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Patient Preferences in Regulatory Benefit-Risk Assessments: A US Perspective.

Publication ,  Journal Article
Johnson, FR; Zhou, M
Published in: Value Health
2016

Demands for greater transparency in US regulatory assessments of benefits and risks, together with growing interest in engaging patients in Food and Drug Administration regulatory decision making, have resulted in several recent regulatory developments. Although Food and Drug Administration's Center for Drug Evaluation and Research (CDER) and Center for Devices and Radiological Health (CDRH) have established patient-engagement initiatives, CDRH has proposed guidelines for considering quantitative data on patients' benefit-risk perspectives, while CDER has focused on a more qualitative approach. We summarize two significant studies that were developed in collaboration and consultation with CDER and CDRH. CDER encouraged a patient advocacy group to propose draft guidance on engaging patient and caregiver stakeholders in regulatory decision making for Duchenne muscular dystrophy. CDRH sponsored a discrete-choice experiment case study to quantify obese respondents' perspectives on "meaningful benefits." CDRH and CDER issued draft guidance in May and June 2015, respectively, on including patient-preference data in regulatory submissions. Both organizations face challenges. CDER is working on integrating qualitative data into existing evidence-based review processes and is exploring options for therapeutic areas not included on a priority list. CDRH has adopted an approach that requires patient-preference data to satisfy standards of valid scientific evidence. Although that strategy could facilitate integrating patient perspectives directly with clinical data on benefits and harms, generating such data requires building capacity.

Duke Scholars

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Published In

Value Health

DOI

EISSN

1524-4733

Publication Date

2016

Volume

19

Issue

6

Start / End Page

741 / 745

Location

United States

Related Subject Headings

  • United States
  • Risk Assessment
  • Prescription Drugs
  • Patient Preference
  • Health Policy & Services
  • Government Regulation
  • Equipment and Supplies
  • 4407 Policy and administration
  • 4203 Health services and systems
  • 3801 Applied economics
 

Citation

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Johnson, F. R., & Zhou, M. (2016). Patient Preferences in Regulatory Benefit-Risk Assessments: A US Perspective. Value Health, 19(6), 741–745. https://doi.org/10.1016/j.jval.2016.04.008
Johnson, F Reed, and Mo Zhou. “Patient Preferences in Regulatory Benefit-Risk Assessments: A US Perspective.Value Health 19, no. 6 (2016): 741–45. https://doi.org/10.1016/j.jval.2016.04.008.
Johnson FR, Zhou M. Patient Preferences in Regulatory Benefit-Risk Assessments: A US Perspective. Value Health. 2016;19(6):741–5.
Johnson, F. Reed, and Mo Zhou. “Patient Preferences in Regulatory Benefit-Risk Assessments: A US Perspective.Value Health, vol. 19, no. 6, 2016, pp. 741–45. Pubmed, doi:10.1016/j.jval.2016.04.008.
Johnson FR, Zhou M. Patient Preferences in Regulatory Benefit-Risk Assessments: A US Perspective. Value Health. 2016;19(6):741–745.
Journal cover image

Published In

Value Health

DOI

EISSN

1524-4733

Publication Date

2016

Volume

19

Issue

6

Start / End Page

741 / 745

Location

United States

Related Subject Headings

  • United States
  • Risk Assessment
  • Prescription Drugs
  • Patient Preference
  • Health Policy & Services
  • Government Regulation
  • Equipment and Supplies
  • 4407 Policy and administration
  • 4203 Health services and systems
  • 3801 Applied economics