Get access

Are Adult Patients More Tolerant of Treatment Risks Than Parents of Juvenile Patients?

Authors

  • F. Reed Johnson,

    Corresponding author
      *Address correspondence to F. Reed Johnson, RTI Health Solutions, 200 Park Drive, PO Box 12194, Research Triangle Park, NC 27709-2194, USA; tel: (919) 541-5958; fax: (919) 541-7222; frjohnson@rti.org.
    Search for more papers by this author
    • 1

      Research Triangle Institute, Research Triangle Park, NC, USA.

  • Semra Özdemir,

    Search for more papers by this author
    • 2

      Department of Environmental Science and Engineering, School of Public Health, University of North Carolina at Chapel Hill, NC, USA. Work conducted while an employee at Research Triangle Institute, Research Triangle Park, NC, USA.

  • Carol Mansfield,

    Search for more papers by this author
    • 1

      Research Triangle Institute, Research Triangle Park, NC, USA.

  • Steven Hass,

    Search for more papers by this author
    • 3

      Elan Pharmaceuticals, San Francisco, CA, USA.

  • Corey A. Siegel,

    Search for more papers by this author
    • 4

      BD Center, Section of Gastroenterology and Hepatology, Dartmouth-Hitchcock Medical Center, Lebanon, NH, USA.

  • Bruce E. Sands

    Search for more papers by this author
    • 5

      MGH Crohn's and Colitis Center and Gastrointestinal Unit, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.


*Address correspondence to F. Reed Johnson, RTI Health Solutions, 200 Park Drive, PO Box 12194, Research Triangle Park, NC 27709-2194, USA; tel: (919) 541-5958; fax: (919) 541-7222; frjohnson@rti.org.

Abstract

Understanding patient-specific differences in risk tolerance for new treatments that offer improved efficacy can assist in making difficult regulatory and clinical decisions for new treatments that offer both the potential for greater effectiveness in relieving disease symptoms, but also risks of disabling or fatal side effects. The aim of this study is to elicit benefit-risk trade-off preferences for hypothetical treatments with varying efficacy and risk levels using a stated-choice (SC) survey. We derive estimates of “maximum acceptable risk” (MAR) that can help decisionmakers identify welfare-enhancing alternatives. In the case of children, parent caregivers are responsible for treatment decisions and their risk tolerance may be quite different than adult patients' own tolerance for treatment-related risks. We estimated and compared the willingness of Crohn's disease (CD) patients and parents of juvenile CD patients to accept serious adverse event (SAE) risks in exchange for symptom relief. The analyzed data were from 345 patients over the age of 18 and 150 parents of children under the age of 18. The estimation results provide strong evidence that adult patients and parents of juvenile patients are willing to accept tradeoffs between treatment efficacy and risks of SAEs. Parents of juvenile CD patients are about as risk tolerant for their children as adult CD patients are for themselves for improved treatment efficacy. SC surveys provide a systematic method for eliciting preferences for benefit-risk tradeoffs. Understanding patients' own risk perceptions and their willingness to accept risks in return for treatment benefits can help inform risk management decision making.

Get access to the full text of this article

Ancillary