Blog

Rare disease clinical trials face significant challenges, and with small patient populations, leveraging innovative statistical methodologies that can enhance statistical power is crucial. The Net Treatment Benefit, estimated from the Generalized Pairwise Comparisons methodology, provides a promising solution by integrating multiple clinical outcomes into a single assessment, extracting more information from the data.

Randomized controlled clinical trials are considered the gold standard for understanding treatment safety and efficacy. Rare disease trials struggle to recruit enough participants to demonstrate efficacy, creating a barrier to research and approval. However, Bayesian statistics utilize existing evidence and expert opinion to quantify uncertainty and inform trial design, reducing necessary sample size. 

A healthy relationship between sites and sponsors is critical to the success of clinical trials, including efficient and effective management, adaptability, innovation, problem-solving, and feedback mechanisms on the parts of those involved on both sides. Clinical research associate (CRA) competencies are key to creating and nurturing this relationship under all circumstances.

Although relatively rare, clinical investigator inspections by the U.S. Food and Drug Administration (FDA) can have a significant business impact. Audits are conducted more frequently than regulatory inspections, which are […]

Many times, individuals who have triumphed over frightening, or even near-death, situations will say that the experience has given them a new and more positive outlook on life. Allison Massari, a two-time survivor of such scenarios, has an additional and unique insight—and it’s on the patient experience. She celebrates how healthcare professionals have an “exceptional power to transform lives,” and will share her perspective on how compassionate care can be incorporated into clinical research in a Signature Series keynote session tailored for attendees of ACRP 2025 in New Orleans, La., in April.