Skip to main content
SpringerLink
Log in

Role of Patient-Reported Outcome Measures in the Assessment of Central Nervous System Agents

  • Original Article
  • Published:
Therapeutic Innovation & Regulatory Science Aims and scope Submit manuscript

Abstract

This work aimed to provide an understanding of the current use and regulatory acceptability of patient-reported outcome (PRO) measures in labeling claims for central nervous system (CNS) agents. A subset of CNS agents was identified from all New Drug Approvals and Biologic License Applications for new drugs approved in the US from January 2006 to June 2012. Clinician-reported outcomes (ClinROs) (62%) and PROs (38%) were the most widely used primary outcome measures. The PROs were frequently used in combination with ClinROs. Twelve PRO claims were granted across 41% of CNS drug approvals: 83%, symptoms; 17%, functioning. The PROs are frequently utilized as primary and secondary end points in CNS agents, and labeling claims are granted at higher levels than for non-CNS agents (41% vs 24%, respectively). These claims are granted at a lower rate than expected, given that direct patient input may lend valuable insight to treatment impacts in most CNS diseases.

This is a preview of subscription content, log in via an institution to check access.

Access this article

Log in via an institution

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Institutional subscriptions

Similar content being viewed by others

References

  1. World Health Organization. Neurological disorders. Public health challenges. https://www.who.int/mental_health/neurology/neurological_disorders_report_web.pdf.

  2. Soni A. The top five therapeutic classes of outpatient prescription drugs ranked by total expense for adults age 18 and older in the U.S. civilian non-institutionalized population, 2005. Statistical Brief #198. January 2008. https://meps.ahrq.gov/data_files/publications/st198/stat198.shtml.

  3. IMS Institute for Healthcare Informatics. The use of medicines in the United States: review of 2010. Available at: https://www.imshealth.com/imshealth/Global/Content/IMS%20Institute/Documents/IHII_UseOfMed_report%20.pdf. Accessed February 25, 2013.

  4. Tufts Center for the Study of Drug Development. Impact report: analysis and insight into critical drug development issues. Pace of CNS drug development and FDA approvals lags other drug classes. Volume 14. March/April 2012. Available at: https://csdd.tufts.edu/files/uploads/mar-apr_2012_ir_summary.pdf. Accessed April 20, 2013.

  5. Johnson GS. Re-purposing translational medicine and other strategies for de-risking CNS. 2007. Available at: https://knowledgebase.definedhealth.net/wp-content/uploads/2008/02/derisking-in-cns-2007-5.pdf. Accessed February 25, 2013.

  6. US Department of Health and Human Services. Guidance for industry. Patient-reported outcome measures: use in medical product development to support labeling claims. Available at: https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf. Accessed February 25, 2013.

  7. Fayers PM, Machin D. Quality of Life: The Assessment, Analysis and Interpretation of Patient-Reported Outcomes. 2nd ed. Chichester, UK: John Wiley and Sons Ltd; 2007.

    Book  Google Scholar 

  8. Kobak KA, Kane JM, Thase ME, Nierenberg AA. Why do clinical trials fail? The problem of measurement error in clinical trials: time to test new paradigms? J Clin Psychopharmacol. 2007;27:1–5.

    Article  Google Scholar 

  9. Gnanasakthy A, Mordin M, DeMuro C, Clark M, Fehnel S, Copley-Merriman K. A review of patient-reported outcomes labels in the US: 2006–2010. Value Health. 2012;15:437–442.

    Article  Google Scholar 

  10. Willke RJ, Burke LB, Erickson P. Measuring treatment impact: a review of patient-reported outcomes and other efficacy endpoints in approved product labels. Control Clin Trials. 2004;25:535–552.

    Article  Google Scholar 

  11. Koivumaa-Honkanen HT, Honkanen R, Antikainen R, Hintikka J, Viinamäki H. Self-reported life satisfaction and treatment factors in patients with schizophrenia, major depression and anxiety disorder. Acta Psychiatr Scand. 1999;5:377–384.

    Google Scholar 

  12. Fleischhacker WW, Rabinowitz J, Kemmler G, Eerdekens M, Mehnert A. Perceived functioning, well-being and psychiatric symptoms in patients with stable schizophrenia treated with long-acting risperidone for 1 year. Br J Psychiatry. 2005;187:131–136.

    Article  Google Scholar 

  13. Coghill D, Danckaerts M, Sonuga-Barke E, Sergeant J; ADHD European Guidelines Group. Practitioner review: quality of life in child mental health. Conceptual challenges and practical choices. J Child Psychol Psychiatry. 2008;50:544–561.

    Article  Google Scholar 

  14. Chue P. The relationship between patient satisfaction and treatment outcomes in schizophrenia. J Psychopharmacol. 2006;20:38–56.

    Article  Google Scholar 

  15. McCabe R, Saidi M, Priebes S. Patient-reported outcomes in schizophrenia. Br J Psychiatry. 2007;191:s21–s28.

    Article  Google Scholar 

  16. US Department of Health and Human Services. Qualification process for drug development tools. Available at: https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM230597.pdf. Accessed February 25, 2013.

  17. Woodcock J, Woosley R. The FDA critical path initiative and its influence on new drug development. Annu Rev Med. 2008;59:1–12.

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Novartis Pharmaceuticals, East Hanover, NJ, USA

    Ari Gnanasakthy MSc & Simu Thomas PhD

  2. RTI Health Solutions, 200 Park Office Drive, Research Triangle Park, NC, 27709, USA

    Carla DeMuro MS & Margaret Mordin MS

  3. RTI Health Solutions, Ann Arbor, MI, USA

    Marci Clark PharmD

Authors
  1. Ari Gnanasakthy MSc
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Carla DeMuro MS
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. Marci Clark PharmD
    View author publications

    You can also search for this author in PubMed Google Scholar

  4. Margaret Mordin MS
    View author publications

    You can also search for this author in PubMed Google Scholar

  5. Simu Thomas PhD
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Carla DeMuro MS.

Rights and permissions

Reprints and permissions

About this article

Cite this article

Gnanasakthy, A., DeMuro, C., Clark, M. et al. Role of Patient-Reported Outcome Measures in the Assessment of Central Nervous System Agents. Ther Innov Regul Sci 47, 613–618 (2013). https://doi.org/10.1177/2168479013495686

Download citation

  • Received:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1177/2168479013495686

Keywords

Search

Navigation