Final Research Report
View this project's final research report.
Related Journal Citations
Peer review of PCORI-funded research helps make sure the report presents complete, balanced, and useful information about the research. It also assesses how the project addressed PCORI’s Methodology Standards. During peer review, experts read a draft report of the research and provide comments about the report. These experts may include a scientist focused on the research topic, a specialist in research methods, a patient or caregiver, and a healthcare professional. These reviewers cannot have conflicts of interest with the study.
The peer reviewers point out where the draft report may need revision. For example, they may suggest ways to improve descriptions of the conduct of the study or to clarify the connection between results and conclusions. Sometimes, awardees revise their draft reports twice or more to address all of the reviewers’ comments.
Peer reviewers commented and the researchers made changes or provided responses. Those comments and responses included the following:
- The reviewers asked for additional details and expanded discussion on various topics, including on the difference between sequential, multiple assignment, randomized trials (SMARTs) versus small n SMARTs (snSMARTs). The researchers added details to various sections of their report and explained that their snSMART methods were specifically designed for studies involving small numbers of samples or rare diseases.
- The reviewers asked whether the estimators calculated in this study could be applied to other settings or used with pooled data, even with larger sample sizes. The researchers noted that the utility of their design and methods diminish as sample size increases but can still be used. They said the greatest benefit of their snSMART design and methods are for studies involving fewer than 100 cases.
- The reviewers asked whether the researchers planned to write nontechnical manuscripts to disseminate their findings to a broader audience. The researchers said they have made efforts to disseminate their findings to broader audiences and regulatory communities. However, they believed the study would be most useful for statisticians and rare disease researchers.
Conflict of Interest Disclosures
Patient / Caregiver Partners
- Ida Hakkarinen
- Kori Jones
Other Stakeholder Partners
- Peter Merkel
- Jennifer Miller
- Rajen Mody
- Susan Murphy
- Christian Pagnoux
Study Registration Information
- Has Results