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...
A target product profile (TPP) is a strategic process tool that streamlines medical device development through cross-functional strategic alignment. It’s a living document that defines the value proposition and key differentiators of an intended commercial product while assisting in product development strategy. A TPP may take a lot of time and resources to craft, but it’s a strategy that promises a major...
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...
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...
As it was starting to get cold in the Northeast, a group of us snowbirded down to warm Florida for the CNS Summit. Before the conference began, we participated in STARR Coalition’s annual meeting. STARR Coalition is a remarkable group whose mission is to create meaningful change by increasing communication, partnerships, and goodwill among stakeholders...
Britain’s plans to leave the European Union could profoundly affect clinical drug research in the U.K. The impact spans a broad range of unknowns, from where the London-based EMA will put down roots to Brexit’s influence on trial regulations, cost, funding, and recruiting of scientific talent. Our Chief Medical Officer, Dr. Colin Hayward, addressed these...