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Patient Involvement in the Design of a Patient-Centered Clinical Trial to Promote Adherence to Supplemental Oxygen Therapy in COPD

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Abstract

Background

Patients are increasingly viewed as key stakeholders who can contribute in meaningful ways to clinical research and are emphasized in research funded by the Patient-Centered Outcomes Research Institute (PCORI). We are not aware of other peer-reviewed publications that report methods and outcomes of patient engagement to refine study design for a PCORI-sponsored clinical effectiveness trial.

Objective

The aim of this report was to describe the process and outcomes of involving patients in the design of a clinical trial to promote adherence to supplemental oxygen therapy among patients with chronic obstructive pulmonary disease.

Methods

In-person focus groups and individual discussions via telephone and email were used to elicit feedback to refine the intervention and clarify outcomes of highest importance to patients.

Results

A total of 25 patients and five caregivers provided feedback. Their feedback has informed decisions regarding the length of intervention sessions (20 min and in some cases longer was acceptable), the importance of including caregivers, and discussion topics (e.g., social discomfort about using oxygen in public, identifying personally relevant reasons to use oxygen, pulmonary rehabilitation). Multiple outcomes were rated as highly important to patients (physical function, fatigue, sleep, anxiety, depression, and ability to participate in social roles and activities), and the outcome that was ranked as most important varied by individual. Therefore, multiple patient-reported outcomes will be used as endpoints for the clinical trial.

Conclusions

Patient involvement led to refinements of the intervention and clinical trial endpoints to better address the expressed needs and concerns of patients and caregivers.

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Acknowledgments

The research team thanks the patients and caregivers who shared their insights. We also acknowledge the contributions by the PELICAN study staff, including Nina Bracken, Julie DeLisa, Lourdes Norwick, Vanessa Gonzalez, Binoy Joese, and Tori Morken.

Author contributions

Kristen Holm: involved with all stages of this project. Jerry Krishnan: principal investigator of the PELICAN project and involved with all stages of this project. Richard Casaburi, Scott Cerreta, Hélène Gussin, Julian Husbands, Janos Porszasz, Valentin Prieto-Centurion, Robert Sandhaus, Jamie Sullivan, Linda Walsh assisted in the development of the project and commented on drafts of the paper.

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Authors and Affiliations

  1. Department of Medicine, National Jewish Health, Denver, CO, USA

    Kristen E. Holm & Robert A. Sandhaus

  2. Department of Community and Behavioral Health, Colorado School of Public Health, Aurora, CO, USA

    Kristen E. Holm

  3. Rehabilitation Clinical Trials Center, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, USA

    Richard Casaburi & Janos Porszasz

  4. COPD Foundation, Washington, DC, USA

    Scott Cerreta, Jamie L. Sullivan & Linda J. Walsh

  5. Population Health Sciences Program, University of Illinois Hospital and Health Sciences System, Chicago, IL, USA

    Hélène A. Gussin & Jerry A. Krishnan

  6. Apria Healthcare, Lake Forest, CA, USA

    Julian Husbands

  7. Department of Medicine, University of Illinois at Chicago, Chicago, IL, USA

    Valentin Prieto-Centurion & Jerry A. Krishnan

  8. AlphaNet, Miami, FL, USA

    Robert A. Sandhaus

  9. Alpha-1 Foundation, Miami, FL, USA

    Robert A. Sandhaus

Authors
  1. Kristen E. Holm
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  2. Richard Casaburi
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  3. Scott Cerreta
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  4. Hélène A. Gussin
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  5. Julian Husbands
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  6. Janos Porszasz
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  7. Valentin Prieto-Centurion
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  8. Robert A. Sandhaus
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  9. Jamie L. Sullivan
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  10. Linda J. Walsh
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  11. Jerry A. Krishnan
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Corresponding author

Correspondence to Kristen E. Holm.

Ethics declarations

Ethical approval

Research was reviewed by the Institutional Review Board of the University of Illinois at Chicago and written informed consent was obtained from focus group participants (protocol #2013-0999).

Conflict of interest

KEH, RC, SC, HAG, JH, JP, VPC, RAS, JLS, LJW and JAK declare that they have no conflict of interest.

Funding

This study was funded by the Patient-Centered Outcomes Research Institute (PCORI; CE-1304-6490). All statements in this report, including its findings and conclusions, are solely those of the authors and do not necessarily represent the views of the Patient-Centered Outcomes Research Institute (PCORI), its Board of Governors or Methodology Committee.

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Holm, K.E., Casaburi, R., Cerreta, S. et al. Patient Involvement in the Design of a Patient-Centered Clinical Trial to Promote Adherence to Supplemental Oxygen Therapy in COPD. Patient 9, 271–279 (2016). https://doi.org/10.1007/s40271-015-0150-z

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  • DOI: https://doi.org/10.1007/s40271-015-0150-z

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