A rare disease, also referred to as an orphan disease, by definition affects a small percentage of the population. Most are genetic and appear early in life, with one in three children with a rare disease dying before their fifth birthday. While the definition of “rare” varies from country to country, the US defines a rare disease as...
A rare cancer is often defined as one with an incidence of less than 15 per 100,000. The patient populations of these conditions are so small that traditional clinical trial design strategies can be unfeasible. But, with such a low incidence, just why is rare oncology research so important? Why Rare Oncology Needs Innovation Keep in mind...
When developing treatments for a relatively common illness or disease, patient recruitment is relatively straightforward. Screening and data collection can generally be done in a centralized location in a highly controlled environment. Now, imagine the concentration of people with the disease is not high enough in your local area, or even your country for that...
Visit the iTunes App Store or Google Play and you will find the smartphone app PoopMD+: A New Parent’s Guide to Baby Poop and Pediatric Liver Disease Study from Johns Hopkins. PoopMD+ is an imaging and clinical survey-based app developed to help screen for a rare but life-threatening liver disorder in babies and collect feedback...
A target product profile is a document that presents a polished explanation and aggregation of all relevant information needed in validating product development. This common template can be used for all products across a company portfolio to guide and align regulatory, preclinical, clinical, marketing, and health economic outcomes and reimbursement strategies. The concept of the TPP was originally developed...
Virtual Reality’s mainstream use in clinical research is no longer a fantasy. At the recent National Institutes of Health (NIH) Research Festival in America’s capital, attending clinical researchers were encouraged to strap on head-mounted virtual reality (VR) devices and image how they might enhance their clinical and basic research. The NIH even showcased its Virtual...
Speakers: Patricia Nowowieski, Head of Global Clinical Operations for Alexion Pharmaceuticals. Patricia’s work in global clinical trials involves specialized populations and complex logistical and regulatory challenges. Angi Robinson, Executive Director and Scientific Account Leader for Premier Research. Angi’s extensive experience includes oversight of global studies in pediatrics and rare diseases. Mallory Bissett, Associate Director of...
When it comes to clinical trials for neurodegenerative disease (NDD), it’s easy to get overwhelmed by complexity. Not only are the mechanisms that underlie NDDs like multiple sclerosis and Alzheimer’s still largely a mystery, their inherent variability is hard to account for. The best way to combat the problem? Break trial design down into more manageable pieces....
Last week, I attended World Orphan Drug Congress in Belgium. It was my first WODC, and it was a productive three days in Brussels – even if it was held at the airport hotel, and we never left the building! The congress agenda covered a wide range of topics in the rare and orphan disease...
Speakers:Anne-Marie Nagy, Ph.D., Executive Director, Strategic Development, Neuroscience, Premier ResearchTodd Leathers, MBA, Executive Director, Strategic Development, Neuroscience, Premier Research Neurodegenerative disorders present some of the biggest challenges in planning and conducting clinical drug trials. Diagnosis alone is tricky, given the often-delayed onset of symptoms, and optimizing study design, recruitment, event adjudication, and biomarker use complicates...