How Technology is Reshaping the Rare Disease Landscape

Although transitioning from a traditional site-centric paradigm to a patient-focused connected trial model may seem daunting, this white paper provides practical strategies for easing the participation burden and introduces considerations for selecting technology that can seamlessly integrate clinical trials into patients’ lives.

Executive Summary

Technology is transforming clinical drug development. In the rare disease space, clinical drug development is moving away from traditional approaches that rely on patients to travel long distances to clinical sites and toward a more patient-focused paradigm. This new paradigm is especially suited to studies in patients with rare diseases; these studies target small populations of geographically dispersed patients who are rarely located near academic medical centers or key treatment centers. By bringing trials to patients, instead of requiring patients to travel to sites, we can alleviate a primary burden of clinical trial participation and democratize clinical trial access. This new approach—highlighting virtual and connected trials—is more flexible and feasible for patients and leverages available innovations in technology.



Scott Schliebner, MPH, Vice President, Scientific Affairs - Rare Diseases 

+1 (206) 795 - 5273 |

Brad Pruitt, MD, Executive Medical Director and CMO, Clinical6 - A PRA Health Sciences Company

+1 (619) 813 - 7059 |

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