Purpose The breakthrough therapy program was established in 2012 to expedite the
development and review of new medicines. We evaluated the times to approval, efficacy …

Background: The breakthrough therapy designation (BTD) facilitates the development of
drugs with a large preliminary benefit in treating serious or life-threatening diseases. This …

The breakthrough therapy designation, established in 2012 by the US Congress to expedite
the development of drugs that show promising early clinical evidence of benefit over …

Importance Clinical trial evidence used to support drug approval is typically the only
information on benefits and harms that patients and clinicians can use for decision-making …

Abstract In July 2012, Congress passed the Food and Drug Administration Safety and
Innovation Act (FDASIA). The Advancing Breakthrough Therapies for Patients Act was …

Background The clinical benefit and pricing of breakthrough‐designated cancer drugs are
uncertain. This study compares the magnitude of the clinical benefit and monthly price of …

With the Breakthrough Therapy Designation program adding to the tools that the US Food
and Drug Administration (FDA) has for expediting drug development, the FDA reassessed …

PURPOSE Clinical trial evidence is routinely evaluated for initial drug approvals, yet the
benefit of indication extensions remains uncertain. This study evaluates the clinical benefit …

Background This study analyzes the development, US Food and Drug Administration (FDA)
approval, benefits, innovation, trials, epidemiology, and price of cancer drugs with multiple …

Concerns have been raised that regulatory programs to accelerate approval of cancer drugs
in cancer may increase uncertainty about benefits and harms for survival and quality of life …