From the Patients

Defining Patient Engagement in Research

Rob Camp, BS, Community Advisory Board Programme, EURORDIS, Barcelona, Spain; Russell Wheeler, BSc, Leber’s Hereditary Optic Neuropathy, Merusac, France; Rainald von Gizycki, MA, PhD, PRO RETINA Deutschland e.V., Aachen, Germany; Robert McBurney, BSc, PhD, Accelerated Cure Project for Multiple Sclerosis, Waltham, MA, USA

The Patient Engagement in Research Working Group of the ISPOR Patient-Centered Special Interest Group worked over several years to research and derive consensus on the definition of “patient engagement in research.” This group was comprised of patient advocates, academics, industry, and health economic and outcomes research students. The team survived many obstacles that seemed insurmountable on the path to publication, including learning how to conduct a literature review, perform qualitative analysis, and develop consensus.

The definition published in the June 2020 issue of Value in Health is excerpted below:

"The active, meaningful, and collaborative interaction between patients and researchers across all stages of the research process, where research decision making is guided by patients' contributions as partners, recognizing their specific experiences, values, and expertise."

-From Value in Health. Vol. 22, Iss 6.

While our definition is easily applicable in health economics and outcomes research, we see potential for it to be used much more broadly. This includes medical research and patients’ rights. The definition reflects a broader understanding of truly engaging with participants in a collaborative setting, where the responsibility could be shared by the “sponsor” to include the “one being experimented on” in a full and transparent way.

It’s adaptability is its beauty. It applies not only to identifying and interpreting new quality-of-life and patient-reported outcome  measures, but also to answering the fundamental questions that need to be considered before the decision to conduct a study has even been taken and the research begins. This includes basic questions such as, “Is this research into a product or area valid?” and “What do patients need?” in any disease or investigative approach. This is why we, the patient researchers, are taking this opportunity to share our thoughts. 

Our original hope for the usefulness in defining the term in our report is the same: all healthcare stakeholders can now adopt the underlying principles of the definition and start using it. This definition can also be used to validate organically grown organizational definitions. Patient engagement is frequently subverted into a tool used by many groups to do their “same old, same old” under the guise of being patient centric. Patient engagement has frequently been used as a buzzword. Buzzwords can be taken out of context and molded into things that they really are not meant to be. Organizations can now be asked how their approach differs from the official ISPOR definition of “patient engagement in research.” This point of comparison provides a means to hold them accountable to common principles.

On a practical level, we are not introducing a new concept, as much as trying to institutionalize something that has maintained itself on an ethereal level. Rather than a sponsor approaching a group of patients and saying, “This is our research project. Do you like it?”, applying the new definition, the approach would be, “You have lymphoma? We research lymphoma. What do you as patients need and how can we accomplish that?”

Through this project we suggest a definition that can be used all along the research pathway. While our definition may seem aspirational, it can be achieved. There are many existing examples of its principles in practice that demonstrate the power of true patient engagement, with patient organizations successfully driving and informing the research process from inception through clinical care. We offer our varied experiences as examples. Each of the following examples resulted in learnings about how to undertake authentic patient engagement in research, as well as the two-way respect and communication during the collaboration can result in an obvious cultural change in both organizations. Our proposed definition reflects these same principles and will hopefully contribute to transformational change in other organizations.

PRO RETINA Deutschland e.V.

The PRO RETINA Deutschland e.V. and its foundation demonstrate the potential of patient organizations in the initiation, acceleration and support of patient-oriented medical research. The foundation aims to identify research needs, partner with research institutes, biomaterial banks, registries, and support patient recruitment for studies. Through patient-initiated and patient-supported research, this organization has achieved the isolation of the choroideremia gene and the development of a retinal implant. The foundation also collaborates on drug approval procedures and other regulatory decision making.

Leber’s Hereditary Optic Neuropathy Society

The Leber’s Hereditary Optic Neuropathy Society has engaged in patient-focused research and healthcare delivery consortia, which has led to the creation of new patient-originated and patient-directed research networks under the umbrella of the European Joint Project for Rare Diseases. The Leber’s Hereditary Optic Neuropathy and the James Lind Alliance engaged in a recent Priority Setting Partnership for primary mitochondrial diseases, which demonstrates the importance of patients and clinicians working together to identify patient-focused research priorities and goals. 

The European Organization for Rare Diseases Community Advisory Boards

Many members are familiar with patient advisory boards, typically organized by the sponsor.  Community Advisory Boards (eg, the EURORDIS Community Advisory Boards in rare diseases) provide an opportunity for sponsors, mainly pharmaceutical and biotech companies, to collaborate with the patient community throughout the research process. Community Advisory Boards are autonomous bodies not related or chosen by the sponsor and provide valuable input to research protocols through transparent dialogue with researchers and the patient community. Quick wins include changes to research protocols such as revision of inclusion/exclusion criteria, numbers of visits to the clinic during a trial, how many biopsies are needed, the relation of investigational drug to placebos, and much more. 

Patient-Focused Drug Development Collaboration

Another example of patient engagement in research is the collaboration between the Accelerated Cure Project’s iConquerMS People-Powered Research Network and a life sciences company. This partnership occurred in the lead up to the launch of pivotal clinical trials of a new candidate medicine for relapsing forms of multiple sclerosis. This patient focused drug development Council met monthly to discuss trial design and implementation, clinical trial logistics, patient-reported outcome measures, clinical trial training materials and draft label statements.

As you can see from these few examples, there are different approaches to patient engagement in research. While PRO RETINA focuses mainly on basic and applied research in a university environment, the other examples are primarily focused on clinical trials and public commitment. This is evidence of engagement in “all phases” of research.

"We, the patient representative authors, worked with the health economics and outcomes researchers, showcasing the gravity and importance of including patients in research..."

Looking Ahead to a Patient-Focused Future

While developing this definition, we found the great majority of the existing literature was from scientific articles with minimal input from patients. Our group worked diligently to be as open as possible, considering the perspectives of different users and stakeholder groups. Hats off to all our fellow authors, reviewers, and Special Interest Group and Working Group members who shepherded us through this process. A definition has been produced that can be used and implemented not only by ISPOR, but also can be easily adapted and used in many engagement settings. This includes settings beyond what might be strictly called research where it can benefit society as a whole.

ISPOR has a long-standing commitment to the engagement of patients in healthcare research and decision making worldwide. We, the patient representative authors, worked with the health economics and outcomes researchers, showcasing the gravity and importance of including patients in research, actively listening to patients, and understanding them and their needs and expertise as a stakeholder group. The proposed definition highlights the importance of inclusion, respect, and equality in research. Our hope is that the published article in Value in Health can build on the various projects ISPOR has undertaken to include patients in the research and decision-making processes. We hope that this definition will be an aspiration for everyone working in research. 


1. Harrington RL, Hanna ML, Oehrlein E, et al. Defining Patient Engagement in Research: Results of a Systematic Review and Analysis: Report of the ISPOR Patient-Centered Special Interest Group. Value Health. 2020;23(6): 677-688.

For more information

Contact patientsig@ispor.org for more information about ISPOR’s work with patient engagement.

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