Establishing Patient Leadership

Hallmarks of Patient Leadership

The foundation of Patient Centered-Core Impact Sets (PC-CIS) creation is a plan that is driven or 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’s (PFMD) Patient Engagement Management Suite resources, including these 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 repository of activities
  • Juvenile Idiopathic Arthritis Patient-Focused Drug Development (PFDD) project and Voice of the Patient Report (VOP)
  • OMERACT Handbook Principles, Chapter 3, Section C engagement

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 myriad functions and roles of patient and patient-group representatives, Who is the Patient? tool.

Collaboration Guidance

Input from various stakeholders is needed in PC-CIS creation. Assuming patient leadership, the first step should be to think through who the various stakeholders are that will 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 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.

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 and family members
  • Clinicians and professional care providers
  • Payers, public and private
  • 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

Collaboration Models

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

  • One or more patient groups that 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 consortia and held 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 within the MS community.
  • Improve Care Now, also originally formed as one of a host of PCORI-funded Patient-Powered Research Networks (PPRN), 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

Arthritis Foundation, Establishing Patient Leadership