Some key aspects of patient-centered research

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It is a truth universally acknowledged that many patients no longer settle for contributing to medical research only as participants in research studies. Over the last decade or so, patients have increasingly made their voices heard in all nooks and crannies of the research process. Some of my favourite examples include:

Tom Isaacs, who was diagnosed with Parkinson’s disease at age 29, found himself frustrated with the state of research into a cure for the disease. He raised money by walking the full distance around Great Britain and co-founded the Cure Parkinson’s Trust (CPT) to “fund research to slow, stop and reverse Parkinson’s”. Tom’s contributions to changing how research in Parkinson’s is being conducted, especially how patients are being included more these days, cannot be overestimated.

Emily Kramer-Golinkoff, who was born with the rare disease cystic fibrosis, co-founded, similarily to Tom, a charity aimed at curing her own disease. The charity Emily’s Entourage, was founded to leverage the power of community in the fight against CF. Read more about Emily here and watch her at the conference Stanford Medicine X here and here.

Dana Lewis, who was diagnosed with type 1 diabetes at age 14, is probably most known for having designed her own pancreas using a RaspberryPi mini computer (read more here and here). But Dana is also PI (Principal Investigator, the title of the person leading a research project, a pretty big deal among researchers) of a project called Opening Pathways, exploring “the processes around discovery, research, and innovation in health and healthcare”.

The British Medical Journal’s patient partnership strategy, launched in 2014. The initiative is spearheaded by Tessa Richards, who is a physician, a family caregiver and a patient. The strategy has, among other things, resulted in a change in BMJ’s editorial process: authors of research papers are required to document if and how patients and the public have been involved in the study apart from being research participants. The document can be seen here. Read more here and here.

Benefits of patient-centered research

According to articles published in scientific journals, involving patients in clinical research beyond their obvious role as research participants, can contribute to:

making the research more relevant to the people its aiming to help
improving research questions and outcome measures
improving trial recruitment and representativity
improving trial experience and adherence to treatment
reducing dropout rates
improving dissemination of results
increase the likelihood that research results lead to changes in healthcare and/or policy
financial benefits

References:

Domecq JP, Prutsky G, Elraiyah T, Wang Z, Nabhan M, Shippee N. Patient engagement in research: a systematic review. BMC Health Serv Res. 2014;14.14.

Hoos A, Anderson J, Boutin M, et al. Partnering with patients in the development and lifecycle of medicines: a call for action. Therapeutic Innovation & Regulatory Science. 2015;49:929-939.

Levitan B, Getz K, Eisenstein EL, Goldberg M, Harker M, Hesterlee S, et al. Assessing the Financial Value of Patient Engagement: A Quantitative Approach from CTTI’s Patient Groups and Clinical Trials Project. Ther Innov Regul Sci [Internet]. 2017; Available from: http://journals.sagepub.com/doi/pdf/10.1177/2168479017716715

Staley K, Minogue V. User involvement leads to more ethically sound research. Clin Ethics. 2006;1(2):95–100.

But what is patient-centered research?

I can honestly say that I am not sure myself and there is a wide range of different types of research on the continuum from fundamentally basic research on cells in a lab to the kind of patient-driven research that, among others, Dana and I are conducting. I have listed some aspects that I believe are key to truly patient-centered research, partly based on BMJ’s document for authors submitting research papers (link here).

Key aspects in patient-centered research:

All scientific articles are published open access. I strongly believe that no research can be patient-centered unless all scientific articles are published open access. On the flip-side this means that also lab-based basic science can also incorporate patient-centered aspects.
Research designed with patients. This means that patients are included as an equal partner when choosing research methods, formulating research questions and choosing the outcome measures/clinical endpoints.
Continuous improvements. Feedback from patients is collected continuously during the study and used for improvements.

What do you think? Do you agree with me? Have I missed important aspects? Do all criteria have to be fulfilled for research to be considered patient-centered or can there be levels? Please add your thoughts in the comments.

patient centered

Comments

3 responses to “Some key aspects of patient-centered research”

July 19, 2019

Fran Lo

Standing and clapping! The publication piece is so important!

Reply
July 20, 2019

Sue B

I’m standing and clapping alongside Fran! Great post, Sara. Thank you! Really makes the case that patients can and should be involved throughout the lifecycle of a clinical trial – from concept to development to implementation to publication and followup.

Reply
July 25, 2019

Sally Okun

Thanks Sara for sharing your perspective and these important principles – indeed we agree!

You have captured the essence of patient centric research both in the examples of how patients can contribute as true partners in research and in the key aspects of patient-centric research that we believe should be embraced by every researcher and investigator.

