Advertisement
Review| Volume 45, ISSUE 3, P347-353, February 2009

Download started.

Ok

Patient-reported outcomes: Assessment and current perspectives of the guidelines of the Food and Drug Administration and the reflection paper of the European Medicines Agency

Published:November 17, 2008DOI:https://doi.org/10.1016/j.ejca.2008.09.032

      Abstract

      Aims

      Patient-reported outcomes (PROs) have recently gained greater credibility with regulatory bodies aiming to standardise their use and interpretation in RCTs, thereby supporting medicinal product submissions. For this reason, the United States (US) Food and Drug Administration (FDA) and the European Medicines Agency (EMEA) have released guidelines. This review paper provides an overview of the current perspectives and views on these guidelines.

      Method

      To evaluate the FDA and EMEA PRO guidelines, 47 expert responses to the FDA guidance were qualitatively reviewed. Two reviewers independently extracted data from these letters and checked these responses to warrant consistency and agreement in the evaluation process. A PubMed literature review was systematically examined to obtain supporting evidence or related articles for both the guidance documents.

      Results

      Generally, there is agreement between regulatory authorities and the research community on the contents of the FDA and EMEA PRO draft guidance. However, disagreements exist on significant philosophical topics (e.g. the FDA focuses more on conceptual models and symptoms than the EMEA) and design topics (e.g. the FDA is more restrictive on issues of recall bias, blinding of oncology trials and degrees of psychometric validation than researchers and the EMEA). This could influence the approval of PRO claims.

      Conclusion

      PRO guidance from the EMEA and FDA has been valuable, and has raised the profile and active debate of PROs in oncology. However, our review of the current opinion shows that there are controversial aspects of the guidance. Consequently, greater latitude should be given to how the guidance is interpreted and applied.

      Keywords

      To read this article in full you will need to make a payment

      Purchase one-time access:

      Academic & Personal: 24 hour online accessCorporate R&D Professionals: 24 hour online access
      One-time access price info
      • For academic or personal research use, select 'Academic and Personal'
      • For corporate R&D use, select 'Corporate R&D Professionals'

      Subscribe:

      Subscribe to European Journal of Cancer
      Already a print subscriber? Claim online access
      Already an online subscriber? Sign in
      Institutional Access: Sign in to ScienceDirect

      References

        • Bottomley A.
        Developing clinical trial protocols for quality of life assessment.
        Appl Clin Trials. 2001; 10: 40-44
        • Lipscomb J.
        • Gotay C.C.
        • Snyder C.
        Introduction to outcomes assessment in cancer.
        in: Lipscomb J. Gotay C.C. Snyder C. Outcomes assessment in cancer: measures, methods, and applications. Cambridge University Press, Cambridge, UK2005: 1-14
        • Bottomley A.
        • Aaronson N.K.
        European Organisation for Research and Treatment of Cancer International perspective on health-related quality-of-life research in cancer clinical trials: the European Organisation for Research and Treatment of Cancer experience.
        J Clin Oncol. 2007; 25: 5082-5086
        • Lipscomb J.
        • Gotay C.C.
        • Snyder C.F.
        Patient-reported Outcomes in cancer: a review of recent research and policy initiatives.
        CA Cancer J Clin. 2007; 57: 278-300
      1. Food and Drug Administration. Guidance for industry. Patient-reported outcome measures: use in medical product development to support labeling claims. US Food and Drug administration website, 2006 [cited 2007 April]. <http:www.fda.gov/cder/guidance/5460dft.pdf>.

      2. European Medicines Agency. Committee for medicinal products for human use (CHMP). Reflection paper on the regulatory guidance for the use of health-related quality of life (HRQL) measures in the evaluation of medicinal products. European Medicines Agency website, 2005 [cited 2007 April]. <http://www.emea.europa.eu/pdfs/human/ewp/13939104en.pdf>.

      3. Burke L, Rock EP, Powers JH. Patient-reported outcome instruments: overview and comments on the FDA draft guidance. US Food and Drug administration website, 2006 [cited 2007 Oct.]. <http://www.fda.gov/cder/present/DJA2006/Bruke_Rock.ppt>.

      4. Lenderking W, Stewart M, Merikle E. Coping with the validation requirements of the FDA PRO guidance: the intersection of science and practice. In: Conference proceedings, 14th annual conference of the international society for quality of life research, Toronto, Canada; 2007. p. 8.

