Additional safety risk to exceptionally approved drugs in Europe?

British Journal of Clinical Pharmacology

Volumn 72, Issue 3, 2011, Pages 490-499

  Arnardottir, Arna H ^a   Haaijer Ruskamp, Flora M ^a   Straus, Sabine M J ^b,c   Eichler, Hans Georg ^d   de Graeff, Pieter A ^a,b   Mol, Peter G M ^a,b  

^a UNIVERSITY MEDICAL CENTER GRONINGEN   (Netherlands)

^b MEDICINES EVALUATION BOARD   (Netherlands)

^c ERASMUS MEDICAL CENTER   (Netherlands)

^d EUROPEAN MEDICINES AGENCY   (United Kingdom)

Author keywords

Conditional approval; Drug safety; Exceptional circumstances; Licensing of drugs; Regulatory affairs

Indexed keywords

ANTINEOPLASTIC AGENT; ANTIVIRUS AGENT; BIOLOGICAL FACTOR; GASTROINTESTINAL AGENT; ORPHAN DRUG;

ARTICLE; COHORT ANALYSIS; DRUG APPROVAL; DRUG SAFETY; EUROPE; KAPLAN MEIER METHOD; MARKET; PRIORITY JOURNAL; PROBABILITY; RETROSPECTIVE STUDY; RISK ASSESSMENT;

ADVERSE DRUG REACTION REPORTING SYSTEMS; CLINICAL TRIALS AS TOPIC; COHORT STUDIES; DRUG APPROVAL; EUROPE; FOLLOW-UP STUDIES; HUMANS; PRESCRIPTION DRUGS; REFERENCE STANDARDS; RETROSPECTIVE STUDIES; RISK FACTORS; TIME FACTORS;

EID: 79961145344     PISSN: 03065251     EISSN: 13652125     Source Type: Journal    
DOI: 10.1111/j.1365-2125.2011.03995.x     Document Type: Article

References (29)

