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...
We knew going in that it could be the perfect recruiting nightmare. We were looking for children ages 2 to 12 for a dermatology study that involved long visits and extensive blood draws. We had extremely complex inclusion/exclusion criteria. And most parents wanted nothing to do with it. “Hello, it’s me again.” We sent feasibility...
A trans-Atlantic study to evaluate an antibody for treatment of B-cell non-Hodgkins lymphoma is still almost three years from completing patient follow-up, but already it has succeeded beyond expectations in the form of five patients declared disease-free a year and counting. The Phase 2 trial involved a CD19 antibody that was tested at 26 sites...
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...
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...
PHILADELPHIA, December 15, 2015 — 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 examine the challenges of conducting trials in such unconventional locales at a webinar on Tuesday, Dec. 15. Opening sites in countries where trials are...
A new survey of clinical trial decision makers commissioned by Premier Research reports that identifying an adequate number of children suitable to be patients is far and away the single greatest challenge in conducting a pediatric clinical trial. The Premier Research survey also revealed a surprising lack of awareness among clinical trial sponsors that, as part of the Safety and Innovation Act (FDASIA), FDA will award priority review vouchers to sponsors of rare pediatric disease product applications that meet specific criteria.
A new survey of clinical trial decision makers commissioned by Premier Research reports that nearly two-thirds (64%) of respondents said that among the most difficult factors in recruiting for a rare disease or orphan drug clinical trial was not only finding patients to join and remain in trials, but identifying and setting up investigative sites for studies.
On Thursday, June 12, 2014, Premier Research announced that a panel of their experts will discuss the design of pediatric studies, recruiting for pediatric research, and the importance of corrective and preventive actions to a company’s quality system at the 2014 Drug Information Association conference, June 15–19 in San Diego.
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.