This insight brief examines the varied definitions and regulatory pathways for rare disease therapies. The authors describe QuintilesIMS’s proven patient centric approaches to study planning, innovative ways to accelerate patient recruitment through an efficient site strategy, and the importance of real-world evidence.
There are at least 7,000 known rare diseases, affecting 1 in 10 Americans and a total of 350 million people worldwide, and causing 35% of deaths in the first year of life. Only 5% of rare diseases have a Food and Drug Administration (FDA)-approved therapy, and more than 450 rare disease therapies are currently in development. It has been estimated that rare diseases will account for around 19% of the worldwide prescription drug market by 2020.3 This insight brief examines the varied definitions and regulatory pathways for rare disease therapies. The authors describe QuintilesIMS’s proven patientcentric approaches to study planning, innovative ways to accelerate patient recruitment through an efficient site strategy, and the importance of real-world evidence.
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Regards,
World Orphan Drug Congress USA