Duchenne muscular dystrophy (DMD) patients almost universally lose the ability to walk as the disease progresses. Patients generally understand that loss of ambulation is to be expected, and as was learned from a patient preference study, they tend to put greater priority on maintaining use of their hands. Yet for many years, DMD drug trials...
At Premier Research, we attend a lot of events throughout the year – and through these events, we have the opportunity to meet individuals from all sides of the clinical development process. At the recent Global Genes RARE Patient Advocacy Summit, we met countless patients, advocates, and their families, including Kim Tuminello and her daughter...
Patient advocacy groups have exploded in number and scope in recent years and in many ways are reshaping the drug development landscape, from trial design to recruitment support to participation in the regulatory approval process. Thousands of organizations now advocate for millions of patients, and while many patients and families eagerly engage with these groups,...
Finding patients for trials in very rare conditions is difficult under any circumstances, and in this case the odds were really stacked high against success. The indication: a basal cell carcinoma known as Gorlin syndrome, a disease that manifests itself in large numbers of benign tumors that grow on various parts of the body. The...
Every so often, you stumble onto a marketing partnership that just seems to fit. When we were approached earlier this year about a rare disease festival happening in October, we knew immediately that we had to be a part of it. Who could pass up the opportunity to be part of a first-of-its-kind event that...
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 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...
Despite advances in the orphan drug development process and the more prominent role that patient advocacy groups now play, researchers in this area still face a number of challenges. But, fortunately, increased collaboration between patient groups, industry leaders, and regulatory bodies can help overcome these hurdles. Barriers to orphan drug research include: Small population By definition,...
DURHAM, N.C., August 10, 2016 — Patient advocacy groups play an important and growing role in developing drugs to treat cancer and rare diseases, contributing to study design, recruiting patients, and even directly funding clinical trials. At a September webinar, Premier Research will team with rare disease advocacy organization Global Genes to explore this important...