Drug developers and regulators are expanding the use of real world evidence to design, test and review rare disease treatments. But market access challenges persist as payers in the U.S. and Europe continue to express doubts. A new survey sheds light on the perception gap. A striking life sciences trend ...

Contributions to keep celebrating Whether they were the first to break down barriers or win a Nobel Prize, women have been essential to the advancement of science, technology, engineering, and mathematics. Their contributions have changed the world and it’s only just beginning. Featured articles Championing women in science Meet some ...

RARE Science, an all-volunteer San Diego nonprofit dedicated to accelerating diagnosis and therapeutic development for kids with rare disease, gifted 200 RARE Bears to local nonprofit Fresh Start Surgical Gifts through its RARE Bear Program created to raise awareness for children’s rare disease. Fresh Start provides free-of-cost reconstructive surgery for ...