For more than 11 years it has been a privilege to be part of a diverse team at PatientsLikeMe that honors these principles as fundamental to the social contract we have with patients as partners in research. There are some who believe a for profit company like ours simply seeks to gather data from patients to sell On the contrary, at PLM we follow rigorous scientific processes and hold ourselves accountable to legal, ethical, and regulatory standards (some of which we impose voluntarily) to establish trust among our patients and other stakeholders interested in learning directly from patients about the everydayness of living with and managing their lives in the presence of illness, complicated aging and caregiving.

Here’s a few examples of how we partner with patients to support the principles you’ve offered for patient-centric research:

1.Open Access – All of our 100+ publications, most in peer reviewed journals are open access whether they are projects initiated by our research team and patients or conducted with external researchers including industry, regulators, academics, policy makers and clinicians. We have always followed the priniciple and regularly update our Research Page with a chronological compendium of all our publications and conference posters. Each item in the compendium includes a brief description of the study, our collaborators, the citation and link to the open access publication.

2. Research designed with patients – ‘Patient First’ has been fundamental to our core values since our founding in 2004. We demonstrate this in many ways but most importantly by listening and learning from patients to ensure our research explores topics and questions that matter to them. This is well described in our publication “Building a Learning Community: by the people, for the people” commissioned by the Learning Health System Journal in 2017. In it we share and encourage others to freely use the patient informed tools we’ve developed including the patient/caregiver journey framework and principles for measurement and design.

Some of our research comes directly from patients such as our well-known lithium study. Activated patients with ALS and their caregivers worked with our team to create data collection tools to replicate a clinical trial that reported lithium slowed the progression of ALS. In partnership with patients these findings were refuted. In another example a PLM member living with ALS for more than 10 years expressed dissatisfaction in the PLM ALS forum that the ALSFRS-R, the “gold standard” measure of ALS function, was insensitive at lower levels. Patients like her, or Stephen Hawking, could be rated a zero in terms of function, despite the fact they could still work, use the computer, and manage their affairs with the support of assistive devices. This PLM member acted as a co-investigator in the development of the ALSFRS-EX that is used in clinical research today and as co-author of the subsequent publication.

3. Continuous improvement – The mantra “give something, get something” is embedded into our culture. We believe it is our responsibility to ensure that when patients update their PLM profile with data that see that data reflected in meaningful visualizations and charts that are useful for monitoring their health, tracking the effect of a new treatment, measuring severity of a symptom or side effect and/or to share with providers the myriad of experiences that occur outside of the clinical encounter – the 1 vs. 8,765 so wonderfully depicted in Sara’s visual. A patient’s PLM profile is essentially an ‘n-of 1’ person-centric research study that is conducted by the patient empowered with PLM’s tools and infrastructure. Our condition, treatment and symptom reports aggregate de-identified data from member profiles continuously each night to reflect the collective experiences of thousands of patients. In addition a ‘give back’ strategy is an integral part of protocol driven research studies including those we conduct with external researchers.

In summary, the infrastructure to support patient-centric research takes time, resources and the commitment of many people, most importantly of patients. However, building the tools to conduct patient-centric research is only the beginning – earning the trustworthiness of patients to partner with us in research requires building relationships, legitimacy, creditability and accountability to do patient-centric research right and well. We commit ourselves to earning that social license from patients every day.

Thanks Sara for bringing attention to these essential principles.

References:
PLM Research Publications Bibliography https://patientslikeme-bibliography.s3.amazonaws.com/PLM%20Research%20Manuscripts%20Bibliography.pdf

PLM Research Posters Bibliography https://patientslikeme-bibliography.s3.amazonaws.com/PLM%20Research%20Manuscripts%20Bibliography.pdf

Okun S, Goodwin K. Building a learning health community: By the people, for the people. Learn Health Sys. 2017;1:e10028. https://doi.org/10.1002/lrh2.10028

Wicks P, Vaughan T, Massagli M, Heywood J. Accelerated clinical discovery using self-reported patient data collected online and a patient-matching algorithm. Nat Biotechnol. 2011. 29:5:411-414. https://www.nature.com/articles/nbt.1837

Wicks P, Massagli M, Wolf C, Heywood J. Measuring function in advanced ALS: validation of ALSFRS-EX extension items. European Journal of Neurology. 2009. 16:3:353-359. https://doi.org/10.1111/j.1468-1331.2008.02434.x

PLM Condition reports https://www.patientslikeme.com/conditions
PLM Treatment Reports https://www.patientslikeme.com/treatments
PLM symptom reports https://www.patientslikeme.com/symptoms

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Sara Riggare