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...
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...
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...
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....
It would be hard to overstate the need for new therapies that target unmet medical needs, especially in rare disease. After all, there are only about 400 approved “orphan drugs,” meaning that 95 percent of rare diseases lack a single approved treatment. Now the good news: U.S. and European regulators, recognizing the size and severity...
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...
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...
Today’s Tales from the Road comes from Corporate Recruiter, Raiza Rivera, who attended Come Tech Out the Triangle earlier this month. We exhibited at this tech-focused job fair in hopes of recruiting for several open technology positions at Premier. “I came to the job fair today because I saw that Premier Research was on the list...