Browsing: trials

Rare paediatric central nervous system (CNS) tumours comprise a biologically-heterogeneous group of low-incidence diseases that present significant challenges to both therapeutic development and clinical research, limiting the availability of high-quality evidence to guide clinical care. The UK3CR Children’s Cancer Research Group (CRG) CNS Tumours Subgroup convened a multidisciplinary workshop to identify research priorities and establish…

Women remain underrepresented in authorship and leadership roles of clinical trials of structural heart disease (SHD), and targeted efforts and policies to diversify trial leadership may help address the downstream inequities in the generation of cardiovascular evidence, according to a systematic review and meta-analysis published Aug. 12 in JACC: Advances

Earlier this year, the U.S. Department of Health and Human Services (HHS) stopped recommending that all children get an annual influenza vaccine. Instead, it made the shot a matter of “shared clinical decision-making”—something for parents and a doctor to weigh case by case—citing, among other reasons, a lack of randomized controlled trials proving the vaccine’s…

“Obtaining regulatory approval is one part of the challenge for rare disease therapies. Following approval, HTA agencies and payers evaluate the clinical, economic, and patient-centered value of a therapy to inform pricing, reimbursement, and patient access decisions. Historically, rare disease trials have often relied on small patient populations, single-arm or open-label designs, surrogate endpoints, and…