Letter to the Editor Received: November 22, 2013 Accepted after revision: November 28, 2013 Published online: January 22, 2014
Psychother Psychosom 2014;83:119 DOI: 10.1159/000357623
Avoiding Surrogate Measures and Incorporating Subjective Experience into Clinical Research Andrew Shepherd University of Manchester, Manchester, UK
Arfken and Balon [1] present a timely overview of the role of outcome measures currently used in the clinical trial of psychotropic medication. Their call for large trials with outcome measures providing information directly relevant to the patient and clinician is important, echoing similar cries across medical practice [2]. I believe that two additional concerns need addressing however. Firstly, the use of symptom-based outcome scales in clinical trials serves as surrogate, or proxy, measures for change. More concrete, distal measures include those identified by Arfken and Balon [1] in their discussion – mortality and quality of life, for example. Recent findings in the treatment of diabetes have demonstrated that a focus on the proximal surrogate measure can lead to the neglect of serious outcomes, for example rising all-cause mortality rates [3, 4]. By focussing on the immediate short-term clinical trial finding we risk missing complications only identified through the consideration of fixed outcome measures. Secondly, the experience of psychiatric symptoms is a subjective experience. While parallels will exist between all those with hallucinatory experiences, the individual experience can never adequately be represented through population-level assessment with standardised, quantitative outcome measure application. The impact of mental disorder, or suffering, can affect the individuals’ narrative appreciation of their own identity and self-trust even before the onset of psychiatric symptoms, as evidenced by a consideration of first-person accounts [5, 6]. Successful receipt of support from mental health services may represent a revision of the personal-narrative experience [7]. Further consideration regarding the individual nature of mental disorder is displayed by the rise of the Recovery Movement, which describes the development of in-
© 2014 S. Karger AG, Basel 0033–3190/14/0832–0119$39.50/0 E-Mail [email protected] www.karger.com/pps
dividual experience and personal flourishing in the face of extreme distress [8, 9]. Appreciation of this subjective experience by an external observer is complex but necessary if we are to develop interventions that can be truly tailored to each individual’s personal recovery needs. In-depth qualitative methods are complex and time consuming but provide us with a lens through which subjective experience can be appreciated. Therefore, in addition to the adoption of simple pertinent quantitative outcome measures in clinical trials, it is also important that qualitative measures be incorporated. Qualitative study can never be fully comprehensive, but the purposive, theoretical sampling of participants within clinical trials will provide a wealth of valuable data. By including these measures as a thread running throughout the trials called for by Arfken and Balon [1] we can begin to gain an appreciation of subjective experience, or well-being, at each stage in the research process or treatment intervention [10]. Such understanding will provide us with the required information to support individuals in their personal treatment choices. References 1 Arfken C, Balon R: Another look at outcomes and outcome measures in psychiatry: cui bono? Psychother Psychosom 2014;83:6–9. 2 Godlee F: Outcomes that matter to patients. BMJ 2012;344:e318. 3 Cohen D: Rosiglitazone: what went wrong? BMJ 2010;341:c4848. 4 Yudkin JS, Lipska KJ, Montori VM: The idolatry of the surrogate. BMJ 2011;343:d7995. 5 Gray J: The chasm within: my battle with personality disorder. Philos Psychiat Psychol 2011;18:185–190. 6 Potter NN: Narrative selves, relations of trust, and bipolar disorder. Philos Psychiatr Psychol 2013;20:57–65. 7 Adler JM, McAdams DP: The narrative reconstruction of psychotherapy. Narrative Inquiry 2007;17:179–202. 8 Anthony WA: Recovery from mental illness: the guiding vision of the mental health service system in the 1990s. Psychosoc Rehabil J 1993;16: 11–23. 9 Deegan P: Recovery as a journey of the heart. Psychiatr Rehabil J 1996; 19:91–97. 10 Lee H, Vlaev I, King D, Mayer E, Darzi A: Subjective well-being and the measurement of quality in healthcare. Soc Sci Med 2013;99:27–34.
Dr. Andrew Shepherd NIHR Doctoral Research Fellow University of Manchester Oxford Road, Manchester M13 9PL (UK) E-Mail andrew.shepherd3 @ nhs.net
Copyright: S. Karger AG, Basel 2014. Reproduced with the permission of S. Karger AG, Basel. Further reproduction or distribution (electronic or otherwise) is prohibited without permission from the copyright holder.
