The Art of Decentralizing Pediatric Rare Disease Studies: Clinical Trials Suited for Daily Life

Pediatric rare disease trials combine rarity of disease with a restricted and geographically-dispersed population of vulnerable subjects for research, creating unique challenges for study design and execution. Recruitment is challenging, as patients, parents, and caregivers need to juggle school schedules, work responsibilities, and family obligations to accommodate study participation. Incorporating decentralized clinical trial (DCT) strategies...

The Art of Decentralizing Pediatric Rare Disease Studies: Clinical Trials Suited for Daily Life

Meet Our Speakers: Betsy ReidSenior Vice President, Pediatrics Adam Bloomfield, MD, FAAPExecutive Director, Medical Affairs, Rare Diseases and Pediatrics

The Art of Decentralizing Pediatric Rare Disease Studies: Clinical Trials Suited for Daily Life

September 28th, 202311:00 a.m. EDT Pediatric rare disease trials combine rarity of disease with a restricted and geographically-dispersed population of vulnerable subjects for research, creating unique challenges for study design and execution. Recruitment is challenging, as patients, parents, and caregivers need to juggle school schedules, work responsibilities, and family obligations to accommodate study participation. Incorporating...

Clinical Research: Phase 1 - Phase 4

Food for Thought: The Power of Decentralized Clinical Trials in Accelerating Infant Formula Studies

Infant formula is an essential food product that serves as the sole source of nutrition for many babies in North America and Europe during their first year of life, supporting health, growth, and development.1,2 More than a year since the shutdown of the largest infant formula plant in the US in February 2022, the country...

Clinical Research: Phase 1 - Phase 4

Resources for Childhood Cancer Awareness Month

Premier Research is dedicated to helping patients and families around the world overcome the burden of debilitating diseases, such as a cancer diagnosis. Cancer is a leading cause of death for children and adolescents, and there are approximately 300,000 children diagnosed with cancer each year around the world.1 With the help of better therapies, more...

Medical and Regulatory Affairs

The FDA Is Ending Its Rare Pediatric Disease Priority Review Vouchers – and Time Is Running Out to Get One

Between 2010 and 2018, 33.8 percent of the 402 orphan indications approved by the FDA were approved for children only or targeted pediatric diseases.[1] The rare pediatric disease priority review voucher program is one incentive that’s helped to spur research and development in this area with more than 20 vouchers granted to date, but it...

Clinical Research: Phase 1 - Phase 4

Addressing a Critical Unmet Need: Considerations for Designing and Conducting Neonatal Clinical Trials

Introduction Due to the challenges associated with studying investigational medical products in neonates, healthcare professionals must often resort to administering drugs in neonatal intensive care units (NICUs) off-label. While numerous pediatric legislative initiatives have been passed worldwide, neonates are still largely treated with medicines that have not been specifically studied or approved in this young,...

Clinical Research: Phase 1 - Phase 4

Neonatal Research Insights: Conducting Clinical Trials in the Smallest Patients

Despite the passage of various legislative initiatives aimed at increasing the frequency and quality of pediatric clinical trials, neonates continue to be treated with medicines that have not been studied or approved in this population.1 Historically, sponsors may have felt that the ethical and logistical challenges limited the opportunities for neonatal research. Our experience has...

Medical and Regulatory Affairs

iPSP Considerations for Molecularly Targeted Cancer Drugs

With the passage of the Pediatric Research Equity Act (PREA) of 2003, drug manufacturers were required to submit an initial pediatric study plan (iPSP) prior to commencement of Phase 3 studies (or new drug application [NDA]/biologics license application [BLA] submission in the absence of a Phase 3 study) for studies involving a new active ingredient,...

Medical and Regulatory Affairs

5 Things to Know About the FDA RACE for Children Act

The Research to Accelerate Cures and Equity (RACE) for Children Act aims to improve and expand treatment options for pediatric cancer patients by mandating that all new adult oncology drugs also be tested in children when the molecular targets are relevant to a particular childhood cancer. Enacted August 18, 2017, as part of the Food...