Recruitment of Patients With Amyotrophic Lateral Sclerosis for Clinical Trials and Epidemiological Studies: Descriptive Study of the National ALS Registry's Research Notification Mechanism

• Published in: Journal of medical Internet research Vol. 23; no. 12; p. e28021
• Main Authors: Mehta, Paul, Raymond, Jaime, Han, Moon Kwon, Larson, Theodore, Berry, James D, Paganoni, Sabrina, Mitsumoto, Hiroshi, Bedlack, Richard Stanley, Horton, D Kevin
• Format: Journal Article
• Language: English
• Published: Canada Gunther Eysenbach MD MPH, Associate Professor 07.12.2021; JMIR Publications
• Subjects: Adolescent; Adult; Age; Aged; Amyotrophic lateral sclerosis; Amyotrophic Lateral Sclerosis - epidemiology; Amyotrophic Lateral Sclerosis - therapy; Clinical research; Clinical trials; Clinical Trials as Topic; Committees; Confidentiality; Disease; Electronic mail systems; Eligibility; Email; Epidemiologic Studies; Family medical history; Females; Gender; Hazardous materials; Humans; Males; Middle Aged; Motor neurone disease; Notification; Original Paper; Participation; Patient Selection; Patients; Proximity; Recruitment; Registries; Research Design; Researchers; Review boards; Statistical power; Terminal illnesses; Websites; Young Adult; United States > US; research notification mechanism; Lou Gehrig disease; motor neuron disease; National ALS Registry; patient recruitment; clinical trials; amyotrophic lateral sclerosis
• DOI: 10.2196/28021
• ISSN: 1438-8871; 1439-4456

Researchers face challenges in patient recruitment, especially for rare, fatal diseases such as amyotrophic lateral sclerosis (ALS). These challenges include obtaining sufficient statistical power as well as meeting eligibility requirements such as age, sex, and study proximity. Similarly, persons with ALS (PALS) face difficulty finding and enrolling in research studies for which they are eligible. The aim of this study was to describe how the federal Agency for Toxic Substances and Disease Registry's (ATSDR) National ALS Registry is linking PALS to scientists who are conducting research, clinical trials, and epidemiological studies. Through the Registry's online research notification mechanism (RNM), PALS can elect to be notified about new research opportunities. This mechanism allows researchers to upload a standardized application outlining their study design and objectives, and proof of Institutional Review Board approval. If the application is approved, ATSDR queries the Registry for PALS meeting the study's specific eligibility criteria, and then distributes the researcher's study material and contact information to PALS via email. PALS then need to contact the researcher directly to take part in any research. Such an approach allows ATSDR to protect the confidentiality of Registry enrollees. From 2013 to 2019, a total of 46 institutions around the United States and abroad have leveraged this tool and over 600,000 emails have been sent, resulting in over 2000 patients conservatively recruited for clinical trials and epidemiological studies. Patients between the ages of 60 and 69 had the highest level of participation, whereas those between the ages of 18 and 39 and aged over 80 had the lowest. More males participated (4170/7030, 59.32%) than females (2860/7030, 40.68%). The National ALS Registry's RNM benefits PALS by connecting them to appropriate ALS research. Simultaneously, the system benefits researchers by expediting recruitment, increasing sample size, and efficiently identifying PALS meeting specific eligibility requirements. As more researchers learn about and use this mechanism, both PALS and researchers can hasten research and expand trial options for PALS.