Imagine rebuilding the engine in an ambulance carrying a critically ill patient to the hospital. Got it? Then you have a pretty good idea what it took to put this pediatric rare disease trial back on track. A pharma company developing a drug to treat urea cycle disorder struggled with a CRO that was...
The client asked the nearly impossible — a task so daunting that two other CROs had simply said “no, thanks.” Completing a new drug application typically takes a year to a year and a half, but the time available here was six months. And the short timeline was only the half of it. So we...
For a fertility study, we needed to recruit young women who had had at least three unexplained miscarriagesand who were currently trying to conceive. The field of prospective recruits was huge: all women 18 to 37 years old living in the United Kingdom. The challenge: cost-effectively reach, screen, and randomize 150 patients. The solution: motivate...
A customer asked us to do a chart review of patients with hypophosphatasia, an extremely rare metabolic bone disease with a live birth incidence of about one in 100,000. The perinatal variant is rapidly fatal. A proliferation of protocols Initially informed consent was required if local regulatory authorities and/or IRBs required it. Then the Premier...
Things were complicated enough to start. The customer, conducting a Phase 2/3 dose-finding/pivotal study for an adult growth hormone drug, changed its protocol midstream in response to regulatory feedback and was rethinking other aspects of the trial, adding to its complexity. For weeks, the sponsor’s requirements changed almost daily, severely testing an incumbent lab vendor...
The assignment sounded simple enough: Recruit 24 patients for a Phase 1 proof-of-concept study of inflammatory bowel disease. The first CRO that received the assignment showed how difficult it really was: They were able to recruit only nine patients in a year and a half. At that point, the sponsor asked Premier Research to step...
When does a clinical trial that falls short of its goal still constitute success? When the experience reveals invaluable lessons in how to avoid a repeat performance — and identifies a highly productive location for siting future studies. A large, multinational study of a drug to control inflammation in patients with recurring high-grade malignant glioma...
A customer came to us with a seven-year project to develop a novel human antibody from scratch, to treat multiple myeloma. There were no benchmarks, no relevant data on similar compounds because there were no similar compounds. The first challenge: engage key opinion leaders This project was never going to be easy or “by the...
Our customer was racing to beat a competitor to registration. One critical part of the timeline: Last patient out to database lock in two weeks. In sister studies that involved 800 patients at 90 sites. And several primary investigators who were heading off on vacation just when we needed their sign-offs on the data. We...
A three-nation study of a treatment for recurring glioblastoma showed the therapy more than five times as effective as the typical protocol, an unexpectedly large advance in fighting these aggressive brain tumors. But it was a long road, and the drug’s efficacy was a big factor in extending the trial to five years — nearly...