Final Research Report

View this project's final research report.

Peer-Review Summary

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. The comments and responses included the following:

  • Reviewers expressed a concern about some of the methodological assumptions related to the statistical models developed in this study. The researchers disagreed that the assumptions could not be met, and therefore, there were major limitations on the study. However, they did acknowledge in the report that since these assumptions could impose restrictions on the data, they should be subjected to goodness-of-fit tests to make sure that the proposed approach is suitable.
  • Noting that the report lists advisory board members and gives selected examples of how they engaged in the study, reviewers suggested the authors also provide examples of  how each stakeholder group  engaged. The researchers indicated that the advisory board engaged on an ad hoc basis.  They reported successful and unsuccessful engagement in the report. For instance, the researchers worked with industry stakeholders on the advisory board to identify datasets that could be used in this study but ultimately used datasets provided through nonadvisory board industry connections.
  • Given that the authors received very little feedback about their new software, reviewers asked how the researchers planned to obtain feedback. The reviewers also asked the researchers to provide more support for their statements that the lack of uptake was due to a lack of incentives and a lack of understanding of new methods. The researchers explained that they were not planning on seeking further feedback at this time. They explained that their speculation about the reasons for lack of uptake came from a senior FDA official who indicated that staff did not have the time and resources to train to use the new software tool. The researchers also compared this lack of uptake to other recommended best practices in analyzing and reporting study results which have not been widely adopted, saying that these best practices would need to be requirements in order to improve the uptake.

Conflict of Interest Disclosures

Project Information

Daniel O. Scharfstein, ScD
Johns Hopkins University
$553,858
10.25302/11.2019.ME.13036016
Sensitivity Analysis Tools for Clinical Trials with Missing Data

Key Dates

September 2013
February 2019
2013
2018

Study Registration Information

Tags

Has Results
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Health Conditions Health Conditions These are the broad terms we use to categorize our funded research studies; specific diseases or conditions are included within the appropriate larger category. Note: not all of our funded projects focus on a single disease or condition; some touch on multiple diseases or conditions, research methods, or broader health system interventions. Such projects won’t be listed by a primary disease/condition and so won’t appear if you use this filter tool to find them. View Glossary
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Last updated: March 4, 2022