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The Role of Health-Related Quality of Life Data in the Drug Approval Processes in the US and Europe

A Review of Guidance Documents and Authorizations of Medicinal Products from 2006 to 2010

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Abstract

Objective: The objective of this research was to review the extent to which health-related quality of life (HR-QOL) and other patient-reported outcomes (PROs) have played a role in drug approval and labelling since 2006, when the US FDA issued its draft guidance on the use of PRO measures and the European Medicines Agency (EMA), its reflection paper on HR-QOL.

Methods: This research was conducted through a systematic manual review of therapy area-specific regulatory guidelines (US and EU) and product labels issued during the period 1 January 2006 to 16 November 2010.

Results: Fifteen (39.5% of all guidance documents) and 34 (35.8%) guidance documents containing recommendations for the inclusion of PRO endpoints in clinical trials were released by the FDA and the EMA, respectively. The FDAreferred to HR-QOL (as a secondary endpoint) in 3 of the 15 (20%) guidances. The EMA recommended use of HR-QOL endpoints in 22 of the 34 (65%) guidance documents. The FDA approved 93 products with PRO endpoints in labelling (out of 432 total approvals). Of those, eight products (8.6% of all products with a PRO claim) documented treatment benefits characterized as HR-QOL. The EMA approved 54 products that included PRO endpoints in labelling (out of 248 total approvals), of which 16 products (29.6% of all products with a PRO claim) reflected HR-QOL data.

Conclusion: Our review shows that the patients’ perspective in clinical research is important for the EMA and FDA, with HR-QOL endpoints still playing a minor role in product claims. Our analysis suggests that the EMA’s receptivity to HR-QOL endpoints is greater than the FDA’s, and that both agencies value patient-reported symptom data. Differences between the agencies’ acceptance of PRO endpoints, especially HRQOL, might reflect differences in their structure and organization. As the world’s largest medical regulatory agency, the FDA employs an ‘army’ of experts to review submissions and develop guidance. This internal expertise enables the FDA to take an active role in defining endpoints, developing guidance and regulations that clarify their expectations, and enforcing regulatory policy. By contrast, the EMA, working with a network of over 4500 European experts, relies on clinicians and the scientific community for the definition of validity and the acceptance of an endpoint. Historically, the EMA has been more likely to accept existing measures, including global assessment and diaries, provided the assessments are supported by peer-reviewed publications of the development and validity of the instruments. Thus, the peer-review process and acceptance by the scientific and medical communities greatly influence the acceptance of PRO claims in EMA product approvals. Finally, the FDA’s stance may be driven, in part, by the potential use of PRO information in direct-to-consumer advertising and whether such measures are meaningful and interpretable by patients. For the future, we believe that the empowerment of patients in healthcare decision making will foster the use of specific PROs for evaluating the benefits of treatments on criteria that are meaningful to patients, in addition to traditional clinical endpoints.

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References

  1. 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: 535–52

    Article  PubMed  Google Scholar 

  2. Apolone G, De Carli G, Brunetti M, et al. Health-related quality of life (HR-QOL) and regulatory issues: an assessment of theEuropeanAgency for the Evaluation of Medicinal Products (EMEA) recommendations on the use of HR-QOL measures in drug approval. Pharmacoeconomics 2001; 19: 187–95

    Article  PubMed  CAS  Google Scholar 

  3. Papanicolaou S, Sykes D, Mossialos E. EMEA and the evaluation of healthrelated quality of life data in the drug regulatory process. Int J Technol Assess Health Care 2004; 20: 311–24

    Article  PubMed  Google Scholar 

  4. Szende A, Leidy NK, Revicki D. Health-related quality of life and other patient-reported outcomes in the European centralized drug regulatory process: a review of guidance documents and performed authorizations of medicinal products 1995 to 2003. Value Health 2005; 8: 534–48

    Article  PubMed  Google Scholar 

  5. US Department of Health and Human Services, Food and Drug Administration. Patient-reported outcome measures: use in medical product development to support labeling claims. Fed Regist 2006; 71 (23): 5862–3

    Google Scholar 

  6. US Department of Health and Human Services, Food and Drug Administration. Patient-reported outcome measures: use in medical product development to support labeling claims. Fed Regist 2009; 74 (35): 65132–3

    Google Scholar 

  7. European Medicines Agency. EMEA/CHMP/EWP/139391/2004. Reflection paper on the regulatory guidance for the use of health-related quality of life (HRQL) measures in the evaluation of medicinal products. London: EMA, 2005 Jul 27

    Google Scholar 

  8. FDA. Clinical studies section of labelling for human prescription drug and biological products: content and format. Guidance for industry [online]. Available from URL: http://www.fda.gov/RegulatoryInformation/Guidances/ucm127509.htm [Accessed 2011 Jun 9]

