Recommendations for defining treatment outcomes in major psychiatric disorders using real-world data

Elise Koch¹, Sophie Smart², Guðmundur Einarsson³, Anders Kämpe⁴, Lina Jonsson⁵, Maris Alver⁶, Matthew Iveson⁷, Andreas Göteson⁵, Antonio F Pardiñas², Ida E Sønderby⁸, Kevin S O'Connell¹, Qingqin Li⁹, Yi Lu¹⁰, Hreinn Stefánsson³, Kári Stefánsson¹¹, Heather Whalley¹², Mikael Landén¹³, Michael C O'Donovan², Knut Smerud¹⁴, Gerard R Dawson¹⁵, Thomas Werge¹⁶, Alfonso Buil¹⁷, Andreas Reif¹⁸, Lili Milani⁶, Espen Molden¹⁹, Chiara Fabbri²⁰, Alessandro Serretti²¹, James Walters², Cathryn M Lewis²², Ole A Andreassen²³

Affiliations

¹ Center for Precision Psychiatry, Division of Mental Health and Addiction, Oslo University Hospital, Oslo, Norway; Institute of Clinical Medicine, University of Oslo, Oslo, Norway.
² Centre for Neuropsychiatric Genetics and Genomics, Division of Psychological Medicine and Clinical Neurosciences, Cardiff University, Cardiff, UK.
³ deCODE Genetics, Reykjavik, Iceland.
⁴ Institute for Molecular Medicine Finland (FIMM), Helsinki, Finland; Department of Molecular Medicine and Surgery (MMK), Karolinska Institutet, Stockholm, Sweden.
⁵ Department of Psychiatry and Neurochemistry, Institute of Neuroscience and Physiology at the Sahlgrenska Academy, University of Gothenburg, Gothenburg, Sweden.
⁶ Estonian Genome Centre, Institute of Genomics, University of Tartu, Tartu, Estonia.
⁷ Centre for Clinical Brain Sciences, University of Edinburgh, Edinburgh, UK.
⁸ Center for Precision Psychiatry, Division of Mental Health and Addiction, Oslo University Hospital, Oslo, Norway; Institute of Clinical Medicine, University of Oslo, Oslo, Norway; Department of Medical Genetics, Oslo University Hospital, Oslo, Norway; KG Jebsen Centre for Neurodevelopmental Disorders, University of Oslo, Oslo, Norway; Oslo University Hospital, Oslo, Norway.
⁹ Janssen Research and Development, Neuroscience, Titusville, NJ, USA.
¹⁰ Department of Medical Epidemiology and Biostatistics, Karolinska Institutet, Stockholm, Sweden.
¹¹ deCODE Genetics, Reykjavik, Iceland; Faculty of Medicine, University of Iceland, Reykjavik, Iceland.
¹² Centre for Clinical Brain Sciences, University of Edinburgh, Edinburgh, UK; Generation Scotland, Centre for Medical Informatics, University of Edinburgh, Edinburgh, UK.
¹³ Department of Psychiatry and Neurochemistry, Institute of Neuroscience and Physiology at the Sahlgrenska Academy, University of Gothenburg, Gothenburg, Sweden; Department of Medical Epidemiology and Biostatistics, Karolinska Institutet, Stockholm, Sweden.
¹⁴ Smerud Medical Research International AS, Oslo, Norway.
¹⁵ P1vital, Wallingford, UK.
¹⁶ Institute of Biological Psychiatry, Mental Health Services, Copenhagen University Hospital, Copenhagen, Denmark; Department of Clinical Medicine, University of Copenhagen, Denmark.
¹⁷ Institute of Biological Psychiatry, Mental Health Services, Copenhagen University Hospital, Copenhagen, Denmark.
¹⁸ Department of Psychiatry, Psychosomatic Medicine and Psychotherapy, University Medical Centre Frankfurt, Frankfurt, Germany; Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Frankfurt, Germany.
¹⁹ Center for Psychopharmacology, Diakonhjemmet Hospital, Oslo, Norway; Department of Pharmacy, University of Oslo, Oslo, Norway.
²⁰ Department of Biomedical and NeuroMotor Sciences, University of Bologna, Bologna, Italy.
²¹ Department of Medicine and Surgery, Kore University of Enna, Enna, Italy; Oasi Research Institute-IRCCS, Troina, Troina, Italy.
²² Social, Genetic and Developmental Psychiatry Centre, King's College London, London, UK; NIHR Maudsley Biomedical Research Centre, South London and Maudsley NHS Trust, London, UK.
²³ Center for Precision Psychiatry, Division of Mental Health and Addiction, Oslo University Hospital, Oslo, Norway; Institute of Clinical Medicine, University of Oslo, Oslo, Norway; KG Jebsen Centre for Neurodevelopmental Disorders, University of Oslo, Oslo, Norway; Oslo University Hospital, Oslo, Norway. Electronic address: ole.andreassen@medisin.uio.no.

PMID: 40222385
DOI: 10.1016/S2215-0366(25)00061-6

Review

Recommendations for defining treatment outcomes in major psychiatric disorders using real-world data

Elise Koch et al. Lancet Psychiatry. 2025 Jun.

. 2025 Jun;12(6):457-468.

doi: 10.1016/S2215-0366(25)00061-6. Epub 2025 Apr 10.

