Rare Disease Clinical Research: Caregivers’ Perspectives on Barriers and Solutions for Clinical Research Participation (I8.001)

Abstract

Objective: To identify barriers and potential solutions to participation in clinical research for people with rare diseases. Background: Clinical research is vital to developing new treatments for rare diseases, and maximizing recruitment and participation feasibility are imperatives for small populations. We sought to characterize barriers and opportunities to clinical research participation and hypothesized that modern technologies might ameliorate some of these barriers. Methods: We conducted an online survey to ascertain disease burden, factors influencing clinical research participation, and access to technology. Subjects were recruited via ResearchMatch and National Organization for Rare Diseases organizations who disseminated the survey to their memberships. Results: Respondents included 221 caregivers of individuals with a rare disease. Many diagnoses were represented; the average number of organ systems affected = 3.86 (SD=2.25), and 49[percnt] of affected individuals required medical equipment (N=113). Affected individuals attended a median of 4 medical appointments monthly, and lived a median of 250 miles from the nearest research center studying their disease. 100 (45[percnt]) caregiver respondents were previously approached concerning research participation by the affected individual; 81[percnt] agreed to participate. The most important factors for those who participated were: altruism (99[percnt]), low risk (98[percnt]), and time with the researcher (90[percnt]). The most important factors for those who declined participation were: travel burden (79[percnt]), time with the researcher (67[percnt]), and inability to take time off work (63[percnt]). Of the 221 respondents, many had access to the internet (98[percnt]), a smartphone (86[percnt]), tablet (80[percnt]) or laptop (78[percnt]). 54[percnt] reported that home-based research visits would increase the likelihood of research participation. Conclusions: Patients with rare diseases experience high medical burden, and live at a distance from research centers. However, their caregivers have high access to technology and many support home visits. Use of remote technologies might broaden the scope of interest and engagement in clinical research.

Disclosure: Dr. Amengual has nothing to disclose. Dr. Adams has received personal compensation for review of documents pertaining to a legal case. Dr. Mink has received personal compensation for activities with Edison Pharmaceuticals, Medtronic, and Biomarin, Inc. as a consultant. Dr. Mink has received personal compensation in an editorial capacity as the Associate Editor of Neurology. Dr. Augustine has received personal compensation for grand rounds and as a consultant.