252 - Enhancing Access to Rare Disease Clinical Trials for Underprivileged Patient Populations: A Strategy for Equitable Participation

Poster Number: 252
Publication Number: 252.2028

Background: Access to clinical trials for rare diseases is a pervasive challenge, particularly for patients residing in rural areas. Rare diseases are those that fit the definition used by the NIH: affecting less than 200,000 people in the U.S. This issue is acutely evident in the Panhandle Region of Texas, prompting the initiation of this project.

Objective: The study’s primary objective was to develop a comprehensive strategy that would bridge the access gap for these underserved patients.

Design/Methods: The study's methodology encompassed interviews with patients within the target region. Inclusion criteria included: 1. Patients treated in the Pediatric clinic (age ≤ 18 y.o.) with rare diseases (per definition in Background) that have ongoing clinical trials on ClinicalTrials.gov 2. Patients and/or legal guardians who are willing and able to sign informed consent/assent forms. Methodology additionally involved the use of pre- and post-participation surveys, designed to extract valuable insights. Data gathered from these surveys were collected, inserted into Excel, and subsequently analyzed using SPSS.

Results: Within the Panhandle Region, patients exhibited a significant interest in engaging in clinical trials (90%). For those that had never participated, concerns included potential side effects (29%) and the financial burden of travel expenses (10%). There was a prevailing desire among patients for more comprehensive information about trial details, with electronic enrollment (48%) and signature processes (55%) garnering notable support. Patients had a distinct preference for referrals from primary care physicians (60%), contrasting with the prevalent reliance on specialists (50%). Feedback from post-survey patients indicated that the information they received proved to be invaluable.

Conclusion(s): The study's findings underscore the evident challenges related to referral and participation in clinical trials for patients in rural areas, particularly in the Panhandle Region of Texas. Consequently, patients remained largely uninformed about relevant clinical trials for their specific medical conditions. Patients demonstrated a resounding interest in participating in clinical trials. Their persistence in the face of these hurdles underscores the immense potential for equitable participation in rare disease clinical trials within underserved areas.