The partnership of patient advocacy groups and clinical investigators in the rare diseases clinical research network.

Michele Manion,Rashmi Gopal-Srivastava,Jeffrey P Krischer,Stephen Groft,H A Jinnah,Peter A Merkel,David Robertson

doi:10.1186/s13023-016-0445-8

Abstract

BackgroundAmong the unique features of the Rare Diseases Clinical Research Network (RDCRN) Program is the requirement for each Consortium to include patient advocacy groups (PAGs) as research partners. This development has transformed the work of the RDCRN and is a model for collaborative research. This article outlines the roles patients and PAGs play in the RDCRN and reports on the PAGs’ impact on the Network’s success.MethodsPrincipal Investigators from the 17 RDCRN Consortia and 28 representatives from 76 PAGs affiliated with these Consortia were contacted by email to provide feedback via an online RDCRN survey. Impact was measured in the key areas of 1) Research logistics; 2) Outreach and communication; and 3) Funding and in-kind support. Rating choices were: 1-very negative, 2-somewhat negative, 3-no impact, 4-somewhat positive, and 5-very positive.ResultsTwenty-seven of the PAGs (96 %) disseminate information about the RDCRN within the patient community. The Consortium Principal Investigators also reported high levels of PAG involvement. Sixteen (94 %) Consortium Principal Investigators and 25 PAGs (89 %) reported PAGs participation in protocol review, study design, Consortium conference calls, attending Consortium meetings, or helping with patient recruitment.ConclusionsPAGs are actively involved in shaping Consortia’s research agendas, help ensure the feasibility and success of research protocols by assisting with study design and patient recruitment, and support training programs. This extensive PAG-Investigator partnership in the RDCRN has had a strongly positive impact on the success of the Network.

Highlights

Among the unique features of the Rare Diseases Clinical Research Network (RDCRN) Program is the requirement for each Consortium to include patient advocacy groups (PAGs) as research partners
A “rare disease” is defined by the Amendment to the Orphan Drug Act of 1983 as a condition affecting fewer than 200,000 Americans or a disease with a greater prevalence but for which no reasonable expectation exists that the costs of developing or distributing a drug can be recovered from the sale of the drug in the United States [1]
Principal Investigators (PIs) from the 17 RDCRN Consortia established at time of this study and representatives from PAGs affiliated with these Consortia were contacted by email and asked to provide feedback via an online RDCRN survey

Summary

Introduction

Among the unique features of the Rare Diseases Clinical Research Network (RDCRN) Program is the requirement for each Consortium to include patient advocacy groups (PAGs) as research partners. Research into rare diseases encounters unique challenges to the scientific community, the biopharmaceutical and medical device and diagnostics industries, academic and public sector researchers, government funding agencies, private foundations, regulatory agencies, and patient advocacy groups. These challenges include difficulties in establishing diagnoses, difficulty in recruiting subjects into clinical studies due to small patient populations distributed over wide geographic areas, few expert centers for diagnosis, management, and research, and a scarcity of investigators focused on these rare diseases. These issues continue to be addressed with steady advances being made in the approach to studying rare diseases and, in parallel, substantial interest by the biopharmaceutical industry in developing products for the treatment of rare diseases

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