Results Summary and Professional Abstract
|This project's final research report is expected to be available by October 2020.|
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 wondered why the researchers revised the propensity scores (PS), calculated to account for differences in patient characteristics between intervention groups, to balance the treatment groups at 180 days after the start of treatment, rather than use the original PS at treatment start. The researchers explained that nontuberculous mycobacterium (NTM) infection can take a considerable amount of time to diagnose, so they felt that any incidences of NTM infection in the first six months were likely to be existing rather than new cases of disease. The researchers went on to explain that they recalculated PS because many things may have changed during those first six months that would affect the group balance established by the first PS calculations.
- The reviewers suggested that the report needed a conclusion section that more clearly interpreted options for patients and physicians, rather than provided a summary of findings. The researchers updated their conclusion section to characterize the strength of evidence from the study as well as the overall results. They stood by the conclusion that macrolide therapy was likely to be a better choice than chronic inhaled corticosteroids (ICS) for older bronchiectasis patients to avoid hospitalization for respiratory infections. However, they also acknowledged that the study results could not determine whether this was due to a benefit of macrolides or a risk of chronic ICS because they could not compare risk related to either of these drug classes to the risk related to not starting any new drug class.
- The reviewers commented that the report touts the involvement of patient advocacy organizations in the project but does not discuss the potential conflict of interest of an NTM advocacy organization receiving funding from pharmaceutical companies that may benefit from diagnosis and treatment of NTM. The researchers said they are very conscious of conflicts of interest and the need for transparency in research and noted that their project focused on two generic and widely available treatments. The researchers acknowledged that a partnership has recently been developed between drug developers and patient advocates for NTM disease, but said they saw this as a positive development, given lack of interest from federal funders or industry in the past.
Conflict of Interest Disclosures
The Conflict of Interest Disclosures for this project will be posted here soon.