If ever a drug development effort called out for priority review and approval, this was it. A U.S. pharmaceutical company was developing a treatment for hypophosphatasia, a progressive, ultra-rare metabolic disease for which only supportive therapy was available. Asfotase alfa, an innovative enzyme replacement drug, was the first real hope for those diagnosed with HPP,...
Speakers: Sachin KulkarniExecutive Director, Strategic Development, Oncology & General MedicinePremier Research Juliet M. MoritzExecutive Director, Strategic Development, Rare DiseasesPremier Research Study of rare cancers tends to look more like rare disease research than standard oncology research. This webinar will highlight some of the challenges in designing and conducting trials for rare cancers and explore positions...
Researching rare and ultra-rare diseases sometimes means taking the study to the patients, setting up sites in countries unaccustomed to hosting clinical research. Premier Research will examines the challenges of conducting trials in such unconventional locales in this webinar. Opening sites in countries where trials are uncommon and not thoroughly regulated requires that sponsors really...
Speaker: Angi Robinson, Executive Director, Pediatrics and Rare Diseases, Premier Research This session includes a regulatory overview of rare diseases and orphan drug status as well as practical considerations for varying stages of the development process. Viewers will learn more about conducting successful Natural History studies and the presenters will review how to avoid common...
7,000 rare diseases, many untreatable. 50% of patients are children. 35% of deaths in the first year of life are due to rare diseases. To everyone at Premier Research, that’s a call to action. So Premier Research employees worldwide will join together on Friday, February 27, to raise awareness of Rare Disease Day 2015.
PHILADELPHIA, March 18, 2013 — Premier Research today announced the addition of four talented clinicians to key leadership roles in its fast-growing pediatric and rare disease clinical trial research disciplines. The Philadelphia-based global contract research organization (CRO) named Dr. Susan P. Tansey, MBCHB, MRCP, CCST, medical director, pediatrics. Her medical specialty areas include vaccines, cardiovascular, and oncology, although her initial training was in neonatology and pediatric medicine. She has considerable experience in pediatric clinical research and trial design.
A new survey of clinical trial decision makers commissioned by Premier Research and involving 50 biotech and pharmaceutical firms in North America and Europe reveals that more than two-thirds (69%) of respondents said that among the most difficult factors in recruiting patients into a rare disease clinical trial was not only finding and motivating patients to join and remain in trials, but identifying and setting up investigative sites for studies.