When you’re looking into conducting a natural history study, you’ve got two things to consider up front: the premise that underlies all of these studies, and which of several types suits your purpose. We tackled these and other topics in our last rare disease webinar. Natural History vs. Registry Studies in Rare Disease considers a...
DURHAM, N.C., AUGUST 14, 2017 — Rare diseases afflict tens of millions of people, and for professionals seeking to treat these conditions, it can be tempting to regard patients more as diagnoses and outcomes than as folks seeking to lead otherwise normal lives. That’s why Premier Research has signed on as a presenting sponsor of...
Adaptive design is a type of clinical trial methodology that incorporates prospectively planned opportunities for modification of one or more aspects of a study’s design or its hypotheses based on interim analysis of study data. Explicitly planning these pre-specified changes helps to maintain scientific integrity while also introducing greater flexibility in the clinical research environment. The major...
DURHAM, N.C., JULY 24, 2017 — Premier Research has named Kimberly Glen an Executive Director for Rare Disease, Strategic Development Department. She brings to the company nearly three decades of experience in contract research, drug development, and pharmaceutical consulting. “Rare disease is an extremely demanding field of study, imposing unique challenges in patient recruitment, deriving...
Both understanding the role of patient advocacy groups in building better clinical trials and improving the research process mean first taking a look at the history of orphan drug development in the United States and Europe. An “orphan drug” is a drug that treats a condition that fewer than 200,000 people have. Because rare diseases...
A little less than five percent of the world’s population are estimated to suffer from more than 7,000 different types of known rare diseases. There are even more medical conditions where those who have them are too embarrassed to talk about what’s wrong or simply don’t know exactly what ails them. Yet, these individuals are...
Knowledge of a disease’s natural history is essential to building the scientific foundation for an effective clinical development program. That’s especially the case when developing drugs to treat rare diseases, which by their very nature are poorly understood. Natural history studies don’t just track the course of diseases over time, but identify demographic, genetic, environmental,...
Patient registry and natural history studies are often thought of as interchangeable, but there are major differences in these approaches to clinical drug development. Understanding the role of each is important, as well as understanding the distinctions on how these study designs are used in pursuing treatments for rare diseases. Patient Registries A patient registry...
The effects of rigorous patient advocacy are easily seen today throughout the pharmaceutical industry. Patients who suffer the debilitating effects of rare diseases are banding together to be heard in an attempt to have their conditions studied to further the development of treatments and cures. However, the influence of patient advocacy groups has not always...
Translational research has the potential to help all patients, but the principles behind bench-to-bedside research hold special promise for patients with rare diseases — many of which have no standard treatments. Here’s how changes to the way rare oncology studies are performed are getting treatments to the patients who need them at a faster rate. What Makes...