Rare Disease

Although regulatory incentives and the designation of “orphan” products have stimulated commercial research for rare diseases, their low prevalence, high complexity and predominance in children create significant barriers. Providers and patients are clamoring for treatment access and improved outcomes, but increasingly payers are making hard choices about reimbursing these therapies. As a result, there are gaps in knowledge, a lack of standards and delays in patient diagnosis.

Rare disease compendium thumb
A compendium of blog posts and bylined articles on optimizing rare disease research.

Quintiles Blog

Fresh ideas and insights from our experts around the globe


Quintiles Authors

Vice President and Head, Pediatric Center of Excellence

Senior Vice President, Real-World Insights

Peer-Reviewed Publications
Implications of a data-driven approach to treatment with growth hormone in children with growth hormone deficiency and Turner syndrome.
Appl Health Econ Health Policy. 2013;11(3):237-49.
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Conducting cystic fibrosis clinical trials: methodological, practical and ethical considerations. 
International Pharmaceutical Industry. 2012;4(2):52-6. 
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