JMIR Publications

ABSTRACT

Background:

Researchers face challenges in patient recruitment, especially for rare, fatal diseases like 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.

Objective:

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.

Methods:

Through the Registry’s online Research Notification Mechanism, 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 (IRB) 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.

Results:

From 2013 – 2019, a total of 46 institutions around the US. have leveraged this tool and over 600,000 emails have been sent resulting conservatively in over 2,000 patients recruited for clinical trials and epidemiological studies.

Conclusions:

The National ALS Registry’s Research Notification Mechanism 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.