1. Lazarou J, Pomeranz BH, Corey PN. Incidence of adverse drug reactions in hospitalized patients: a meta-analysis of prospective studies. JAMA 1998; 279: 1200-5.
2. Leendertse AJ, Egberts AC, Stoker LJ, van den Bemt PM, HARM Study Group. Frequency of and risk factors for preventable medication-related hospital admissions in the Netherlands. Arch Intern Med 2008; 168: 1890-6.
3. Califf RM, CERTs Benefit Assessment Workshop Participants. Benefit assessment of therapeutic products: the Centers for Education and Research on Therapeutics. Pharmacoepidemiol Drug Saf 2007; 16: 5-16.
4. Wieringa NF, Peschar JL, Denig P, de Graeff PA, Vos R. Connecting pre-marketing clinical research and medical practice: opinion-based study of core issues and possible changes in drug regulation. Int J Technol Assess Health Care 2003; 19: 202-19.
5. Eichler HG, Pignatti F, Flamion B, Leufkens H, Breckenridge A. Balancing early market access to new drugs with the need for benefit/risk data: a mounting dilemma. Nat Rev Drug Discov 2008; 7: 818-26.
6. Lasser KE, Allen PD, Woolhandler SJ, Himmelstein DU, Wolfe SM, Bor DH. Timing of new black box warnings and withdrawals for prescription medications. JAMA 2002; 287: 2215-20.
7. Mol PG, Straus SM, Piening S, de Vries JT, de Graeff PA, Haaijer-Ruskamp FM. A decade of safety-related regulatory action in the Netherlands: a retrospective analysis of direct healthcare professional communications from 1999 to 2009. Drug Saf 2010; 33: 463-74.
8. International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use. The Clinical Evaluation of QT/QTc Interval Prolongation and Proarrhythmic Potential for Non-Antiarrhythmic Drugs: E14 2005.
9. Darpo B, Nebout T, Sager PT. Clinical evaluation of QT/QTc prolongation and proarrhythmic potential for nonantiarrhythmic drugs: the International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use E14 guideline. J Clin Pharmacol 2006; 46: 498-507.
10. Lehman R, Yudkin JS, Krumholz H. Licensing drugs for diabetes. BMJ 2010; 341: 513-4.
11. Epstein RA. The pharmaceutical industry at risk: how excessive government regulation stifles innovation. Clin Pharmacol Ther 2007; 82: 131-2.
12. European Commission. Commission regulation (EC) No 507/2006 of 29 March 2006 on the conditional marketing authorisation for medicinal products for human use falling within the scope of Regulation (EC) No 726/2004 of the European Parliament and of the Council. 2006.
13. Committee for Medicinal Products for Human Use (CHMP). Guideline on procedures for the granting of a marketing authorisation under exceptional circumstances, pursuant to article 14 (8) of regulation (EC) No 726/2004 (EMEA/357981/2005). 2005.
14. Carpenter D, Zucker EJ, Avorn J. Drug-review deadlines and safety problems. N Engl J Med 2008; 358: 1354-61.
15. Eichler HG, Aronsson B, Abadie E, Salmonson T. New drug approval success rate in Europe in 2009. Nat Rev Drug Discov 2010; 9: 355-6.
16. European Commission. Notice to Applicants. Volume 2A: procedures for marketing authorisation. Chapter 1: marketing authorisation. 2005; Available at (last accessed 25 May 2010).
17. EMA Human Medicines Webpage. Available at (last accessed 29 January 2009).
18. Dutch Medicines Evaluation Board website. Available at (last accessed 1 June 2009).
19. WHO Collaborating Centre for Drug Statistics Methodology website. Available at (last accessed 18 January 2009).
20. European Medicines Agency. Note for guideance on population exposure: the extent of population exposure to assess clinical safety (CPMP/ICH/375/95). 1995.
21. Giezen TJ, Mantel-Teeuwisse AK, Straus SM, Schellekens H, Leufkens HG, Egberts AC. Safety-related regulatory actions for biologicals approved in the United States and the European Union. JAMA 2008; 300: 1887-96.
22. European Medicines Agency. Committee for medicinal products for human use (CHMP). ICH Topic E 2 E Pharmacovigilance Planning (Pvp). Note for guidance on planning pharmacovigilance activities (CPMP/ICH/5716/03). 2005.
23. US Department of Health and Human Services, Food and Drug Administration Task Force on Risk Management. Report to the FDA Commissioner. Managing the risks from medical product use: creating a riskmanagement framework. 1999.
24. Heemstra HE, Giezen TJ, Mantel-Teeuwisse AK, de Vrueh RL, Leufkens HG. Safety-related regulatory actions for orphan drugs in the US and EU: a cohort study. Drug Saf 2010; 33: 127-37.
25. Richey EA, Lyons EA, Nebeker JR, Shankaran V, McKoy JM, Luu TH, Nonzee N, Trifilio S, Sartor O, Benson AB 3rd, Carson KR, Edwards BJ, Gilchrist-Scott D, Kuzel TM, Raisch DW, Tallman MS, West DP, Hirschfeld S, Grillo-Lopez AJ, Bennett CL. Accelerated approval of cancer drugs: improved access to therapeutic breakthroughs or early release of unsafe and ineffective drugs? J Clin Oncol 2009; 27: 4398-405.
26. Boon WP, Moors EH, Meijer A, Schellekens H. Conditional Approval and Approval Under Exceptional Circumstances as regulatory instruments for stimulating responsible drug innovation in Europe. Clin Pharmacol Ther 2010; 88: 848-53.
27. Jones TC. A call to restructure the drug development process: government over-regulation and non-innovative late stage (Phase III) clinical trials are major obstacles to advances in health care. Sci Eng Ethics 2005; 11: 575-87.
28. Schiller LR, Johnson DA. Balancing drug risk and benefit: toward refining the process of FDA decisions affecting patient care. Am J Gastroenterol 2008; 103: 815-9.
29. European Commission. Volume 9A of the Rules Governing Medicinal Products in the European Union - Guidelines on Pharmacovigilance for Medicinal Products for Human Use. 2008.