Skip to main content

Advertisement

SpringerLink
Log in

Unique Challenges in Development, Psychometric Evaluation, and Interpretation of Daily and Event Diaries as Endpoints in Clinical Trials

  • Special Section on Clinical Outcome Assessments: Review
  • Published:
Therapeutic Innovation & Regulatory Science Aims and scope Submit manuscript

Abstract

By bringing data collection closer to real time and minimizing recall bias, patient diaries or event-driven logs offer substantial benefits over retrospective questionnaires for many patient-reported disease concepts. Such assessments are increasingly used to support primary and secondary endpoints in randomized controlled trials. These diaries have the potential to provide more reliable and valid assessment of patients’ subjective experiences of symptoms and disease events. However, use of such diaries presents significant and unique challenges depending on the context of use. Of note, while symptom-related label claims are those most frequently granted by regulatory authorities, no guidance specific to support the development, psychometric evaluation, and interpretation of endpoints derived from patient diaries exists. This article provides an overview of key methodological, statistical, and clinical considerations for implementation of patient diaries with a regulatory perspective in mind. Approaches and solutions covered in this article include (1) techniques to establish content validity based on obtaining qualitative insights in naturalistic settings and real-life experience of diary completion, (2) demonstration of psychometric properties with respect to day-to-day variability, and (3) aggregation of data from multiple days/events to move from items to endpoints. The importance of the patients’ engagement is highlighted in order to help overcome these challenges throughout all stages of diary and endpoint development and evaluation. This article can inform researchers who are developing or implementing patient diaries as clinical trial endpoints to ensure that the nuances of this mode of data collection are considered in the development of endpoints and prior to regulatory interactions.

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. US Food & Drug Administration. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims. http://www.da.gov/downloads/drugs/guidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf. Published 2009. Accessed December 5, 2012.

  2. Mehl MR, Conner TS. Handbook of Research Methods for Studying Daily Life. New York: Guilford; 2013.

    Google Scholar 

  3. Nelsen LM, Gater A, Hall R, Coons S. Identifying and measuring the core symptoms reported by persons with asthma: a review of the existing qualitative literature and patient-reported outcome measures. Am J Respir Crit Care Med. 2014;189:A4029.

    Google Scholar 

  4. Globe G, Martin M, Schatz M, et al. Symptoms and markers of symptom severity in asthma—content validity of the asthma symptom diary. Health Qual Life Outcomes. 2015;13:21.

    Article  Google Scholar 

  5. Mocarski M, Gater A, Fleming S, Nelsen LM, Coons SJ. Patient-Reported Outcome (PRO) Consortium’s Asthma Working Group. Development of the initial version of the asthma daily symptom diary (ADSD). Am J Respir Cril Care Med. 2014;189:A1347.

    Google Scholar 

  6. Vincent K, Kennedy S, Stratton P. Pain scoring in endometriosis: entry criteria and outcome measures for clinical trials. Report from the Art and Science of Endometriosis meeting. Fertil Steril. 2010;93 (1): 62–67.

    Article  Google Scholar 

  7. Brown JS, McNaughton KS, Wyman JF, et al. Measurement characteristics of a voiding diary for use by men and women with overactive bladder. Urology. 2003;61:802–809.

    Article  Google Scholar 

  8. Cardozo L, Lisec M, Millard R, et al. Randomized, double-blind placebo controlled trial of the once daily antimuscarinic agent solifenacin succinate in patients with overactive bladder. J Urol. 2004;172:1919–1924.

    Article  CAS  Google Scholar 

  9. Haab F, Stewart L, Dwyer P. Darifenacin, an M 3 selective receptor antagonist, is an effective and well-tolerated once-daily treatment for overactive bladder. Eur Urol. 2004;45:420–429.

    Article  CAS  Google Scholar 

  10. Dmochowski RR, Sanders SW, Appell RA, Nitti VW, Davila GW. Bladder-health diaries: an assessment of 3-day vs 7-day entries. BJU Int. 2005;96:1049–1054.

    Article  Google Scholar 

  11. US Food & Drug Administration. Guidance for Industry. Migraine: developing drugs for acute treatment. Center for Drug Evaluation and Research (CDER). http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM419465.pdf. Published 2014.

  12. Simon JA, Chandler J, Gottesdiener K, et al. Diary of hot flashes reported upon occurrence: results of a randomized double-blind study of raloxifene, placebo, and paroxetine. Menopause. 2014;21(9):938–944.

    Article  Google Scholar 

  13. US Food & Drug Administration. Guidance for Industry. Estrogen and estrogen/progestin drug products to treat vasomotor symptoms and vulvar and vaginal atrophy symptoms—recommendations for clinical evaluation. Center for Drug Evaluation and Research (CDER). http://www.fda.gov/downloads/ScienceResearch/SpecialTopics/WomensHealthResearch/UCM133343.pdf. Published 2003.

