Establishing Patient Leadership

Hallmarks of Patient Leadership

The foundation for Patient-Centered Core Impact Set (PC-CIS) creation is a plan that is led by patients or co-led by patients in partnership with other stakeholders. Patient involvement can and should contribute to each aspect of PC-CIS creation, including highly technical aspects such as data analysis. The following provide guidance for best facilitating patient leadership:

  • NHC Patient Engagement Rubric
  • EveryLife Foundation Guide to Patient Involvement in Rare Disease Therapy Development
  • Patient-Centered Outcomes Research Institute (PCORI) Patient and Family Engagement Rubric
  • Patient-Focused Medicines Development (PFMD) Patient Engagement Management Suite resources, including How-To Guides and guidance on high-quality engagement

Many efforts similar to PC-CIS creation have employed robust patient leadership. Examples are provided below for your reference:

  • Parent Project Muscular Dystrophy (PPMD) repository of activities
  • Juvenile Idiopathic Arthritis (JIA) Patient-Focused Drug Development (PFDD) project and Voice of the Patient Report (VOP)
  • OMERACT Handbook Principles, Chapter 3, Section C engagement

Considerations for Patient Leadership

A key consideration in patient leadership and engagement is clearly defining patient and patient representatives’ roles and the perspectives needed. The National Health Council (NHC) has developed guidance for identifying and describing the many functions and roles of patient and patient-group representatives [Who is the Patient?].

An important step is outlining the time commitment and level of engagement expected of patient partners. There must be a sufficient number of patients involved in the effort and workload, and responsibility can be shared as required. For example, patients who want to be involved but cannot offer a significant amount of time, may need an option for involvement that is more flexible and allows them to contribute asynchronously as their schedules and commitments permit.

For those patients and patient groups who are invited by other stakeholders to partner in the creation of a PC-CIS, (versus leading the effort themselves) it is imperative all collaborators come to the table with the same high level of appreciation for the critical role of patients. Patients should carefully evaluate the authenticity of the invitation. Are they being brought in at the last minute? Are they being invited to co-lead the initiative or is the role more limited? Can they trust that their priorities and insights will be honored? These are some of the questions they need to ask.

Collaboration Guidance

Input from various stakeholders is needed in PC-CIS development. Assuming patient leadership, the first step is to think through who the various stakeholders are that also need to be engaged and how to engage them. Clinicians, clinical specialists, and others who provide services related to the condition should be involved, as well as payers for health care services, including government and private insurance companies when possible. Private employers, who in the US and elsewhere are the source of health insurance for many employed persons and their families, are another stakeholder for consideration. These efforts can also include researchers from academia, government/regulatory entities, and life science industries (biopharmaceutical, device, and diagnostic).

Lastly, thought should be given to the inclusion of experts from nonprofit organizations that might be able to contribute to the effort. These would include professional societies, research foundations, and charitable organizations that provide services or research funding related to the disease.  Depending on the group aiming to develop the PC-CIS, there may be partners outside of the healthcare sector who could be important such as educators (pediatric conditions) or occupational safety experts (musculoskeletal or neurological diseases).

In alignment with ensuring all relevant stakeholders are included, there will likely be the need to form collaborations and consortia to accomplish PC-CIS development. These partnerships can take many forms. But, again, the patient community should lead or have a leadership role when not the lead organization.

Potential Collaborators

  • Patients, patient groups, other patient community members including carers/caregivers and family members
  • Clinicians and professional care providers
  • Public and private payers
  • Employers
  • Government agencies and regulatory bodies
  • Value and health technology assessment (HTA) bodies
  • Researchers from academia, government, and industry
  • Professional associations
  • Philanthropic research and funding organizations
  • Policymakers and regulators
  • Community and city planners and social service providers
  • Others with unique experiences and perspectives


Collaboration Models

It is expected that collaborations and consortia will be needed to bring all stakeholders together for successful PC-CIS development. Examples of such models include but are not limited to:

  • One or more patient groups which form a consortium and invite other stakeholders to join. For example, groups representing the Lupus community, The Lupus and Allied Diseases Association, the Lupus Foundation of America, and the Lupus Research Alliance, formed a consortium to hold an externally-led Lupus PFDD meeting and produced a Lupus Voice-of-the-Patient Report. 
  • Initially a PCORI-funded Patient-Powered Research Network (PPRN), iConquerMS is a consortium of several patient advocacy and membership organizations from the multiple sclerosis community, working together to conduct research and take on other health and quality-of-life work.
  • Improve Care Now, also originally formed as one of a host of PCORI-funded Patient-Powered Research Networks, is a research collaborative of young, adult inflammatory bowel disease (IBD) patients, parents of pediatric IBD patients, and IBD clinicians and researchers, all committed to improving treatment and quality of life for people with IBD. The IBD Partners PPRN Principles for Patient Collaboration guidance document was created through this work.
  • Yet another model is the Patient-Led Research Collaborative, described as a “self-organized group of Long COVID patients working on patient-led research around the long COVID experience.” These patients come from diverse backgrounds including research and public policy and connected over their collective long COVID experience to form the collaborative, soon drawing in other partners and ultimately publishing important research on Long COVID.
  • The Patient-Centered Outcomes Research Institute (PCORI) has created a guidance for building and maintaining multi-stakeholder collaborations.

Arthritis Use Case

  • Multiple staff project leads had extensive lived experience and a long history of working with patients and families from the Arthritis Foundation community. Individual patients living with forms of arthritis were recruited to provide insights on an ongoing basis.
  • The Juvenile Arthritis (JA) version of the INSIGHTS program focused on issues of importance to pediatric populations [*Note: The pediatric surveys are completed by parents as observers of the children since collecting patient-reported outcome data from those under 18 years of age added complexity and required resources the program did not have access to].
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