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Informing Drug Development and Clinical Practice Through Patient-Centered Outcomes Research

  • Cheryl D. Coon
    Affiliations
    Pharmacoeconomics, Outcomes, and Health Policy Section

    Associate Director, Endpoint Development & Outcomes Assessment Adelphi Values
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  • Author Footnotes
    ⁎ The content of this editorial does not necessarily represent the official views of the Centers for Disease Control and Prevention or the National Center for Health Statistics.
    Denys T. Lau
    Footnotes
    ⁎ The content of this editorial does not necessarily represent the official views of the Centers for Disease Control and Prevention or the National Center for Health Statistics.
    Affiliations
    Pharmacoeconomics, Outcomes, and Health Policy Section

    Deputy Division Director Centers for Disease Control and Prevention, National Center for Health Statistics, Hyattsville, Maryland Adjunct Associate Professor, University of Illinois at Chicago, College of Pharmacy, Chicago, Illinois
    Search for articles by this author
  • Author Footnotes
    ⁎ The content of this editorial does not necessarily represent the official views of the Centers for Disease Control and Prevention or the National Center for Health Statistics.
      In the last 2 decades, the health care field has made an effort to emphasize the patient experience and voice in the quality and value of health care in the United States. US Food and Drug Administration (FDA) guidance on patient-reported outcome (PRO) instruments in obtaining labeling claims, some may argue, is the endeavor that has helped push patient-centered outcomes to the forefront of health care research. Although PRO instruments have been used for years, especially in the field of psychology research, they have gained favor over time as a means for diagnosing medical conditions, assessing treatment efficacy, understanding the burden of illness, and gaining insight into the impact of disease directly from the patient’s own perspective. The concept of patient-centered outcomes allows for health care research to go beyond its traditional model, with a focus on the impact of disease and treatment on a patient’s quality of life from his or her own eyes.
      Although the patient’s voice has increasingly been incorporated into pharmaceutical clinical trials and clinical practice, researchers and clinicians are still striving to define and apply ways by which the patient can be the focal point of the health care industry. This special issue of Clinical Therapeutics compiles conceptual and empirical articles that explore and discuss scientific approaches to incorporating the patient’s voice into health care research. Our intention was to continue the dialogue on advancing patient-centered outcomes research to better inform drug development and clinical practice.
      The issue opens with insight from Hasnain-Wynia and Beal
      • Hasnain-Wynia R.
      • Beal A.C.
      The Patient-Centered Outcomes Research Institute’s (PCORI) role in addressing disparities and engaging patients in clinical research.
      as members of the Patient-Centered Outcomes Research Institute, whose remit is to fund research that can be used to inform health decisions from the perspective of patients and caregivers, in addition to the traditional stakeholders (eg, physicians, payers, policy makers). The institute is integrating patient engagement into health care research via a number of initiatives, including a key focus on reducing disparities in health care outcomes (eg, by promoting research and care in underserved populations). The authors note that the FDA is seeking greater patient engagement through public meetings and workshops on patient-focused drug development, which underscores the relevance of patient-centered outcomes research in the current health care landscape.
      Peay et al
      • Peay H.L.
      • Hollin I.
      • Fischer R.
      • Bridges J.F.
      A community-engaged approach to quantifying caregiver preferences for the benefits and risks of emerging therapies for Duchenne muscular dystrophy.
      then provide an example of research that specifically incorporates the caregiver’s perspective in a pediatric population (ie, the caregiver as a patient-proxy). Duchenne’s muscular dystrophy, a rare disease, was not included at first in the initiative by the FDA promoting patient-centered drug development; instead, an advocacy group supporting this patient population and their caregivers led their own research with the goal of informing the FDA of preferences for therapy benefits and risks. The authors argue that by quantifying patient or caregiver preferences, stakeholders have access to evidence that may be incorporated into regulatory decisions; access to therapy can then be based on the desires of the patients receiving the therapy or of the caregivers administering the therapy, rather than on a one-size-fits-all model based on efficacy and safety alone.
      Next, Clerson et al
      • Clerson P.
      • Graesslin O.
      • Gater A.
      • et al.
      EVAPIL-R scale: continuous development and validation of a tool to assess patient-reported tolerability of different contraceptive methods in longitudinal studies.
      describe qualitative steps taken to revise an existing PRO instrument to better represent the perspective of the full population of women using contraception. In addition to researching the published literature and consulting with prescribing gynecologists, the authors conducted open-ended interviews to elicit information about the symptoms and effects associated with their contraceptive methods and to ensure that the PRO instrument items are relevant and comprehensive to their experiences. By assessing patient-reported tolerability of treatment, the authors seek to inform health care decision making with the aim of facilitating treatment adherence and ultimately limiting negative patient outcomes.
      Also included in this issue are several methodologic articles on statistical approaches to incorporating the patient’s voice as the focal point of outcomes research. First, Cappelleri et al
      • Cappelleri J.C.
      • Lundy J.J.
      • Hays R.D.
      Overview of classical test theory and item response theory for quantitative assessment of items in developing patient-reported outcome measures.
      describe the application of different psychometric methods at an early stage of instrument development to support the construct validity of the PRO instrument, with the intention of ensuring that the resulting PRO instrument is a relevant and reliable measure of the patient experience. Second, in measuring the impact of irritable bowel syndrome on quality of life, Andrae et al
      • Andrae D.A.
      • Covington P.S.
      • Patrick D.L.
      Item-level assessment of the Irritable Bowel Syndrome Quality of Life Questionnaire (IBS-QOL) in patients with diarrheal irritable bowel syndrome.
      used item response theory to ensure that the PRO instrument’s scoring algorithm is most reflective of the patient perception of quality of life. Third, Edelen et al
      • Edelen M.O.
      • Stucky B.D.
      • Sherbourne C.
      • et al.
      Correspondence between the RAND Negative Impact of Asthma on Quality of Life Item Bank and the Marks Asthma Quality of Life Questionnaire.
      also applied item response theory to their research in asthma, with the goal of scoring separate PRO instruments on a common metric; in doing so, this method may aid researchers in comparing the results of separate studies to gain a broader picture of the patient experience and to compare the efficacy of different therapies that may not be assessed head-to-head. Finally, Hays et al
      • Hays R.D.
      • Berman L.J.
      • Kanter M.H.
      • et al.
      Evaluating the psychometric properties of the CAHPS patient-centered medical home survey.
      demonstrate a rigorous psychometric approach to evaluating and refining a survey to measure care experiences in patient-centered medical homes; in turn, the measure may be used to identify patients who are not getting the level of care that they need.
      This special issue puts a spotlight on the ongoing movement toward including the patient’s voice in health care research and practice. Stakeholders are already taking steps toward patient engagement through funding patient-centered research, holding public meetings on patient involvement, and seeking scientific evidence that incorporates the patient viewpoint. Our goal for this issue was to continue this dialogue as “patient-centered research” continues to be embraced by the health care field and matures into being a staple in health care research.

