Development of a stated-preference instrument to prioritize treatment goals in recent onset schizophrenia

Abstract

Objective: This study sought to evaluate a new stated-preference instrument to prioritize multiple treatment goals among people with recent onset schizophrenia.

Methods: A draft survey instrument was developed to assess preferences for 13 key treatment goals that were identified based on the literature. The survey incorporates best–worst scaling (BWS), which shows repeated subsets comprising 4 of the 13 goals, and respondents identify which is most important and which is least important to them. Pre-test interviews were conducted in the UK, Italy, and Germany among people aged 18 to 35 diagnosed with schizophrenia within the past 5 years. Specifically, participants completed the instrument online in their native language, followed by a telephone interview to provide feedback on the clarity, relevance, and comprehensiveness of the survey. The interview data were analyzed to assess interpretation and content validity of the survey.

Results: Fifteen participants (five per country) provided feedback (mean age = 31; 60% male). Feedback was comparable across countries and confirmed that the key treatment goals assessed were relevant and meaningful. Probing by interviewers ascertained that respondents interpreted the questions appropriately and identified the treatment goals that were most and least important to them. Based on the characterization of the goals, a conceptual model was developed illustrating hypothesized associations among them.

Conclusion: The results confirm that the BWS methodology and key treatment goals in this new instrument are appropriate for use in recent onset schizophrenia. These results will help guide measurement of patient-relevant endpoints in future studies.

Keywords:

• Schizophrenia
• treatment goals
• conceptual model

Transparency

Declaration of funding

This study was funded by Otsuka Pharmaceutical Europe Ltd. and H. Lundbeck A/S.

Author contributions: K.B. and K.A. were involved in study conception, design, analysis, interpretation, and drafting of this paper. E.C., A.J.L., J.Y.L., P.S., S.H., A.G.N., P.R. and J.F.P.B. were involved in study conception, design, interpretation of the data, and critical review of the draft paper. All authors approved the final version and agree to be accountable for all aspects of this work.

Declaration of financial/other relationships

K.B. and K.A. have disclosed that they provide consulting services to Otsuka Pharmaceutical Europe and Lundbeck. E.C., A.J.L., and J.Y.L. have disclosed that they are employees of Otsuka. P.S. and A.G.N. have disclosed that they are employees of Lundbeck. P.R. has disclosed that at the time of the study he was an employee of Otsuka. S.H. has disclosed that he has received speaker honoraria and/or participated in clinical trials supported by Janssen-Cilag, Eli Lilly, Sanofi-Aventis, Otsuka, Lundbeck, Johnson & Johnson, Pfizer, Bristol-Myers-Squibb, AstraZeneca, Novartis, Servier, Pierre Fabre, Organon, Roche and Merck, Teva, and Janssen. J.F.P.B. has disclosed that he advises ORS Health, and has received travel expenses from Lundbeck/Otsuka.

CMRO peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Acknowledgements

No assistance was used in the preparation of this article.

Previous presentation: The abstract of this paper was a poster presentation at the 19th Annual International Society for Pharmacoeconomics and Outcomes Research (ISPOR) European Congress, 2 November 2016, Vienna, Austria. The poster’s abstract was published in Value in Health 2016;19(7).