Comprehensive methods for evaluating safety are needed to objectively assess the full risk
profile of a medication. The confidence of the prescribing provider in the safety and …

Importance A critical question in health care is the extent of scientific evidence that should
be required to establish that a new therapeutic agent has benefits that outweigh its risks …

Certain limitations of evidence available on drugs and devices at the time of market approval
often persist in the post-marketing period. Often, post-marketing research landscape is …

Policy Points Regulatory agencies may have limited evidence on the clinical benefits and
harms of new drugs when deciding whether new therapeutic agents are allowed to enter the …

The COVID-19 pandemic highlighted the importance of timely access to clinical trial results
for public health. Despite decades-long efforts to improve results reporting for clinical …

Real-world evidence (RWE) is the clinical evidence about benefits or risks of medical
products derived from analyzing real world data (RWD), which are data collected through …

Importance Clinical research supporting US Food and Drug Administration (FDA) drug
approvals is largely conducted outside the US. Objective To characterize where drugs were …

Importance Adequate representation of demographic subgroups in premarketing and
postmarketing clinical studies is necessary for understanding the safety and efficacy …

The US Food and Drug Administration (FDA) has 3 regulatory programs intended to
expedite both clinical development and FDA's review of novel therapeutics targeting serious …

Background A small number of regenerative medicines (RMs) have received market
authorization (MA) worldwide, relative to the large number of clinical trials currently being …