Final Research Report

View this project's final research report.

Journal Citations

Related Journal Citations

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. Those comments and responses included the following:

  • The reviewers asked for more justification for why it is necessary or helpful to include a large number of patients and caregivers in developing clinical practice guidelines. The reviewers asked if there is any previous research on how much patient input is needed. The researchers replied that they were not aware of literature that recommends input from a particular number of patients but said one or two patients are typically involved. The researchers added that patients with less education, income, mobility, and with cognitive impairments are typically not included. A larger, more diverse patient group can reflect a broader range of healthcare experiences, the researchers explained.
  • The reviewers suggested that key findings from the report be made more accessible to patients and patient advocates, for example, in a how-to manual or other report that would be easier to digest and use. The researchers responded that they had shared their results with the Duchenne muscular dystrophy community and planned to continue their efforts to disseminate their work to other rare disease communities. The researchers also addressed this in the future research section of the report’s discussion, stating that although their methods were meant to be agnostic to disease or condition, it would be helpful to test the methods in groups that differ by age, disease area, and other characteristics. They went on to recommend that the emphasis in these future opportunities should be in making sure the methodology is accessible to patients with different levels of education.
  • The reviewers noted that one consideration in online patient engagement is that it is difficult to confirm who might be responding to the request for input and therefore, how the respondents are viewing their own experiences or those of their loved ones. The researchers agreed this is a concern, which is why they relied in this study on the Duchenne Registry to recruit study participants. The researchers also created a recruitment survey to ensure that their participants had the necessary expertise and experience to respond. Furthermore, the researchers recommended in the report that groups using these methods should strive to recruit participants with diverse perspectives, and to confirm participants’ eligibility to participate.
  • The reviewers suggested consistently referring to patients and caregivers rather than patients and representatives. The researchers said they deliberately used the different terms, using patients and caregivers when discussing their own data collection. However, they chose to use patients and their representatives when referring to literature that uses those terms or discussing the implications of their work to the guideline development process. The researchers explained that patient representatives include family members, patient advocates, and others who are not necessarily caregivers.

Conflict of Interest Disclosures

Project Information

Dmitry Khodyakov, PhD, MA
RAND Corporation
Engaging Patients and Caregivers Managing Rare Diseases to Improve the Methods of Clinical Guideline Development

Key Dates

April 2016
May 2020

Study Registration Information


Has Results
Award Type
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
Intervention Strategy Intervention Strategies PCORI funds comparative clinical effectiveness research (CER) studies that compare two or more options or approaches to health care, or that compare different ways of delivering or receiving care. View Glossary
State State The state where the project originates, or where the primary institution or organization is located. View Glossary
Last updated: April 26, 2022