Quintiles Marks Rare Disease Day

RESEARCH TRIANGLE PARK, N.C.--Marking the recent commemoration of ‘World Rare Disease Day’, Quintiles points to the 350 million people around the globe suffering from rare diseases – gathered together they would be the world’s third most populous nation – by highlighting its own contribution to tackling this challenge with a new collection of papers, podcasts and videos covering the latest developments and insights. A rare disease is one that affects fewer than 200,000 Americans at any given time, according to the U.S. Food and Drug Administration.[i] In Europe, a disease or disorder is defined as rare when it affects less than 1 in 2000.[ii] Milena Kanova, M.D., senior director, project management at Quintiles, explains. “Because rare diseases occur in relatively small numbers of people, patient recruitment can pose one of the greatest challenges in these type of clinical trials.”

Quintiles has been involved in more than 80 rare disease clinical trials, involving almost 11,000 patients in 52 countries. Based on that experience, the released today presents insights and perspectives on the design of clinical trials for rare diseases, drug commercialization and patient outcomes.

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Supporting the publication of these insights, in a new podcast, Dr. Kanova explains the approach used for rare disease clinical trials and how the model can be adapted to address any rare disease. This approach involves designing trials to include data-driven monitoring, triggered initiation of sites and recruitment planning tailored for the rare disease space.

In addition to clinical development, Quintiles’ global footprint helps improve patient-reported outcomes. By partnering with sponsors and research organizations, Quintiles has been able to develop numerous registries for rare diseases, including hemophilia, cystic fibrosis and rare cancer indications, among others.

“The depth of our market penetration allows us to conduct relatively large long-term studies, including some that aim to capture information on every patient treated with a particular product,” said Kathryn Starzyk, Sc.M., director of epidemiology, Real-World Late Phase Research at Quintiles.

Amongst the new insights, John Doyle, Dr.PH., M.P.H., senior vice president and managing director, Consulting at Quintiles and Jeremy Broadis, vice president, Commercial Solutions at Quintiles, discuss considerations for developing a go-to-market strategy, including access to physicians, market access and medical education versus traditional sales activities.

Additional insight and perspective on rare disease drug development are available on the company’s website.

About Quintiles
Quintiles (NYSE: Q) is the world’s largest provider of biopharmaceutical development and commercial outsourcing services with a network of more than 28,000 employees conducting business in approximately 100 countries. We have helped develop or commercialize all of the top-50 best-selling drugs on the market. Quintiles applies the breadth and depth of our service offerings along with extensive therapeutic, scientific and analytics expertise to help our customers navigate an increasingly complex healthcare environment as they seek to improve efficiency and effectiveness in the delivery of better healthcare outcomes. To learn more about Quintiles, please visit www.quintiles.com.