Danielle Whicher

TL;DR: This article addresses several fundamental limitations of traditional RCTs for meeting CER objectives and offers 3 potentially transformational approaches to enhance their operational efficiency, analytical efficiency, and generalizability for CER.

TL;DR: While the level of interest and activity in conducting pRCTs is increasing, many challenges remain for their routine use and some potential ways forward are offered.

TL;DR: A CER-ECD observational study that imposes no or minimal additional risk to or burden on patients may proceed ethically without express informed consent from participants in settings where: patients are regularly informed of the health care institution’s commitment to learning through the integration of research and practice and there are appropriate protections for patients’ rights and interests.

TL;DR: A landscape review of available methodological and analytic approaches to address the challenges of rare disease research suggests that use of a broader array of methodological approaches to RCTs --such as adaptive trials, cross-over trials, and early escape designs can improve the productivity of robust research in rare diseases.

TL;DR: This study describes an Agency for Healthcare Research and Quality-funded pilot project to translate one current comparative effectiveness review into a prioritized list of evidence gaps and research questions reflecting the views of the healthcare decision makers involved in the pilot.