Clinical Research: Phase 1 - Phase 4

The Science and Art of Conducting Clinical Trial Feasibility in Rare Disease and Pediatric Studies

Rare disease and pediatric studies present significant challenges, including country selection, site selection, and patient recruitment. A thoughtfully-designed, well-executed feasibility study can help sponsors obtain a realistic assessment of the best path forward for developing and conducting successful global clinical trials. Performing a thorough assessment of clinical trial feasibility is an important early step in...

Patient and Stakeholder Engagement

Tales from the Road – World Congress Patient Advocacy Summit

This week’s Tales from the Road is from Angi Robison, Executive Director, Strategic Development Department, Rare Disease & Pediatrics, who gives us a bit about what she took away from the World Congress Patient Advocacy Summit back in October. Greetings from the road! I just had an excellent two days at the World Congress Patient Advocacy...

Data Management & Biostatistics

Premier Insight 238: Back On Track: Data Rescue On Pediatric Rare Disease Trial

  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...

Patient and Stakeholder Engagement

Premier Insight 242: How Ramping Up Communications Helped Overcome a Next-to- Impossible Recruitment Challenge

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...


Premier Insight 263: The Case of the Elusive Protocol

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...

Adult Drug Studies Play Important Role as Need for Pediatric Research Grows

Jennifer Strickler will describe the main differences between pediatric and adult study designs, outline the medical considerations pediatric protocols should include, and break down protocol elements specific to children and family participation.

Patient and Stakeholder Engagement

Premier Research’s New Pediatric Survey Reveals Challenge in Identifying Sufficient Clinical Patients

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.

Patient and Stakeholder Engagement

Premier Research Experts to Examine Pediatric Studies at DIA 2014

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.

Premier Research Augments Staff in Fast-Growing Pediatrics and Rare Disease Clinical Trials Division

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.

Medical and Regulatory Affairs

Premier Research Survey Finds Many Sponsors Ignore Pediatric Requirements for Clinical Trials

The Pediatric Research Equity Act (PREA)—requiring drug makers to test all drugs for safety and efficacy in pediatric populations as well as in adults—was passed in 2003 and updated in 2007. And in Europe, a similar requirement has been in effect since 2007.