  9. European Communities Commission, Directorate-General for Industry, Pharmaceuticals and Cosmetics. Eudralex, the rules governing medicinal products in the European Union. Volume 2C-Regulatory Guidelines. Guideline on Summary of Product Characteristics-SmPC (September 2009) [online]. Available from URL: http://ec.europa.eu/health/files/eudralex/vol-2/c/smpc_guideline_rev2_en.pdf [Accessed 2011 May 23]

    Google Scholar 

  10. Caron M, Emery MP, Marquis P, et al. Recent trends in the inclusion of patient-reported outcome (PRO) data in approved drugs labeling by the FDA and EMEA. Patient-Report Outcomes Newsletter 2008; 40: 8–10

    Google Scholar 

  11. Caron M, Emery MP, Marquis P, et al. Patient-reported outcome (PRO) labeling claims in pain treatment: overview of US and European drug approvals [abstract]. Presented at the ISPOR 14th Annual International Meeting; May 16–20, 2009; Orlando (FL)

    Google Scholar 

  12. Scott J, Caron M, Emery MP. Label claims based on patient-reported outcomes in EMEA and FDA approvals since 2000 [abstract]. Presented at the 11th Annual European Meeting of the ISPOR; Nov 2008; Athens

    Google Scholar 

  13. US Food and Drug Administration. Chronic cutaneous ulcer and burn wounds: developing products for treatment, 1 June 2006 [online]. Available from URL: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm071324.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  14. US Food and Drug Administration. Sinusitis: designing clinical development programs of nonantimicrobial drugs for treatment, 21 Nov 2006. Draft guidance [online]. Available from URL: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm072037.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  15. US Food and Drug Administration. Developing products for weight management, revision 1, 14 Feb 2007 [online]. Available from URL: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm071612.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  16. US Food and Drug Administration. Orally inhaled and intranasal corticosteroids: evaluation of the effects on growth in children, 5 March 2007 [online]. Available from URL: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm071968.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  17. US Food andDrugAdministration. Clinical trial endpoints for the approval of cancer drugs and biologics, 15 May 2007. Draft guidance [online]. Available from URL: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm071590.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  18. US Food and Drug Administration. Acute bacterial sinusitis: developing antimicrobial drugs for treatment, 29 Oct 2007. Draft guidance [online]. Available from URL: http://www.fda.gov/cder/guidance/3895dft.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  19. US Food and Drug Administration. Chronic obstructive pulmonary disease: developing drugs for treatment, 8 Nov 2007. Draft guidance [online]. Available from URL: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm071575.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  20. US Food and Drug Administration. Acute bacterial otitis media: developing drugs for treatment, 17 Jan 2008 [online]. Available from URL: http://www.fda.gov/cder/guidance/3892dft.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  21. US Food and Drug Administration. Acute bacterial exacerbations of chronic bronchitis in patients with chronic obstructive pulmonary disease: developing antimicrobial drugs for treatment, 21 Aug 2008. Draft guidance [online]. Available from URL: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm070935.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  22. US Food and Drug Administration. Influenza: developing drugs for treatment and/or prophylaxis, 19 Feb 2009. Draft guidance [online]. Available from URL: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm091219.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  23. US Food and Drug Administration. Community-acquired bacterial pneumonia: developing drugs for treatment, 19 March 2009. Draft guidance [online]. Available from URL: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm123686.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  24. US Food and Drug Administration. Assessment of abuse potential of drugs, 26 Jan 2010. Draft guidance [online]. Available from URL: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM198650.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  25. US Food and Drug Administration. Irritable bowel syndrome: clinical evaluation of products for treatment, 22 March 2010. Draft guidance [online]. Available fromURL: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM205269.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  26. US Food andDrug Administration. Systemic lupus erythematosus: developing drugs for treatment, 21 Jun 2010 [online]. Available from URL: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm072063.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  27. US Food and Drug Administration. Acute bacterial skin and skin structure infections: developing drugs for treatment, 26 Aug 2010. Draft guidance [online]. Available from URL: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM071185.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  28. European Medicines Agency. CHMP/EWP/3635/03. Guideline on clinical investigation ofmedicinal products indicated for the treatment of social anxiety disorder (SAD), London, 26 Jan 2006 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003490.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  29. European Medicines Agency. CHMP/EWP/4713/03. Guideline on clinical investigation of medicinal products for the treatment of sepsis, London, 1 June 2006 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003459.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  30. European Medicines Agency. CPMP/EWP/234/95/rev. 1. Guideline on the clinical investigation of anti-anginal medicinal products in stable angina pectoris, London, 1 June 2006 [online]. Available fromURL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003316.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  31. European Medicines Agency. EMEA/CPMP/EWP/504/97 Rev 1. Guideline on clinical investigation of medicinal products in the treatment of patients with acute respiratory distress syndrome, London, 21 September 2006 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003553.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  32. European Medicines Agency. CPMP/EWP/422/04. Guideline on clinical investigation of medicinal products for the treatment of juvenile idiopathic arthritis, London, 18 Oct 2006 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003409.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  33. European Medicines Agency. CPMP/EWP/561/98. Guideline on clinical investigation of medicinal products for the treatment of multiple sclerosis, London, 16 Nov 2006 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003485.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  34. European Medicines Agency. CPMP/EWP/4937/03. Guideline on non-clinical and clinical development of medicinal products for the prevention of nausea and vomiting associated with cancer chemotherapy, London, 14 Dec 2006 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/12/WC500017746.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  35. European Medicines Agency. CHMP/EWP/438/04. Guideline on clinical investigation of medicinal products for the treatment of psoriatic arthritis, London, 14 Dec 2006 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003413.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  36. European Medicines Agency. CPMP/EWP/788/01. Guideline on clinical investigation ofmedicinal products for the treatment ofmigraine, London, 24 Jan 2007 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003482.