In rare disease research, it is particularly challenging to find enough patients to conduct rigorous clinical studies. Patients are spread far and wide and are often required to travel long distances — or even move — to access care and participate in studies. And patients with rare neurological diseases often have physical and/or mental disabilities that make travel even more burdensome.
The scarcity of patients and medical experts leads to a lack of "natural history" knowledge about these diseases — what they are, who they impact, how symptoms unfold over time – which is extremely important for developing clinical trials.
"Understanding natural history helps you target the right symptoms, know how to measure those symptoms, and know that those are factors that actually matter to patients and families," said Erika Augustine, M.D., M.S., symposium organizer and Child Neurologist at the URMC Center for Human Experimental Therapeutics.
TRNDS aims to bring together patients, patient advocates, clinical research policy and regulatory experts, and researchers from academia, biotechnology, and pharmaceutical industries. Putting these key players together will hopefully foster new collaborations and new ideas about applying novel mobile health technologies to rare neurological diseases.
At the symposium, biotechnology and pharmaceutical industry leaders will discuss using mobile technology to conduct virtual visits in site-less clinical trials, building digital platforms to engage with patients and the Electronic Health Record, and developing wearable technologies to measure signs of disease. These technological advances could reduce or eliminate travel burden on patients, increase researchers' natural history knowledge about rare neurological diseases, and offer new solutions for tracking symptoms.
Including patients and policy experts in these discussions is also crucial to weigh privacy or confidentiality concerns alongside the great potential these technologies have to benefit patients and research participants.
Technology and Rare Neurological Diseases Symposium (TRNDS)
Friday, May 12, 2017
7:30 am – 3:45 pm
Helen Wood Hall Auditorium, University of Rochester
Questions? Email Erika Augustine or Mulin Xiong.