TY - JOUR

T1 - Patient-focused drug development methods for benefit-risk assessments

T2 - A case study using discrete choice experiment for antiepileptic drugs

AU - Holmes, Emily A.F.

AU - Plumpton, Catrin

AU - Baker, Gus A.

AU - Jacoby, Ann

AU - Ring, Adele

AU - Williamson, Paula

AU - Marson, Anthony

AU - Hughes, Dyfrig A.

N1 - Funded by the National Institute for Health Research (NIHR), under its Research for Patient Benefit (RfPB) Programme (Grant Reference Number PB-PG-0909-20161). PW, AM, DAH are also supported by the Medical Research Council North West Hub for Trials Methodology Research (NWHTMR Reference number MR/K025635/1). AM is part-funded by NIHR Collaboration for Leadership in Applied Health Research and Care North West Coast (NIHR CLAHRC NWC); and DAH is a Health and Care Research Wales Senior Research Leader (SRL/15/029). © 2018 The Authors Clinical Pharmacology & Therapeutics published by Wiley Periodicals, Inc. on behalf of American Society for Clinical Pharmacology and Therapeutics.

PY - 2019/3

Y1 - 2019/3

N2 - Regulatory decisions may be enhanced by incorporating patient preferences for drug benefit and harms. This study demonstrates a method of weighting clinical evidence by patients' benefit-risk preferences. Preference weights, derived from discrete choice experiments, were applied to clinical trial data to estimate the expected utility of alternative drugs. In a case study, the rank ordering of antiepileptic drugs (AEDs), as indicated from clinical studies, was compared with ordering based on weighting clinical evidence by patients' preferences. A statistically significant change in rank ordering of AEDs was observed for women of childbearing potential who were prescribed monotherapy for generalized or unclassified epilepsy. Rank ordering inferred from trial data, valproate > topiramate > lamotrigine, was reversed. Modeling the expected utility of drugs might address the need to use more systematic, methodologically sound approaches to collect patient input that can further inform regulatory decision making.

AB - Regulatory decisions may be enhanced by incorporating patient preferences for drug benefit and harms. This study demonstrates a method of weighting clinical evidence by patients' benefit-risk preferences. Preference weights, derived from discrete choice experiments, were applied to clinical trial data to estimate the expected utility of alternative drugs. In a case study, the rank ordering of antiepileptic drugs (AEDs), as indicated from clinical studies, was compared with ordering based on weighting clinical evidence by patients' preferences. A statistically significant change in rank ordering of AEDs was observed for women of childbearing potential who were prescribed monotherapy for generalized or unclassified epilepsy. Rank ordering inferred from trial data, valproate > topiramate > lamotrigine, was reversed. Modeling the expected utility of drugs might address the need to use more systematic, methodologically sound approaches to collect patient input that can further inform regulatory decision making.

KW - Adolescent

KW - Adult

KW - Aged

KW - Anticonvulsants/administration & dosage

KW - Drug Development/methods

KW - Female

KW - Focus Groups/methods

KW - Humans

KW - Male

KW - Middle Aged

KW - Patient-Centered Care/methods

KW - Qualitative Research

KW - Risk Assessment/methods

KW - Seizures/drug therapy

KW - Young Adult

U2 - 10.1002/cpt.1231

DO - 10.1002/cpt.1231

M3 - Article

C2 - 30204252

VL - 105

SP - 672

EP - 683

JO - Clinical Pharmacology and Therapeutics

JF - Clinical Pharmacology and Therapeutics

SN - 0009-9236

IS - 3

ER -