From researchers to sponsors to patients to advocacy groups to clinicians, rare disease research is full of passionate, driven, and determined people. As the greatest asset to the field, all people involved must be utilized to their greatest potential for a study to be successful. That’s why significant attention must be paid to the operational challenges within...
As market demand for AVR (transcatheter aortic valve replacement) and TMVR (transcatheter mitral valve replacement) has exploded, companies face increasing pressure to rapidly deliver actionable clinical trial data. The key to success lies in the team. And the foundation of an effective team begins by understanding each role—and, where applicable, how to best select each...
Rare disease and orphan drug research has a long history of serious challenges, thanks to both intrinsic factors—fewer patients means less available data; less available data means a less complete picture—and market forces—it’s more difficult to find investors when a smaller pool of patients may mean a more uncertain potential return on investment. Luckily, recent changes...
Big changes are planned in 2018 and beyond for how new medical devices are reviewed and approved by the U.S. Food and Drug Administration (FDA) if a proposal put forth moves forward. In a blogpost this week, FDA Commissioner Scott Gottlieb, M.D. announced the FDA’s Center for Devices and Radiological Health (CDRH) will publish a series...
Want to know how to build strong strategic sponsor-CRO partnerships? Commit to the three Ds: Start with a dialogue that establishes the project’s direction, ultimately making a difference in the relationship. In these relationships, a CRO must bring to the table the supplemental expertise a sponsor doesn’t know they don’t have, but needs for success. 1. Foster a Dialogue Bidirectional communication is probably the...
Most trials for potential Alzheimer disease (AD) treatments fail—and the reason may be poor protocol design. After all, typical AD studies are double-blind placebo-controlled parallel group clinical trials with a dual outcome, including a cognitive measure and a global impression of aptitude for the activities of daily living—a trial design originally developed to study cholinesterase...
This is the fifteenth and final installment of our look at the increasingly high placebo response that is plaguing clinical trials in analgesia and psychiatry. Read the rest of the posts in the series here. As our Placebo Problem series draws to a close, we conclude by taking a brief look at three ethical issues...
Phase I and II trials may have different overall goals (i.e., demonstrating safety vs. efficacy), but the two both struggle with a major challenge in oncology study design: finding the right dose. Luckily, decades of data and innovations have given researchers the tools necessary to plan a successful dose-finding trial. Read on for a look...
This is the fourteenth installment of our look at the increasingly high placebo response that is plaguing clinical trials in analgesia and psychiatry. Read the rest of the posts in the series here. The term “placebo” first took hold in an unlikely place: funerals. Placebo, Latin for “I shall please” first came into use in...
Roughly 47 million people around the world are living with Alzheimer’s disease (AD) — a number that is expected to increase to 75 million by 2030 and 150 million by 2050. Researchers are pursuing a range of treatments: disease modifying, symptomatic treatment and therapy for behavioral issues. Yet no new therapy has been approved since...