If new restrictions are placed on the FDA’s Accelerated Approval pathway, as many as two thirds of the treatments that use this pathway would never reach patients, according to research released at the BIO International Convention last week.
A new multi-stakeholder group of experts has been set up to provide input and ideas in the context of the ongoing evaluation of the orphan medicinal products (OMP) regulation in Europe.
As technology has advanced, rare diseases have been met with therapies that target the cause of the condition, enabling the global market for orphan drugs to reach an expected $262bn by 2024.
