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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. |
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| Rare
disease studies are complex and challenging. With the lack of an
established operational pathway to follow, biopharmaceutical companies
with rare and ultra-rare disease drugs in development face unique issues
throughout the clinical development process including: |
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Limited disease knowledge such as clinical course |
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Undefined clinical endpoints |
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New regulatory pathways |
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Few patients, many countries |
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Site targeting and engagement challenges |
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Post-marketing commitments often required |
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Unknown obstacles due to no preexisting pathways |
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| Quintiles,
with experience in 245 rare disease studies across 96 countries, helps
anticipate and address the complex challenges of rare disease studies by
bringing together a specialized team with rare disease experience and
therapeutic expertise — along with tailored patient-centric approaches
to global study planning and execution in order to speed treatments to
market that will transform the lives of patients and their families. We
want to share our knowledge with you. |
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| In this webinar, Quintiles experts Dr. Cynthia Jackson and new treatments. |
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| You will learn: |
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How to put together a patient-centric global operational strategy for rare disease drug development |
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The importance of creating a site strategy that supports development goals and facilitates patient recruitment |
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How to effectively transport clinical supplies and importance of "just in time" drug delivery |
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The importance of using centralized, remote monitoring to points |
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Ways to effectively and safely transport patients care between visits |
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How to use strategic insights and real-world data earlier inand endpoints, as well as smarter trial designs |
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| Presenters: |
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Cynthia Jackson, D.O., FAAP
Vice President and Head, Pediatric and Rare Disease Centers of Excellence at Quintiles
Sheetal Telang
Therapeutic Strategy Director, Immunology and Internal
Medicine, Therapeutic Science & Strategy Unit at Quintiles |
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| Moderator: |
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Lisa Henderson
Editorial Director, Applied Clinical Trials |
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US Toll free: 1 866 267 4479
US Direct: +1 973 850 7571
Non US: +44 203 564 4649
www.quintiles.com
Clinical@quintiles.com |
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For technical questions about this webinar,
please contact Kristen Moore at kristen.moore@ubm.com |
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