EMEA and the evaluation of health-related quality of life data in the drug regulatory process
- PMID: 15446761
- DOI: 10.1017/s0266462304001138
EMEA and the evaluation of health-related quality of life data in the drug regulatory process
Abstract
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.
Similar articles
-
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 Sep-Oct;8(5):534-48. doi: 10.1111/j.1524-4733.2005.00051.x. Value Health. 2005. PMID: 16176492 Review.
-
Health-related quality of life (HR-QOL) and regulatory issues. An assessment of the European Agency for the Evaluation of Medicinal Products (EMEA) recommendations on the use of HR-QOL measures in drug approval.Pharmacoeconomics. 2001;19(2):187-95. doi: 10.2165/00019053-200119020-00005. Pharmacoeconomics. 2001. PMID: 11284382 Review.
-
[New drugs and European procedures of approval. The European Agency for the Evaluation of Medicinal Products' role].Ugeskr Laeger. 2003 Apr 14;165(16):1648-9. Ugeskr Laeger. 2003. PMID: 12756819 Danish.
-
Clinical trials for registration in the European Union: the EMEA 5-year experience in oncology.Crit Rev Oncol Hematol. 2002 May;42(2):123-35. doi: 10.1016/s1040-8428(02)00009-4. Crit Rev Oncol Hematol. 2002. PMID: 12007970 Review.
-
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 Feb;45(3):347-53. doi: 10.1016/j.ejca.2008.09.032. Epub 2008 Nov 14. Eur J Cancer. 2009. PMID: 19013787 Review.
Cited by
-
Monthly migraine days, tablet utilization, and quality of life associated with Rimegepant - post hoc results from an open label safety study (BHV3000-201).J Headache Pain. 2022 Jan 17;23(1):10. doi: 10.1186/s10194-021-01378-5. J Headache Pain. 2022. PMID: 35038983 Free PMC article. Clinical Trial.
-
Relative efficacy of drugs: an emerging issue between regulatory agencies and third-party payers.Nat Rev Drug Discov. 2010 Apr;9(4):277-91. doi: 10.1038/nrd3079. Epub 2010 Feb 26. Nat Rev Drug Discov. 2010. PMID: 20186141 Review.
-
A promising method for identifying cross-cultural differences in patient perspective: the use of Internet-based focus groups for content validation of new patient reported outcome assessments.Health Qual Life Outcomes. 2006 Sep 22;4:64. doi: 10.1186/1477-7525-4-64. Health Qual Life Outcomes. 2006. PMID: 16995935 Free PMC article.
-
Clearing up the hazy road from bench to bedside: a framework for integrating the fourth hurdle into translational medicine.BMC Health Serv Res. 2008 Sep 24;8:194. doi: 10.1186/1472-6963-8-194. BMC Health Serv Res. 2008. PMID: 18816378 Free PMC article.
Publication types
MeSH terms
Substances
LinkOut - more resources
Medical