Drug Trials Snapshots: IBTROZI

by Pill Pals Customer Service | Sep 4, 2025 | News

HOW TO USE THIS SNAPSHOT The information provided in Snapshots highlights who participated in the key clinical trials that supported the original FDA approval of this drug, and whether there were differences among sex, race, age, and ethnic groups. The “MORE INFO” bar...

FDA Advances Rare Disease Drug Development with New Evidence Principles

by Pill Pals Customer Service | Sep 3, 2025 | News

For Immediate Release: September 03, 2025 The U.S. Food and Drug Administration today introduced the Rare Disease Evidence Principles (RDEP) to provide greater speed and predictability in the review of therapies intended to treat rare diseases with very small patient...

CDER/CBER Rare Disease Evidence Principles (RDEP)

by Pill Pals Customer Service | Sep 3, 2025 | News

SummaryFDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) propose a new process under Rare Disease Evidence Principles (RDEP) to facilitate the approval of drugs to treat rare diseases with very small...

FDA Approves Drug to Treat Adults with Persistent or Chronic ITP

by Pill Pals Customer Service | Sep 2, 2025 | News

ActionThe U.S. Food and Drug Administration (FDA) approved Wayrilz (rilzabrutinib) tablets to treat adults with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to immunoglobulins, anti-D therapy, or corticosteroids. Disease...

Efficacy Endpoints in Ophthalmologic Rare Disease Drug Development

by Pill Pals Customer Service | Sep 2, 2025 | News

On This Page Date: September 17, 2025 Time: 9:30 a.m. – 2:30 p.m. ET The Duke-Margolis Institute for Health Policy, under a cooperative agreement with the U.S. Food and Drug Administration, is convening a one-day hybrid public meeting on September 17, 2025...

Drug Safety Oversight Board

by Pill Pals Customer Service | Sep 2, 2025 | News

BackgroundThe DSB, created in 2005 and mandated by law in the FDA Amendments Act of 2007, advises the CDER Center Director on the handling and communicating of important and often emerging drug safety issues. The DSB meets to provide a forum for discussion and input...