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...
The study of rare cancers poses special challenges for drug developers, who often must draw on their experience in both oncology and rare disease. Careful consideration of clinical trial design and regulatory pathways can help increase the likelihood of success in rare oncology clinical trials. Researchers studying rare cancers must call on expertise in both...
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...
“Rare disease” may be the biggest misnomer in the pharmaceutical industry. Around the world, 350 million people live with conditions classified as rare because each afflicts a relatively small segment of the population. In the U.S., 30 million—nearly one in 10 Americans—suffer from one of about 7,000 known rare diseases. As it applies to this...
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,...
What do you do when patients are too embarrassed to talk about what’s wrong with them? It was a trial for fecal incontinence, and patients wanted help – they just didn’t want to admit it. We needed a new outreach and recruitment process, so we tried radio, outdoor, and subway. We rigorously tracked what worked...
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...