Simplifying the complexities of global rare disease drug development

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September 15, 2016

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While rare diseases individually affect very few patients, the collective impact is staggering. Globally, the list of rare diseases has grown to approximately 7,000, and 350 million people are living with a rare disease. These diseases are serious, often life-threatening, and approximately half of those affected are children. The lack of available treatments leave patients and their families searching for new options and new hope.

In this webinar, QuintilesIMS experts Dr. Cynthia Jackson and Sheetal Telang address current industry challenges of rare and ultra-rare disease drug development and why developing seamless, efficient global operational solutions designed with the patient at heart is critical to help accelerate development of new treatments.

You will learn:

  • How to put together a patient-centric global operational strategy for rare disease drug development
  • The importance of creating a site strategy that supports development goals and facilitates patient recruitment
  • How to effectively transport clinical supplies and importance of "just in time" drug delivery
  • The importance of using centralized, remote monitoring to support on-site monitoring of key study visits and data points
  • Ways to effectively and safely transport patients (particularly pediatric patients) to and from sites and across borders, as well as suggestions for handing interim care between visits
  • How to use strategic insights and real-world data earlier in the lifecycle to develop clearer drug development pathways and endpoints, as well as smarter trial designs


Cynthia Jackson, D.O., FAAP
Vice President and Head, Pediatric and Rare Disease Centers of Excellence

Sheetal Telang
Therapeutic Strategy Director, Immunology and Internal Medicine, Therapeutic Science & Strategy Unit