Search Results for: clinical trials

Ferris Bueller and the Cost of Pivotal Clinical Trials

It turns out asking what a pivotal phase 3 clinical trial costs is a little like asking, “What does a car cost?” The answer will depend on whether the car in question is a $12,000 Nissan Versa, or a $13 million Rolls-Royce Sweptail. Like cars,…Continue Reading

FDA Awards 12 Grants to Fund Clinical Trials to Advance Rare Disease Treatments

Rare Daily Staff The U.S. Food and Drug Administration said that it has awarded 12 new clinical trial research grants totaling more than $18 million over the next four years to enhance the development of medical products for patients with rare diseases. “Developing a treatment…Continue Reading

FDA Clears Ultragenyx to Begin Clinical Trials of Gene Therapy for Glycogen Storage Disease Type Ia

Rare Daily Staff The U.S. Food and Drug Administration gave Ultragenyx Pharmaceutical clearance to begin clinical trials of DTX401, an adeno-associated virus vector-based gene therapy for the treatment of glycogen storage disease type Ia, a genetic enzyme deficiency that result in the inability to regulate…Continue Reading

A Matchmaker for Patients Seeking Clinical Trials

In the past day, I’ve had several rare and common diseases, including some horrendous stuff, but all in the name of science. My sudden bout with a truckload of ailments came about when I learned that the American Liver Foundation had initiated an effort to…Continue Reading

Naia Rare Diseases Gets Green Light for Clinical Trials of Treatment for Short Bowel Syndrome

Rare Daily Staff Naia Rare Diseases, a biopharmaceutical company developing drugs for rare gastrointestinal diseases, said it has received authorization from the Medicine and Healthcare Products Regulatory Agency in the United Kingdom to initiate a clinical trial of NB 1001, its experimental therapeutic for adults…Continue Reading

FDA Awards 15 Grants for Clinical Trials to Stimulate Product Development for Rare Diseases

Rare Daily Staff The U.S. Food and Drug Administration said it awarded 15 new clinical trial research grants totaling more than $22 million over the next four years to boost the development of products for patients with rare diseases. These new grants were awarded to…Continue Reading

Rare Disease Report Interviews Janet Woodcock – Patients, Clinical Trials, and the FDA

At the NORD Rare Disease and Orphan Drug Breakthrough Summit in Arlington Virginia, we talked with Janet Woodcock, MD, Director of the Center for Drug Evaluation and Research (CDER) at the Food and Drug Administration (FDA) about the increasing role patients have in the development…Continue Reading