  14. Patrick DL, Burke LB, Gwaltney CJ, et al. Content validity—establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO Good Research Practices Task Force report: part 1—eliciting concepts for a new PRO instrument. Value Health. 2011;14:967–977.

    Article  Google Scholar 

  15. Wilhelm P, Perrez M, Pawlik K. Conducting research in daily life: a historical review. In: Mehl MR, Conner TS, eds. Handbook of Research Methods for Studying Daily Life. New York, NY: Guilford; 2012:62–86.

    Google Scholar 

  16. Rudell K, Tatlock S, Panter C, Arbuckle R, Symonds T. Assessing the methodological value of digital real-time collection of qualitative content in supporting in-depth qualitative interviews exploring the symptoms and impacts of gout on health-related quality of life. Value Health. 2014;7(17):A572.

    Article  Google Scholar 

  17. Arbuckle R, Tolley C, Burbridge C. How burdensome is completion of electronic patient-reported outcomes (ePRO)? Item completion times and qualitative evidence from studies in four different health conditions. Value Health. 2013;7(16):A604.

    Article  Google Scholar 

  18. Patrick DL, Burke LB, Gwaltney CJ, et al. Content validity—establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO Good Research Practices Task Force report: part 2—assessing respondent understanding. Value Health. 2011;14:978–988.

    Article  Google Scholar 

  19. Coons SJ, Gwaltney CJ, Hays RD, et al. Recommendations on evidence needed to support measurement equivalence between electronic and paper-based patient-reported outcome (PRO) measures: ISPOR ePRO Good Research Practices Task Force report. Value Health. 2009;12:419–429.

    Article  Google Scholar 

  20. Leidy NK, Wilcox TK, Jones PW, Roberts L, Powers JH, Sethi S. Standardizing measurement of chronic obstructive pulmonary disease exacerbations: reliability and validity of a patient-reported diary. Am J Respir Crit Care Med. 2011;183:323–329.

    Article  Google Scholar 

  21. Mulhall JP, Goldstein I, Bushmakin AG, Cappelleri JC, Hvidsten K. Validation of the Erection Hardness Score. J Sex Med. 2007;4:1626–1634.

    Article  Google Scholar 

  22. Wilhelm P, Schoebi D. Assessing mood in daily life. Eur J Psychol Assess. 2007;23:258–267.

    Article  Google Scholar 

  23. Shrout P, Lane S. Psychometrics. In: Mehl MR, Conner TS, eds. Handbook of Research Methods for Studying Daily Life. New York: Guilford; 2012:302–320.

    Google Scholar 

  24. Cappelleri JC, Zou KH, Bushmakin AG, Alvir JMJ, Alemayehu D, Symonds T. Patient-Reported Outcomes: Measurement, Implementation and Interpretation. Boca Raton, FL: CRC Press; 2013.

    Book  Google Scholar 

  25. De Vet HC, Terwee CB, Mokkink LB, Knol DL. Measurement in Medicine: A Practical Guide. Cambridge: Cambridge University Press; 2011.

    Book  Google Scholar 

  26. Streiner DL, Norman GR, Cairney J. Health Measurement Scales: A Practical Guide to Their Development and Use. New York: Oxford University Press; 2014.

    Google Scholar 

  27. US Food & Drug Administration. Guidance for industry: Irritable bowel syndrome—clinical evaluation of products for treatment. Draft Guidance for Industry. http://www.fda.gov/downloads/Drugs/…/Guidances/UCM205269.pdf. Published 2012.

  28. Farrar JT, Young JP, LaMoreaux L, Werth JL, Poole RM. Clinical importance of changes in chronic pain intensity measured on an 11-point numerical pain rating scale. Pain. 2001;94:149–158.

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Adelphi Values, Adelphi Mill, Grimshaw Lane, Bollington, Macclesfield, Cheshire, SK10 5JB, UK

    Adam Gater MSc

  2. Outcometrix, Tucson, AZ, USA

    Cheryl D. Coon PhD

  3. GlaxoSmithKline, King of Prussia, PA, USA

    Linda M. Nelsen MHS

  4. CERobs Consulting, Chapel Hill, NC, USA

    Cynthia Girman DrPH

Authors
  1. Adam Gater MSc
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Cheryl D. Coon PhD
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. Linda M. Nelsen MHS
    View author publications

    You can also search for this author in PubMed Google Scholar

  4. Cynthia Girman DrPH
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Adam Gater MSc.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Gater, A., Coon, C.D., Nelsen, L.M. et al. Unique Challenges in Development, Psychometric Evaluation, and Interpretation of Daily and Event Diaries as Endpoints in Clinical Trials. Ther Innov Regul Sci 49, 813–821 (2015). https://doi.org/10.1177/2168479015609649

Download citation

  • Received:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1177/2168479015609649

Keywords

Search

Navigation