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  37. European Medicines Agency. CPMP/EWP/252/03. Guideline on clinical medicinal products intended for the treatment of neuropathic pain, London, 24 Jan 2007 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003478.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  38. European Medicines Agency. EMEA/CPMP/555/95. Guideline on clinical trials with haematopoietic growth factors for the prophylaxis of infection following myelosuppressive or myeloablative therapy, London, 22 March 2007 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/12/WC500017738.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  39. European Medicines Agency. CPMP/EWP/281/96. Guideline on clinical evaluation of medicinal products used in weight control, London, 15 Nov 2007 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003264.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  40. European Medicines Agency. CHMP/EWP/18463/2006. Guideline on the development of new medicinal products for the treatment of ulcerative colitis, London, 24 Jan 2008 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003266.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  41. European Medicines Agency. CPMP/EWP/2284/99. Guideline on the development of new medicinal products for the treatment of Crohn’s disease, London, 24 July 2008 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003265.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  42. European Medicines Agency. CPMP/EWP/563/95. Guideline on clinical investigation of medicinal products in the treatment of Parkinson’s disease, London, 24 July 2008 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003540.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  43. European Medicines Agency. CHMP/EWP/358650/2006. Guideline on the development of medicinal products for the treatment of post-traumatic stress disorder (PTSD), London, 24 July 2008 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003489.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  44. European Medicines Agency. CHMP/EWP/263148/06. Guideline on clinical investigation of immunosuppressants for solid organ transplantation, London, 24 July 2008 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003593.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  45. European Medicines Agency. CHMP/EWP/18504/2006. Guideline on the clinical development of products for specific immunotherapy for the treatment of allergic diseases, London, 20 Nov 2008 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003605.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  46. European Medicines Agency. EMEA/CPMP/EWP/633/02. Guideline on the clinical development of medicinal products for the treatment of HIV infection, London, 20 Nov 2008 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003460.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  47. European Medicines Agency. CHMP/EWP/369963/05. Guideline on the development ofmedicinal products for the treatment of smoking, London, 18 Dec 2008 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003509.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  48. European Medicines Agency. CPMP/EWP/4151/00. Guideline on the requirements for clinical documentation for orally inhaled products (OIP) including the requirements for demonstration of therapeutic equivalence between two inhaled products for use in the treatment of asthma and chronic obstructive pulmonary disease (COPD) in adults and for use in the treatment of asthma in children and adolescents, London, 22 January 2009 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003504.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  49. European Medicines Agency. CPMP/EWP/4891/03. Guideline on clinical investigation of medicinal products for the treatment of ankylosing spondylitis, London, 23 April 2009 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003424.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  50. European Medicines Agency. EMEA/CHMP/EWP/9147/2008. Guideline on the clinical development of medicinal products for the treatment of cystic fibrosis, London, 22 October 2009 [online]. Available fromURL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/12/WC500017055.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  51. European Medicines Agency. EMEA/16274/2009. Guideline on medicinal products for the treatment of insomnia, London, 22 October 2009 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/11/WC500011987.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  52. European Medicines Agency. EMEA/CHMP/EWP/356954/2008. Guideline on the clinical investigations of medicinal products for the treatment of pulmonary arterial hypertension, London, 22 October 2009 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/12/WC500016686.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  53. European Medicines Agency. EMEA/CHMP/EWP/342691/2009. Guideline on the evaluation of drugs for the treatment of gastroesophageal reflux disease, London, 17 December 2009 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2010/01/WC500052741.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  54. European Medicines Agency. CPMP/EWP/784/97 Rev.1. Guideline on clinical investigation of medicinal products used in the treatment of osteoarthritis, London, 20 January 2010 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003440.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  55. European Medicines Agency. EMA/CHMP/EWP/20097/2008. Guideline on the development of medicinal products for the treatment of alcohol dependence, London, 18 February 2010 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2010/03/WC500074898.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  56. European Medicines Agency. EMA/CHMP/EWP/520088/2008. Appendix 2 to the guideline on the evaluation of anticancer medicinal products in man (CPMP/EWP/205/95 Rev. 3) on confirmatory studies in haematological malignancies, London, 18 February 2010 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2010/03/WC500074949.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  57. European Medicines Agency. EMA/CHMP/EWP/607022/2009 Guideline on the treatment of premenstrual dysphoric disorder (PMDD), London, 20 May 2010 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2010/05/WC500090882.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  58. European Medicines Agency. CPMP/EWP/562/98 Rev. 1. Guideline on clinical investigation of medicinal products in the treatment of chronic obstructive pulmonary disease (COPD), London, 2 July 2010 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2010/09/WC500096769.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  59. European Medicines Agency. EMEA/CHMP/EWP/431734/2008. Guideline on the clinical investigation of medicinal products for the treatment of attention deficit hyperactivity disorder (ADHD), London, 22 July 2010 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2010/08/WC500095686.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  60. European Medicines Agency. EMA/CHMP/EWP/213056/2010. Addendum to the guideline on antiarrhythmics on atrial fibrillation and atrial flutter, London, 22 July 2010 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2010/09/WC500096802.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  61. European Medicines Agency. CHMP/EWP/566/98 Rev.2/Corr. Guideline on clinical investigation of medicinal products in the treatment of epileptic disorders, London, 22 July 2010 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2010/01/WC500070043.pdf [Accessed 2010 Dec 7]

