title: Incorporating patient preferences into drug development and regulatory decision making: Results from a quantitative pilot study with cancer patients, carers, and regulators.
abstract: Currently, patient preference studies are not required to be included in marketing authorization applications to regulatory authorities, and the role and methodology for such studies have not been agreed upon. The European Medicines Agency (EMA) conducted a pilot study to gain experience on how the collection of individual preferences can inform the regulatory review. Using a short online questionnaire, ordinal statements regarding the desirability of different outcomes in the treatment of advanced cancer were elicited from 139 participants (98 regulators, 29 patient or carers, and 12 healthcare professionals). This was followed by face-to-face meetings to gather feedback and validate the individual responses. In this article we summarize the EMA pilot study and discuss the role of patient preference studies within the regulatory review. Based on the results, we conclude that our preference elicitation instrument was easy to implement and sufficiently precise to learn about the distribution of the participants' individual preferences.
authors.affiliation: European Medicines Agency, London, UK.|University of Groningen, University Medical Center Groningen, The Netherlands.
authors.forename: D
authors.initials: D
authors.lastname: Postmus
authors.affiliation: European Medicines Agency, London, UK.
authors.forename: M
authors.initials: M
authors.lastname: Mavris
authors.affiliation: University of Groningen, University Medical Center Groningen, The Netherlands.
authors.forename: H L
authors.initials: HL
authors.lastname: Hillege
authors.affiliation: European Medicines Agency, London, UK.|Läkemedelsverket Medical Products Agency, Uppsala, Sweden.
authors.forename: T
authors.initials: T
authors.lastname: Salmonson
authors.affiliation: Melanoma Patient Network Europe.
authors.forename: B
authors.initials: B
authors.lastname: Ryll
authors.affiliation: Myeloma Patients Europe.
authors.forename: A
authors.initials: A
authors.lastname: Plate
authors.affiliation: European Medicines Agency, London, UK.
authors.forename: I
authors.initials: I
authors.lastname: Moulon
authors.affiliation: European Medicines Agency, London, UK.
authors.forename: H-G
authors.initials: HG
authors.lastname: Eichler
authors.affiliation: European Medicines Agency, London, UK.
authors.forename: N
authors.initials: N
authors.lastname: Bere
authors.affiliation: European Medicines Agency, London, UK.
authors.forename: F
authors.initials: F
authors.lastname: Pignatti
mesh_terms: D017028:Caregivers / Q000523:psychology; D003657:Decision Making; D015195:Drug Design; D004335:Drug and Narcotic Control / Q000379:methods*; D005062:European Union; D006801:Humans; D009369:Neoplasms / Q000188:drug therapy* / Q000523:psychology; D057240:Patient Preference; D010865:Pilot Projects; D011795:Surveys and Questionnaires