With experience of helping develop or commercialize 100% of the top 30 best- selling orphan products or compounds in 2013, Quintiles understands the unique challenges in rare disease research and can help you generate the evidence you need to meet evolving stakeholder demands, whether for regulators, payers, providers or, most importantly, patients.
With the list of rare diseases across the globe expanding to nearly 7,000 it is critical to make new treatments available for these uncommon conditions.
While 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. Real-world evidence can play a huge role in expediting the development and commercialization of orphan medical products, and Quintiles can help you meet your rare disease research objectives.
Download our brochure, “Developing evidence for medical breakthroughs: Real-world & late phase expertise in rare diseases” to learn more about Quintiles’ expertise in rare disease research.
Visit us at the World Orphan Drug Congress in Europe to see how you can achieve better patient outcomes for your orphan products with comprehensive evidence development solutions to:
- Monitor safety and evaluate benefit-risk
- Demonstrate effectiveness and efficacy
- Gain market access and expand labeling/approved indications
- Understand natural history and treatment
Quintiles helps navigate the rare disease research landscape. Learn more at www.quintiles.com.
Download our brochure here.