Introducing Tales from the Road, a series of blog shorts, where our Premier People report in from their travels outside the office. Because sometimes, not everything that happens on the road stays on the road! Today’s inaugural post comes from Juliet Moritz, Executive Director, Strategic Development Department, Rare Diseases, reporting back on her trip to Amsterdam...
There are more than 7,000 known rare diseases, and these conditions afflict an estimated 30 million Americans (almost one in 10). Worldwide, some 350 million are living with a rare disease. But for 95 percent of these conditions, there are no FDA-approved treatments — not one. Conventional wisdom holds that innovation follows need: that if...
Quality and effective risk management are vital to every clinical trial, and their importance is greater still when working in rare indications. Your patient population is small, vulnerable, and geographically dispersed, so there’s limited data and little opportunity to replicate clinical trial results. It’s as if the phrase “get it right the first time” had...
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...
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.