    Google Scholar 

  62. FDA. Guidances (Drugs): search drugs and vaccines, blood & biologics guidances [online]. Available from URL: http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm [Accessed 2011 Jun 9]

    Google Scholar 

  63. European Medicines Agency. Clinical efficacy and safety guidelines introduction [online]. Available from URL: http://www.ema.europa.eu/htms/human/humanguidelines/efficacy.htm [Accessed 2011 Jun 9]

    Google Scholar 

  64. European Medicines Agency. EMEA/42754/2008: assessment report for Ivemend. Procedure no. (EMEA/H/C/743) [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/000743/WC500037156.pdf [Accessed 2011 Jun 9]

    Google Scholar 

  65. European Medecines Agency. EMA/559664/2010: assessment report for Ivemend. Procedure no. EMEA/H/C/000743/X/00 [online]. Available from URL: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Assessment_Report_-_Variation/human/000743/WC500097052.pdf [Accessed 2011 Jun 9]

    Google Scholar 

  66. Gondek K, Sagnier PP, Gilchrist K, et al. Current status of patient-reported outcomes in industry sponsored oncology clinical trials and product labels. J Clin Oncol 2007 10; 25: 5087–93

    Article  PubMed  Google Scholar 

  67. Rock EP, Kennedy DL, Furness MH, et al. Patient-reported outcomes supporting anticancer product approvals. J Clin Oncol 2007 10; 25: 5094–9

    Article  PubMed  CAS  Google Scholar 

  68. US Department of Health and Human Services, Food and Drug Administration. Qualification process for drug development tools. Fed Regist 2010; 75 (205): 65495–6

    Google Scholar 

  69. Bottomley A, Jones D, Claassens L. 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. Eur J Cancer 2009; 45: 347–53

    Article  PubMed  Google Scholar 

  70. Doward LC, Gnanasakthy A, Baker MG. Patient reported outcomes: looking beyond the label claim. Health Qual Life Outcomes 2010 Aug 20; 8: 89

    Article  PubMed  Google Scholar 

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Acknowledgements

This study was not funded by any agencies external to Mapi Values USA, LLC or Mapi Research Trust. Mapi Values and Mapi Research Trust have developed the PROLabels database. Mapi Research Trust maintains and sells access to the PROLabels database, which is used in this work to facilitate our review of the data on PRO claims. The authors have no conflicts of interest that are directly relevant to this study.

The authors gratefully acknowledge the support of Laure-Lou Perrier, Virginie Vaissier and Mylène Castex, Project Managers at Mapi Research Trust, for their assistance in data collection and analysis.

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Marquis, P., Caron, M., Emery, MP. et al. The Role of Health-Related Quality of Life Data in the Drug Approval Processes in the US and Europe. Pharm Med 25, 147–160 (2011). https://doi.org/10.1007/BF03256856

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