The Coalition of Patient Advocacy Groups seeks to amplify the voices of patient advocates in all activities of the Rare Diseases Clinical Research Network. The Coalition works together to find the causes of all rare diseases studied in the Network. The Coalition has representatives on the Network’s Joint Leadership and Steering Committees. This gives the Coalition a defined role within the Network structure.

The Coalition also creates resources for patients and patient advocacy groups. Their first webinar series, available for free online, describes how patient advocacy groups can work with industry representatives and regulators to improve the patient experience in future rare disease clinical trials. The second webinar series, also available for free online, spotlights resources available for patient advocacy groups to accomplish their work.

Stage(s) of Research Process:

• Study Planning: The Coalition works directly with Network research groups to design studies, improve data collection, and develop recruitment materials.
• Study Conduct: The Coalition helps Network research groups and clinical sites by connecting patients and families to potential studies. They also educate healthcare providers so that they can refer patients to potential studies.
• Post-Study: The Coalition helps Network research groups share research findings to patients and caregivers through webinars, social media posts and videos, and personal appearances. The Coalition also helps share results with regulators, policy makers, industry representatives, and committees that write clinical practice guidelines.

Who Might Benefit:

• Patients, Caregivers, and Patient Advocacy Groups
  • Can use the information found in the Coalition’s webinar series, including a detailed description of the patient’s experience in rare disease clinical trials and resources available to patient advocacy groups.
  • Can use tools and information on the Resources for Patients and Families section of the Rare Diseases Clinical Research Network. The Coalition has direct input on these materials. The information covers topics such as finding financial aid, how to communicate with healthcare providers, and how to join or make a patient registry for rare disease.
  • Will also benefit from the progress towards new treatments and cures for rare diseases that result from their contributions in Network decision-making.
• Researchers can use the structure of the Coalition as an example for including the voices and perspectives of patients and patient advocacy groups in decision-making for research efforts.

Key Takeaways and Examples in Practice:

• The Coalition provides researchers with study design tools to increase the chance of study success, provides information about the research network to their patient communities, and assists with patient recruitment.
• Shared leadership of a research network accelerates progress towards the testing of new treatments in clinical trials and turning research findings into treatments.
• Patient advocates provide meaningful information to researchers to help decide research priorities and agendas.
• Researchers are inspired by patient advocates and appreciate their contributions to study design, recruitment, and sharing of results.
• It takes time to build the understanding, trust and flexible governance that are needed for these new models of shared leadership between patient advocates and researchers.

This work was done with the direct support by each of the following partners who advance this work:

• Rare Disease Clinical Research Network Consortia
• National Institute of Health Partners and Key Personnel
• Rare Disease Clinical Research Network Committees

Keywords: Collaborative Decision-Making; Methods of Engagement; Sustainable Engagement & Capacity Building; Return of Research Value; Patients, Caregivers, and Patient Advocacy Groups; Researchers

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Links to Relevant Resources:

• About the Coalition of Patient Advocacy Groups
• Coalition of Patient Advocacy Groups Steering Committee
• Coalition of Patient Advocacy Groups Webinar Series
• Scientific Publication Describing the Partnership Between Patient Advocacy Groups and Research Teams in the Rare Diseases Clinical Research Network
• Resources for Patients and Families
• Rare Disease Research Network Research Teams
• NIH Partners and Staff for the Rare Disease Research Network
• Contact the Rare Disease Research Network

Definitions for each engagement objective can be found in the glossary.