        • Bolton J.E.
        Accuracy of recall of usual pain intensity in back pain patients.
        J Pain. 1999; 83: 533-539
        • Jamison R.N.
        • Raymond S.A.
        • Slawsby E.A.
        • McHugo G.J.
        • Baird J.C.
        Pain assessment in patients with low back pain: comparison of weekly recall and momentary electronic data.
        J Pain. 2006; 7: 192-199
        • Middel B.
        • Goudriaan H.
        • de Greef M.
        • Stewart R.
        • van Sonderen E.
        • Bouma J.
        • et al.
        Recall bias did not affect perceived magnitude of change in health-related functional status.
        J Clin Epidemiol. 2006; 59: 503-511
        • Erskine A.
        • Morley S.
        • Pearce S.
        Memory of pain: a review.
        Pain. 1990; 41: 255-265
        • Stone A.A.
        • Broderick J.E.
        • Shiffman S.S.
        • Schwartz J.E.
        Understanding recall of weekly pain from a momentary assessment perspective: absolute agreement, between- and within-person consistency, and judged change in weekly pain.
        Pain. 2004; 107: 61-69
        • Brauer C.
        • Thomsen J.F.
        • Loft I.P.
        • Mikkelsen S.
        Can we rely on retrospective pain assessments?.
        Am J Epidemiol. 2003; 157: 552-557
        • Gendrau M.
        • Hufford M.R.
        • Stone A.A.
        Measuring clinical pain in chronic widespread pain: selected methodological issues.
        Best Pract Res Clin Rheumatol. 2003; 17: 575-592
        • Revicki D.A.
        • Gnanasakthy A.
        • Weinfurt K.
        Documenting the rationale and psychometric characteristics of patient reported outcomes for labeling and promotional claims: the PRO Evidence Dossier.
        Qual Life Res. 2007; 16: 717-723
        • Sloan J.A.
        • Frost M.H.
        • Berzon R.
        • et al.
        The clinical significance of quality of life assessments in oncology: a summary for clinicians.
        Support Care Cancer. 2006; 14: 988-998
        • Joly F.
        • Vardy J.
        • Pintilie M.
        • Tannock I.F.
        Quality of life and/or symptom control in randomized clinical trials for patients with advanced cancer.
        Ann Oncol. 2007; 18 ([Epub 2007]): 1935-1942
        • Marquis P.
        • Arnould B.
        • Acquadro C.
        • Roberts W.M.
        Patient-reported outcomes and health-related quality of life in effectiveness studies: pros and cons.
        Drug Develop Res. 2006; 67: 193-201
        • Rock E.P.
        • Scott J.A.
        • Kennedy D.L.
        • Sridhara R.
        • Pazdur R.
        • Burke L.B.
        Challenges to use of health-related quality of life for Food and Drug Administration approval of anticancer products.
        J Natl Cancer Inst Monogr. 2007; : 27-30
        • Acquadro C.
        • Conway K.
        • Hareendran A.
        • Aaronson N.
        European Regulatory Issues and Quality of Life Assessment (ERIQA) Group Literature review of methods to translate health-related quality of life questionnaires for use in multinational clinical trials.
        Value Health. 2008; 11 (Epub 2007): 509-521
        • Rock E.P.
        • Kennedy D.L.
        • Furness M.H.
        • Pierce W.F.
        • Pazdur R.
        • Burke L.B.
        Patient-reported outcomes supporting anticancer product approvals.
        J Clin Oncol. 2007; 25: 5094-5099
        • Coombs J.H.
        • McBurney C.R.
        • Gondek K.
        • Copley-Merriman K.
        Evidence of patient reported outcomes in labelling for oncology products: evaluation of United States and European labels.
        Qual Life Newslett. 2002; : 29
        • Aaronson N.K.
        • Ahmedzai S.
        • Bergman B.
        • et al.
        The European Organization for Research and Treatment of Cancer QLQ-C30: a quality-of-life instrument for use in international clinical trials in oncology.
        J Natl Cancer Inst. 1993; 85: 365-376
        • Bergman B.
        • Aaronson N.K.
        • Ahmedzai S.
        • Kaasa S.
        • Sullivan M.
        The EORTC QLQ-LC13: a modular supplement to the EORTC core quality of life questionnaire (QLQ-C30) for use in lung cancer clinical trials. EORTC study group on quality of life.
        Eur J Cancer. 1994; 30A: 635-642
        • Bottomley A.
        The journey of health-related quality of life assessment.
        Lancet Oncol. 2008; 9: 906