戻る Agenda
Recruiting Rare Disease Patients: A Unique Set of Challenges
Session Chair(s)
William B. Smith, MD
CEO
Alliance for Multispecialty Research, LLC , United States
It is estimated that about one-third of clinical trial failures may be due to enrollment challenges. Rare disease trials are no exception and encounter many patient recruitment and retention difficulties. Small numbers of patients who are spread over large geographic areas, as well as singular aspects of specific diseases that may not be well understood, contribute to the unique set of challenges faced by rare disease studies. Traditional methods for recruitment of patients are frequently not adequate to populate rare disease trials. This session will discuss the importance of tailoring the rare disease research process to the specific patient population to be studied, from early trial design and protocol planning through enrollment and study conduct. Successful approaches to improving recruitment through collaboration with patient groups in early planning stages and identification of patients through innovative analyses of real world data will be examined.
Learning Objective : Identify new tools leveraging real-world data and innovation in advanced analytics including the growing field of machine learning technology, and how the advancements in this field are creating opportunities for dramatic improvements in identifying hard-to-find patients.
Speaker(s)
Big Data with Investigator and Patient Advocacy Outreach in Idiopathic Pulmonary Fibrosis: A More Complete Picture
Adina Kay Knight, MD
QuintilesIMS, United States
Senior Medical Director