Drug Development for Rare Diseases

A disease is defined as rare if the prevalence is fewer than 200,000 in the United States. It is estimated that there are more than 7,000 rare diseases, which collectively affect 30 million Americans. This diverse and complex disease area poses challenges for patients, caregivers, regulators, drug developers and other stakeholders.

Auteur

Bo Yang is the Vice President, Biometrics and Real World Evidence at Vertex Pharmaceuticals, USA.

Yang Song is the Executive Director, Biostatistics Group Head of Pipeline Development at Vertex Pharmaceuticals, USA.

Yijie Zhou is the Executive Director, Real World Statistics and Analytics at Vertex Pharmaceuticals, USA.