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Engaging patients and caregivers managing rare diseases to improve the methods of clinical guideline development
Investigator (PI): Khodyakov, Dmitry
Performing Organization (PO): (Current): RAND Corporation / (310) 393-0411
Supporting Agency (SA): Patient-Centered Outcomes Research Institute (PCORI)
Initial Year: 2016
Final Year: 2020
Record Source/Award ID: PCORI/ME-1507-31052
Funding: Total Award Amount: $1,015,936
Award Type: Contract
Award Information: PCORI: More information and project results (when completed)
Abstract: Clinical guidelines provide recommendations for necessary and appropriate health care options for specific conditions and clinical circumstances. Unfortunately, guideline development groups have typically not directly involved patients. We address this critical gap by creating a new step in guideline development--an assessment of patient-centeredness (i.e., how well the proposed approach to care addresses the most common needs, concerns, values, or preferences of an average patient with a given condition of already developed care guidelines). We will do so by pursuing three specific aims: (aim 1) develop an approach to systematically engage patients with rare diseases (here, those with Duchenne muscular dystrophy [DMD]) and their caregivers in clinical guideline development that mirrors and complements the RAND/UCLA Appropriateness Method--the gold standard approach for conducting clinical expert panels that uses a modified-Delphi format; (aim 2) test the modified-Delphi approach for systematic engagement of patients with rare diseases and their caregivers in the process of developing clinical guidelines; and (aim 3) develop best practices for engaging patients and caregivers in the process of care guideline development. Specifically, we will adapt ExpertLens (EL)--a previously evaluated online modified-Delphi system that combines two rounds of rating with a round of feedback and moderated online discussions--for use by DMD patients and their caregivers. We will also develop an approach to rating patient-centeredness--a new step in developing care guidelines. The primary project outcome is a new method and an online system for patient and caregiver engagement in guideline development modeled after a gold standard approach to soliciting clinicians' input in this process. The secondary outcome is a list of best practices for enhancing the level of patient and caregiver involvement in the guideline development process. This effort is important to patients because it will augment the typical approach to guideline development by introducing a new step of systematically engaging patients in a culturally appropriate yet scientifically rigorous manner consistent with how clinical experts are engaged. Doing so will improve how patients and caregivers make decisions and will inform clinicians about patient viewpoints that could increase adherence to guidelines. Also, the online nature of EL will benefit patients with conditions like DMD, who are physically limited in their mobility. This project is an equal partnership between academic and community investigators. It is truly patient-centered because the idea for it was developed in close collaboration with our community partner, Parent Project Muscular Dystrophy, and engages DMD patients from DuchenneConnect, the largest U.S. patient/caregiver self-report registry for individuals with DMD. It will yield results likely to improve care outcomes, such as quality of life, that DMD patients are particularly concerned about.
MeSH Terms:
  • California
  • * Caregivers
  • Cultural Characteristics
  • Delivery of Health Care /*standards
  • Guideline Adherence
  • Guidelines as Topic
  • Humans
  • Internet
  • Muscular Dystrophy, Duchenne /*therapy
  • Outcome Assessment (Health Care)
  • * Patient Participation
  • Patient Preference
  • Patient-Centered Care
  • Program Development
  • Rare Diseases /*therapy
  • United States
  • Universities
Country: United States
State: California
Zip Code: 90401
UI: 20163126
Project Status: Ongoing
Record History: ('2017: Project extended to 2020.',)