Skip to main content
    • Aa
    • Aa

EMEA and the evaluation of health-related quality of life data in the drug regulatory process

  • Sotiria Papanicolaou (a1), David Sykes (a1) and Elias Mossialos (a2)

Objectives: This study reviewed the European guidelines of the Committee for Proprietary Medicinal Products (CPMP) on how health-related quality of life (HRQOL) research should be conducted in clinical trials. Published product-level information was also reviewed to investigate the actual role of HRQOL data in the European regulatory process.

Methods: All disease-specific notes for guidance and concept papers on clinical investigations, development and evaluation of human medicinal products, as well as the European Public Assessment Reports (EPAR) of all approved drugs published on the European Agency for the Evaluation of Medicinal Products (EMEA) Web site were evaluated for their HRQOL recommendations.

Results: Only twenty of the fifty CPMP guidance notes for clinical investigation of pharmaceutical products in specific disease areas included a reference to HRQOL. Most of the recommendations were generic and vague, and the terminology used was inconsistent across documents. The EPAR provided nonspecific information about HRQOL and contradictory conclusions on the effect of a drug on HRQOL sometimes occurred in different documents. The criteria used by the CPMP to assess the HRQOL data could not be identified due to an ad hoc approach to the inclusion of data in the EPAR.

Conclusions: A more systematic approach is needed on the way health outcomes data are considered, reviewed, and interpreted by the regulatory authorities. For this to be achieved, CPMP should develop general guidelines on the importance of HRQOL and how research should be conducted if data are to be included in the registration process.

Linked references
Hide All

This list contains references from the content that can be linked to their source. For a full set of references and notes please see the PDF or HTML where available.

ApoloneG, De CarliG, BrunettiM, GarattiniS. 2001Health-related quality of life (HR-QOL) and regulatory issues. Pharmacoeconomics.19: 187195.

BechP. 1992Issues of concern in the standardization and harmonization of drug trials in Europe: Health-related quality of life, ESCT meeting, Strasbourg, May 23–24, 1991. Qual Life Res.1: 143145.

BurkeLB. 2001US regulation of pharmaceutical outcomes research. Value Health.4: 57.

ChassanyO, SagnierP, MarquisP, FullertonS, AaronsonN. 2002Patient-reported outcomes: The example of health-related quality of life—a European guidance document for the improved integration of health-related quality of life assessment in the drug regulatory process. Drug Information J.36: 209238

CorcosJ, BeaulieuS, DonovanJ, NaughtonM, GotohM, and members of the symptom and quality of life assessment committee of the first international consultation on incontinence. 2002Quality of life assessment in men and women with urinary incontinence. J Urol.168: 896905.

DowieJ. 2002Decision validity should determine whether a generic or condition-specific HRQOL measure is used in health care decisions. Health Econ.11: 18.

FantoniM, IzziI, Del BorgoC, et al. 1999Inter-rater reliability of a modified Karnofsky scale of performance status for HIV-infected individuals. AIDS Patient Care STDS.13: 2328.

FayersP, BottomleyA. 2002Quality of life research within the EORTC—the EORTC QLQ-C30. European Organisation for Research and Treatment of Cancer. Eur J Cancer.38(Suppl 4): S125S133.

FeenyD. 2002Commentary on Jack Dowie, “Decision validity should determine whether a generic or condition-specific HRQOL measure is used in health care decisions.”Health Econ.11: 1316.

GuyattG. 2002Commentary on Jack Dowie, “Decision validity should determine whether a generic or condition-specific HRQOL measure is used in health care decisions.”Health Econ.11: 912.

GuyattGH, JaeschkeRJ. 1997Reassessing quality-of-life instruments in the evaluation of new drugs. Pharmacoeconomics.12: 621626.

LeeCW, ChiKNJ. 2000The standard of reporting of health-related quality of life in clinical cancer trials. Clin Epidemiol.53: 451458.

LeidyNK, RevickiDA, GenesteB. 1999Recommendations for evaluating the validity of quality of life claims for labelling and promotion. Value Health2: 113127.

MontazeriA, McEwenJ, GillisCR. 1996Quality of life in patients with ovarian cancer: Current state of research. Support Care Cancer.4: 169179.

NeymarkN, KiebertW, TorfsK, et al. 1998Methodological and statistical issues of quality of life (QoL) and economic evaluation in cancer clinical trials: Report of a workshop. Eur J Cancer.34: 13171333.

RadenskyP. 2001Regulation of pharmacoeconomics and outcomes research. Value Health.4: 1215.

RevickiDA, EhrethJL. 1997Health-related quality-of-life assessment and planning for the pharmaceutical industry. Clin Ther.19: 11011115.

RevickiDA, OsobaD, FaircloughD, et al. 2000Recommendations on health-related quality of life research to support labeling and promotional claims in the United States. Qual Life Res.9: 887900.

RevickiDA, RothmanM, LuceB. 1992Health-related quality of life assessment and the pharmaceutical industry. Pharmacoeconomics.1: 394408.

RogersSN, GwanneS, LoweD, et al. 2002The addition of mood and anxiety domains to the University of Washington quality of life scale. Head Neck.24: 521529.

SantanelloNC, BakerDB, CappelleriJC, et al. 2002Regulatory issues for health-related quality of life-PhRMA Health Outcomes Committee Workshop, 1999. Value Health.5: 1425.

SloanJA, CellaD, FrostM, et al. 2002Clinical Significance Consensus Meeting Group. Assessing clinical significance in measuring oncology patient quality of life: Introduction to the symposium, content overview, and definition of terms. Mayo Clin Proc.77: 367370.

SymondsT, BerzonR, MarquisP, RummansTA. 2002The clinical significance of quality-of-life results: Practical considerations for specific audiences. Clinical Significance Consensus Meeting Group. Mayo Clin Proc.77: 572583.

TraskPC, PatersonAG, WangC, et al. 2001Cancer-specific worry interference in women attending a breast and ovarian cancer risk evaluation program: Impact on emotional distress and health functioning. Psychooncology.10: 349360.

Recommend this journal

Email your librarian or administrator to recommend adding this journal to your organisation's collection.

International Journal of Technology Assessment in Health Care
  • ISSN: 0266-4623
  • EISSN: 1471-6348
  • URL: /core/journals/international-journal-of-technology-assessment-in-health-care
Please enter your name
Please enter a valid email address
Who would you like to send this to? *



Full text views

Total number of HTML views: 2
Total number of PDF views: 3 *
Loading metrics...

Abstract views

Total abstract views: 43 *
Loading metrics...

* Views captured on Cambridge Core between September 2016 - 21st September 2017. This data will be updated every 24 hours.