      References

        • Hasnain-Wynia R.
        • Beal A.C.
        The Patient-Centered Outcomes Research Institute’s (PCORI) role in addressing disparities and engaging patients in clinical research.
        Clin Ther. 2014; 36: 618-622
        • Peay H.L.
        • Hollin I.
        • Fischer R.
        • Bridges J.F.
        A community-engaged approach to quantifying caregiver preferences for the benefits and risks of emerging therapies for Duchenne muscular dystrophy.
        Clin Ther. 2014; 36: 624-637
        • Clerson P.
        • Graesslin O.
        • Gater A.
        • et al.
        EVAPIL-R scale: continuous development and validation of a tool to assess patient-reported tolerability of different contraceptive methods in longitudinal studies.
        Clin Ther. 2014; 36: 638-647
        • Cappelleri J.C.
        • Lundy J.J.
        • Hays R.D.
        Overview of classical test theory and item response theory for quantitative assessment of items in developing patient-reported outcome measures.
        Clin Ther. 2014; 36: 648-662
        • Andrae D.A.
        • Covington P.S.
        • Patrick D.L.
        Item-level assessment of the Irritable Bowel Syndrome Quality of Life Questionnaire (IBS-QOL) in patients with diarrheal irritable bowel syndrome.
        Clin Ther. 2014; 36: 663-679
        • Edelen M.O.
        • Stucky B.D.
        • Sherbourne C.
        • et al.
        Correspondence between the RAND Negative Impact of Asthma on Quality of Life Item Bank and the Marks Asthma Quality of Life Questionnaire.
        Clin Ther. 2014; 36: 680-688
        • Hays R.D.
        • Berman L.J.
        • Kanter M.H.
        • et al.
        Evaluating the psychometric properties of the CAHPS patient-centered medical home survey.
        Clin Ther. 2014; 36: 689-696