PharmaVOICE Blog Post

Registries in Rare Disease Research – Approaches to Optimize Success

Posted By: Dan Limbach
April 15, 2019

Submitted by:  Linda Ross, MPH, Senior Director, Peri- and Post-Approval Operations, Evidera

Advancements in gene therapy and transformative medicine have had a major impact on the development of treatments for rare diseases, resulting in a growing need for evidence of the safety, effectiveness, and value of these treatments in the real world. Opportunities to improve healthcare, clinical outcomes, and patient and caregiver quality of life are abundant, but for successful market access of novel treatments, robust – and frequently longitudinal – clinical and outcomes data from the usual care setting are necessary. A significant obstacle to collecting this data, however, is the low number of patients with the disease being studied. The US Food and Drug Administration (FDA) classifies a rare disease as any disease or condition affecting less than 200,000 patients in the US. Hence, the identification and long-term engagement and retention of these patients is a primary challenge.

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Key topics in the paper:

  • Registries can be an incredibly useful tool in gathering data on patient usual care, current treatment landscape(s), and long-term clinical outcomes
  • Success of a registry hinges largely on the study design
  • Study design for treatment-specific and disease-specific registries
  • Key factors for increased treatment-specific and disease-specific registry engagement
  • Importance of patient centricity associated with geographical regions (North America & Europe, Latin America & Asia Pacific)

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About the Blog Poster: Dan Limbach

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