Authors

Affiliations

¹ Center for Precision Psychiatry, Division of Mental Health and Addiction, Oslo University Hospital, Oslo, Norway; Institute of Clinical Medicine, University of Oslo, Oslo, Norway.
² Centre for Neuropsychiatric Genetics and Genomics, Division of Psychological Medicine and Clinical Neurosciences, Cardiff University, Cardiff, UK.
³ deCODE Genetics, Reykjavik, Iceland.
⁴ Institute for Molecular Medicine Finland (FIMM), Helsinki, Finland; Department of Molecular Medicine and Surgery (MMK), Karolinska Institutet, Stockholm, Sweden.
⁵ Department of Psychiatry and Neurochemistry, Institute of Neuroscience and Physiology at the Sahlgrenska Academy, University of Gothenburg, Gothenburg, Sweden.
⁶ Estonian Genome Centre, Institute of Genomics, University of Tartu, Tartu, Estonia.
⁷ Centre for Clinical Brain Sciences, University of Edinburgh, Edinburgh, UK.
⁸ Center for Precision Psychiatry, Division of Mental Health and Addiction, Oslo University Hospital, Oslo, Norway; Institute of Clinical Medicine, University of Oslo, Oslo, Norway; Department of Medical Genetics, Oslo University Hospital, Oslo, Norway; KG Jebsen Centre for Neurodevelopmental Disorders, University of Oslo, Oslo, Norway; Oslo University Hospital, Oslo, Norway.
⁹ Janssen Research and Development, Neuroscience, Titusville, NJ, USA.
¹⁰ Department of Medical Epidemiology and Biostatistics, Karolinska Institutet, Stockholm, Sweden.
¹¹ deCODE Genetics, Reykjavik, Iceland; Faculty of Medicine, University of Iceland, Reykjavik, Iceland.
¹² Centre for Clinical Brain Sciences, University of Edinburgh, Edinburgh, UK; Generation Scotland, Centre for Medical Informatics, University of Edinburgh, Edinburgh, UK.
¹³ Department of Psychiatry and Neurochemistry, Institute of Neuroscience and Physiology at the Sahlgrenska Academy, University of Gothenburg, Gothenburg, Sweden; Department of Medical Epidemiology and Biostatistics, Karolinska Institutet, Stockholm, Sweden.
¹⁴ Smerud Medical Research International AS, Oslo, Norway.
¹⁵ P1vital, Wallingford, UK.
¹⁶ Institute of Biological Psychiatry, Mental Health Services, Copenhagen University Hospital, Copenhagen, Denmark; Department of Clinical Medicine, University of Copenhagen, Denmark.
¹⁷ Institute of Biological Psychiatry, Mental Health Services, Copenhagen University Hospital, Copenhagen, Denmark.
¹⁸ Department of Psychiatry, Psychosomatic Medicine and Psychotherapy, University Medical Centre Frankfurt, Frankfurt, Germany; Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Frankfurt, Germany.
¹⁹ Center for Psychopharmacology, Diakonhjemmet Hospital, Oslo, Norway; Department of Pharmacy, University of Oslo, Oslo, Norway.
²⁰ Department of Biomedical and NeuroMotor Sciences, University of Bologna, Bologna, Italy.
²¹ Department of Medicine and Surgery, Kore University of Enna, Enna, Italy; Oasi Research Institute-IRCCS, Troina, Troina, Italy.
²² Social, Genetic and Developmental Psychiatry Centre, King's College London, London, UK; NIHR Maudsley Biomedical Research Centre, South London and Maudsley NHS Trust, London, UK.
²³ Center for Precision Psychiatry, Division of Mental Health and Addiction, Oslo University Hospital, Oslo, Norway; Institute of Clinical Medicine, University of Oslo, Oslo, Norway; KG Jebsen Centre for Neurodevelopmental Disorders, University of Oslo, Oslo, Norway; Oslo University Hospital, Oslo, Norway. Electronic address: ole.andreassen@medisin.uio.no.

PMID: 40222385
DOI: 10.1016/S2215-0366(25)00061-6

Abstract

Although information from real-world data can be used to identify factors that aid treatment choice, there are no guidelines for the use of such data. The aim of this Review is to summarise and evaluate definitions of treatment outcomes for antidepressants, antipsychotics, and mood stabilisers when using real-world data, and to suggest standards for the field. Given that no standards for the use of these data in estimating treatment outcomes exist, variability is high for treatment outcome definitions. We make recommendations for different scenarios of available data and highlight the importance of using other sources of information to validate proxy measures such as continued treatment, switching between medications, or polypharmacy of psychotropic medications. Well defined and validated treatment outcome measures that incorporate real-world data could facilitate the development of precision psychiatry approaches and support regulatory decision making regarding psychopharmacological agents.

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Conflict of interest statement

Declaration of interests OAA has received speaker fees from Lundbeck, Janssen, Otsuka, and Sunovion and is a consultant to Cortechs.ai. and Precision Health. CML sits on the scientific advisory board for Myriad Neuroscience. AS has served as a consultant or speaker for Abbott, AbbVie, Angelini Pharma, AstraZeneca Clinical Data, Boehringer Ingelheim, Bristol Myers Squibb, Eli Lilly, GlaxoSmithKline, InnovaPharm, Italfarmaco, Janssen, Lundbeck, Naurex, Pfizer, Polifarma, Sanofi, Servier, and Taliaz. EM has received speaker's honoraria from Lundbeck and Otsuka. GRD is the owner of P1vital. GE, KSt, and HS are employed at deCODE genetics, a subsidiary of Amgen.

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Recommendations for defining treatment outcomes in major psychiatric disorders using real-world data

Affiliations

Recommendations for defining treatment outcomes in major psychiatric disorders using real-world data

Authors

Affiliations

Abstract

Conflict of interest statement

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LinkOut - more resources

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Medical