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. With Quintiles as your strategic partner, we can help you develop an end to end lifecycle research solution to meet your rare disease research objectives and stakeholder