clinical trials for rare diseases Archives

Do Patients Care About What Your Drug Does?
Clinical trials

Do Patients Care About What Your Drug Does?

Do Patients Care About What Your Drug Does? By Sara Ray, MA Be sure the trial outcomes echo the patient voice, article says In most randomized pharmaceutical clinical trials, researchers are looking for a statistically significant performance difference between the two courses of therapy. But which is more important to the patient: Statistically significant improvement of a variable, or clinically significant improvement? “The operation was a success, but the patient died” is a succinct example of a misplaced sense of priorities. The patient was probably hoping for a better result than academic satisfaction. Here’s another example. [...]

Clinical Trials and PROs: What Patients with Rare Diseases Say

Clinical Trials and PROs: What Patients with Rare Diseases Say By Kathleen Hoffman, PhD MSPH Challenges to developing clinical trials for rare diseases begin with their rarity – each condition affects fewer than 200,000 Americans. Approximately seventy-five percent of those affected by rare diseases are children. It is estimated that 80 percent of rare diseases are caused by genetic changes. Many rare diseases are progressive and debilitating. One third of the children affected by these conditions do not reach 5 years of age.1 Getting to a rare disease diagnosis can take years. In a way, it [...]

Virtual Patient Engagement: Inspire and the NIH Clinical Center

Virtual Patient Engagement: Inspire and the NIH Clinical Center Engagement with patients and caregivers is at the heart of Inspire’s community of cancer, chronic, and rare disease support groups. While there are many ways Inspire can help the industry engage with a targeted patient population, virtual patient engagement and authoritative expert content are central to that engagement. For example, we recently conducted five webinars and started a new discussion post series called “Ask the Expert.” During these webinars and through the question-answer format of the “Ask the Expert” series, Inspire educates and engages members, [...]