Psychother Psychosom 2014;83:119 DOI: 10.1159/000357623
Avoiding Surrogate Measures and Incorporating Subjective Experience into Clinical Research Andrew Shepherd University of Manchester, Manchester, UK
Arfken and Balon [1] present a timely overview of the role of outcome measures currently used in the clinical trial of psychotropic medication. Their call for large trials with outcome measures providing information directly relevant to the patient and clinician is important, echoing similar cries across medical practice [2]. I believe that two additional concerns need addressing however. Firstly, the use of symptom-based outcome scales in clinical trials serves as surrogate, or proxy, measures for change. More concrete, distal measures include those identified by Arfken and Balon [1] in their discussion – mortality and quality of life, for example. Recent findings in the treatment of diabetes have demonstrated that a focus on the proximal surrogate measure can lead to the neglect of serious outcomes, for example rising all-cause mortality rates [3, 4]. By focussing on the immediate short-term clinical trial finding we risk missing complications only identified through the consideration of fixed outcome measures. Secondly, the experience of psychiatric symptoms is a subjective experience. While parallels will exist between all those with hallucinatory experiences, the individual experience can never adequately be represented through population-level assessment with standardised, quantitative outcome measure application. The impact of mental disorder, or suffering, can affect the individuals’ narrative appreciation of their own identity and self-trust even before the onset of psychiatric symptoms, as evidenced by a consideration of first-person accounts [5, 6]. Successful receipt of support from mental health services may represent a revision of the personal-narrative experience [7]. Further consideration regarding the individual nature of mental disorder is displayed by the rise of the Recovery Movement, which describes the development of in-
© 2014 S. Karger AG, Basel 0033–3190/14/0832–0119$39.50/0 E-Mail [email protected] www.karger.com/pps
dividual experience and personal flourishing in the face of extreme distress [8, 9]. Appreciation of this subjective experience by an external observer is complex but necessary if we are to develop interventions that can be truly tailored to each individual’s personal recovery needs. In-depth qualitative methods are complex and time consuming but provide us with a lens through which subjective experience can be appreciated. Therefore, in addition to the adoption of simple pertinent quantitative outcome measures in clinical trials, it is also important that qualitative measures be incorporated. Qualitative study can never be fully comprehensive, but the purposive, theoretical sampling of participants within clinical trials will provide a wealth of valuable data. By including these measures as a thread running throughout the trials called for by Arfken and Balon [1] we can begin to gain an appreciation of subjective experience, or well-being, at each stage in the research process or treatment intervention [10]. Such understanding will provide us with the required information to support individuals in their personal treatment choices. References 1 Arfken C, Balon R: Another look at outcomes and outcome measures in psychiatry: cui bono? Psychother Psychosom 2014;83:6–9. 2 Godlee F: Outcomes that matter to patients. BMJ 2012;344:e318. 3 Cohen D: Rosiglitazone: what went wrong? BMJ 2010;341:c4848. 4 Yudkin JS, Lipska KJ, Montori VM: The idolatry of the surrogate. BMJ 2011;343:d7995. 5 Gray J: The chasm within: my battle with personality disorder. Philos Psychiat Psychol 2011;18:185–190. 6 Potter NN: Narrative selves, relations of trust, and bipolar disorder. Philos Psychiatr Psychol 2013;20:57–65. 7 Adler JM, McAdams DP: The narrative reconstruction of psychotherapy. Narrative Inquiry 2007;17:179–202. 8 Anthony WA: Recovery from mental illness: the guiding vision of the mental health service system in the 1990s. Psychosoc Rehabil J 1993;16: 11–23. 9 Deegan P: Recovery as a journey of the heart. Psychiatr Rehabil J 1996; 19:91–97. 10 Lee H, Vlaev I, King D, Mayer E, Darzi A: Subjective well-being and the measurement of quality in healthcare. Soc Sci Med 2013;99:27–34.
Dr. Andrew Shepherd NIHR Doctoral Research Fellow University of Manchester Oxford Road, Manchester M13 9PL (UK) E-Mail andrew.shepherd3 @ nhs.net
Copyright: S. Karger AG, Basel 2014. Reproduced with the permission of S. Karger AG, Basel. Further reproduction or distribution (electronic or otherwise) is prohibited without permission from the copyright holder.
