Background Over the past decades, US Congress enabled the US Food and Drug
Administration (FDA) to facilitate and expedite drug development for serious conditions …

Background Surrogates are frequently used in cancer medicine as the end-point of clinical
trials and as the basis of United States Food and Drug Administration approvals, but they do …

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 …

Objective To characterize the therapeutic value of new drugs approved by the US Food and
Drug Administration (FDA) and European Medicines Agency (EMA) and the association …

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 …

Importance Since the introduction of the Fast Track designation in 1988, the number of
special regulatory programs available for the approval of new drugs and biologics by the US …

In March, 2019, the US Food and Drug Administration (FDA) approved two new
antidepressants: esketamine for treatment-resistant depression1 and brexanolone for …

Fewer than half of new drugs have data on their comparative benefits and harms against
existing treatment options at the time of regulatory approval in Europe and the USA. Even …

Highlights•Breakthrough-therapy-designated (BTD) drugs in China can significantly reduce
drug development time.•Simultaneous BTD applications in the US and China might be …

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