title: Integration of patient-reported outcomes in multiregional confirmatory clinical trials.
abstract: BACKGROUND
  The increasing complexities of conducting multiregional trials and an evolving regulatory environment contribute to unprecedented new challenges for use of patient-reported outcome measures (PROMs)(1) within clinical trials. This paper presents these challenges and potential solutions.
  
  
  METHODS
  Real-world examples and situations are reviewed from an industry and patient-reported outcome (PRO)(2) expert position.
  
  
  CONCLUSIONS
  An increase in the pursuit of new therapeutic targets, changes to the regulatory environment, and business pressures to expand clinical trials to more countries have significantly increased the complexity of confirmatory clinical studies that incorporate PROMs. Decisions to participate in collaborative efforts for endpoint development or proceed independently are made in the context of competing priorities of drug development timelines, drug differentiation strategies, the need for patient-related value messages, and the depth of a sponsor pipeline within specific disease areas. Study logistics are critically important; factors such as concept cultural relevancy, respondent literacy level, and quality of cross-cultural adaptation of PROMs must be evaluated when integrating into confirmatory clinical trials. Awareness of the issues relating to PROs in multiregional studies will enable companies to better plan studies and interpret results.
authors: Novartis Pharmaceuticals Corporation, 1 Health Plaza East, Hanover, NJ, USA. ari.gnanasakthy@novartis.com
authors: Ari
authors: A
authors: Gnanasakthy
authors: Carla
authors: C
authors: DeMuro
authors: Caroline
authors: C
authors: Boulton
mesh_terms: D015982:Bias; D004345:Drug Industry; D004335:Drug and Narcotic Control; D006801:Humans; D015337:Multicenter Studies as Topic; D010361:Patients; D016896:Treatment Outcome