting is delayed, the chair decides the meeting should be put back by an hour, and then it also overruns (meaning the patient blows their activity capacity for the day and ends up really unwell with PEM), they might reasonably ask to be compensated for that additional time. Even if it weren't possible due to budgetary constraints, it's a really important learning point for the researchers; they get to understand that, when it comes to working with disabled people, minor issues can have major impacts and they need to add a contingency line next time.

It's one way to look at it, anyway.

 

Other aspects that I think need to be factored into the conversation around patient and carer involvement are:

1. The reduction in community capacity that might happen when an advocate is involved in a research project.

For patient communities that have a more established and extensive pool of advocates this might be less of an issue, but I believe it currently is an issue for the ME community, especially when an advocate might be a patient as well. The simple example is myself - my involvement with DecodeME has meant that I have had to reduce my level of involvement with the forum and elsewhere. This has then meant a reduction in the capacity of the forum.

I don't think that there is necessarily an answer to this but if in the future, as we all hope, we see more research into ME and more patient involvement, this may well have a negative impact elsewhere.

2. "Isolation" of involved patients from the community.

I've put isolation in quotes as I don't think it's quite the right word but I can't think of an alternative at the moment.

Particularly as our energy is restricted in the way it is, any patient that is involved in a research project is likely to be restricted in their ability to involve themselves in, and receive support from, the patient community to the same extent as they might have been before. Add in that they will often have to keep confidentiality about the project they are involved with, and that some community members might express negative opinions about their involvement with the project, and the involved patients might well experience a disconnection from their community.

 

@Andy, I like the idea of including as many aspects of a patient participants experience as possible.

You could also think of this one like when an academic leaves one university to go to another, but as if there were also a shortage of lecturers to fill their previous position.

 

Not read this properly yet but a scan of the concept I assume being:

- most researchers would need so much time and experience to 'get' ME/CFS and its limitations in their entireity and even then still consult (recruitment 'samples' being representative and 'inadvertent' exertion during trials being obvious issues)

- not all PwME are 'qualified' on all aspects needed either (!) because of the differences between severity, situation and the fact we've all been gaslighted affecting how we even look at ourselves and each other, what we'd rather try or like to think/hope for, but also taken several 'rounds' of trial and error to realise what you can and can't beat and what you are kidding yourself with when you think you feel better etc.

- you then have the overlap between the health/energy/situation of PwME (and that will vary due to virus or exertions, as well as probably one day we might get to the stage where we can categorise envelopes vs situation so someone knows if they are a 10min a week or 2hr a week brain capacity)

- and the skillset and knowledge of PwME

... so trying to do all of this for one one-off trial is quite an uphill climb, whereas if you recruit 'panels' (and I use this term because.e.g in market research there are studentpanels or sectorpanels where they basically can draw from as an email list for when they need to do a survey or focus group those who fit the criteria for certain pieces of research) you've a pool that you can then slowly train and find out their different boxes they tick on the above.

By training it could be as simple as if they were involved as a patient rep for a trial then if there were loads of boxes to tick e.g. reading protocols or policies but these are consistent then they could be done over a longer time period if someone was on a panel and e.g. the E&D policy could be read and lasts for 2yrs vs if someone was signed up and had to read a bundle of docs within weeks.

But it could also go as far as it being easier to get those with direct experience up to the speed to converse with the researchers terms than the other way around to make co-creation constructive.

And yes, this could even mean turning the current cliche on its head of having the patients crowbarred into the usual routine of researchers designing projects, to top-down - where things can really make the most difference to trials and research fitting to the idiosyncracies the illness presents genuinely being understood so they don't undermine the research question by not being accommodated - patients designing and inventing with the support of researchers from the very start of a trial or inventing ideas and templates that can be used in general and built from for research.

If this is anywhere near heading in this direction then great - I'm glad to see it being written about. And am intrigued to see how close anyting gets to/can get to currently thinking on these lines.