Information de reference pour ce titreAccession Number: | 00002256-201309000-00009.
|
Author: | Shah, Rashmi R. 1; Roberts, Samantha A. 2; Shah, Devron R. 1
|
Institution: | (1)Rashmi Shah Consultancy Ltd, Gerrards Cross, UK (2)Friends of Cancer Research, Washington, DC, USA
|
Title: | |
Source: | British Journal of Clinical Pharmacology. 76(3):396-411, September 2013.
|
Abstract: | : We compared and determined the reasons for any differences in the review and approval times of tyrosine kinase inhibitors (TKIs) by the US Food and Drug Administration (FDA) and the European EMA/CHMP. Applications for these novel cancer drugs were submitted to them within a mean of 31.2 days of each other, providing a fair basis for comparison. The FDA had granted priority review to 12 TKIs but the EMA/CHMP did not grant the equivalent accelerated assessment to any. The FDA granted accelerated approvals to six (38%) and CHMP granted (the equivalent) conditional approvals to four (29%) of these agents. On average, the review and approval times were 205.3 days in the US compared with 409.6 days in the European Union (EU). The active review times, however, were comparable (225.4 days in the EU and 205.3 days in the US). Since oncology drug development lasts about 7 years, the 20 days difference in review times between the two agencies is inconsequential. Clock stops during review and the time required to issue an approval had added the extra 184.2 days to review time in the EU. We suggest possible solutions to expedite the EU review and approval processes. However, post-marketing emergence of adverse efficacy and safety data on gefitinib and lapatinib, respectively, indicate potential risks of expedited approvals. We challenge the widely prevalent myth that early approval translates into early access or beneficial impact on public health. Both the agencies collaborate closely but conduct independent assessments and make decisions based on distinct legislation, procedures, precedents and societal expectations.
Copyright (C) 2013 Blackwell Publishing Ltd.
|
Author Keywords: | accelerated approval; conditional approval; EMA; FDA; priority review; tyrosine kinase inhibitors.
|
References: | 1 Downing NS, Aminawung JA, Shah ND, Braunstein JB, Krumholz HM, Ross JS. Regulatory review of novel therapeutics - comparison of three regulatory agencies. N Engl J Med, 2012; 366: 2284-2293.
2 Downing NS, Ross JS. Review of novel therapeutics by three regulatory agencies. N Engl J Med, 2012; 367: 1166-1167.
3 Trotta F, Leufkens HG, Schellens JH, Laing R, Tafuri G. Evaluation of oncology drugs at the European Medicines Agency and US Food and Drug Administration: when differences have an impact on clinical practice. J Clin Oncol, 2011; 29: 2266-2272.
4 Roberts SA, Allen JD, Sigal EV. Despite criticism of the FDA review process, new cancer drugs reach patients sooner in the United States than in Europe. Health Aff (Millwood), 2011; 30: 1375-1381.
5 Tufts Center for the Study of Drug Development. U.S. offers patients faster, greater access to cancer drugs than Europe. July/August 2012, Vol. 14(4). Summary. Available at http://csdd.tufts.edu/files/uplo...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
6 Tufts Center for the Study of Drug Development. Onocology drugs get faster approvals than non-oncology drugs in U.S. September/October 2012, Vol. 14(5). Summary. Available at http://csdd.tufts.edu/files/uplo...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
7 European Commission. Procedures for marketing authorisation. EudraLex - Volume 2A - Chapter 6. Decision making procedure for the adoption of Commission Decisions. (November 2005). Available at http://ec.europa.eu/health/files...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
8 Food and Drug Administration. The CDER Handbook. 1998. Available at http://druganddevicelaw.net/CDER...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
9 Food and Drug Administration. Performance report to the President and the Congress for the Prescription Drug User Fee Act. 2005. Available at http://www.fda.gov/downloads/Abo...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
10 Food and Drug Administration. PDUFA reauthorization performance goals and procedures. Available at http://www.fda.gov/downloads/For...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
11 Food and Drug Administration. Guidance for the Public and FDA Staff on Convening Advisory Committee Meetings. Draft dated. August 2008. Available at http://www.fda.gov/downloads/Reg...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
12 Roden DM, Temple R. The US Food and Drug Administration Cardiorenal Advisory Panel and the drug approval process. Circulation, 2005; 111: 1697-1702.
13 European Commission. Pharmaceutical Legislation Notice to applicants and regulatory guidelines medicinal products for human use. EudraLex - Volume 2 - Chapter 1. Procedures for Marketing Authorisation. (November 2005). Available at http://ec.europa.eu/health/files...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
14 European Commission. Pharmaceutical Legislation Notice to applicants and regulatory guidelines medicinal products for human use. EudraLex - Volume 2 - Chapter 4. Centralised Procedure. (April 2006). Available at http://ec.europa.eu/health/files...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
15 European Commission. Pharmaceutical Legislation Notice to applicants and regulatory guidelines medicinal products for human use. EudraLex - Volume 2 - Chapter 2. Mutual recognition procedure and decentralised procedure. (February 2007). Available at http://ec.europa.eu/health/files...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
16 European Commission. REGULATION (EC) No 726/2004 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency. Official Journal of the European Union, 2004; L136: 1-33. Consolidated version. Available at http://ec.europa.eu/health/files...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
17 European Medicines Agency. Annual Report 2011. London: European Medicines Agency, 2012. Available at http://www.ema.europa.eu/docs/en...- ouverture dans une nouvelle fenêtre (last accessed 2 September 2012).
18 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. Official Journal of the European Union, 2006; L/92: 6-9. Available at http://ec.europa.eu/health/files...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
19 European Medicines Agency. PRESS RELEASE: AstraZeneca Withdraws Its Marketing Authorisation Application for Zactima (Vandetanib) (EMEA/698692/2009 Corr). London: European Medicines Agency, 2009; Available at http://www.ema.europa.eu/docs/en...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
20 European Medicines Agency. Questions and Answers on the Withdrawal of the Marketing Authorisation Application for Zactima (Vandetanib) (EMEA/726915/2009). London: European Medicines Agency, 2009. Available at http://www.ema.europa.eu/docs/en...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
21 Netzer T. European Union centralised procedure for marketing authorisation of oncology drugs: an in-depth review of its efficiency. Eur J Cancer, 2006; 42: 446-455.
22 Food and Drug Administration. Gefitinib: administrative documents - part 1. Available at http://www.accessdata.fda.gov/dr...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
23 Food and Drug Administration. Gefitinib: approval letter. Available at http://www.accessdata.fda.gov/dr...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
24 AstraZeneca Global. Press release (11 February 2003): AstraZeneca submits IRESSA (gefitinib, ZD1839) for approval in Europe for advanced non-small cell lung cancer. Available at http://www.astrazeneca.com/Media...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
25 AstraZeneca Global. Press release (4 January 2005): Gefitinib (IRESSA) marketing authorisation application withdrawn in EU. Available at http://www.astrazeneca.com/Media...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
26 AstraZeneca Global. IRESSA (ZD1839, gefitinib) Tablets Oncologic Drugs Advisory Committee (ODAC) meeting briefing document. March 4, 2005. Available at http://www.fda.gov/ohrms/dockets...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
27 European Medicines Agency. Assessment Report for IRESSA (Procedure No EMEA/H/C/001016). Available at http://www.emea.europa.eu/docs/e...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
28 Food and Drug Administration. TYKERB (lapatinib) Label. (13 March 2007). Available at http://www.accessdata.fda.gov/dr...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
29 European Medicines Agency. Assessment Report for TYVERB (Procedure No EMEA/H/C/795). Available at http://www.emea.europa.eu/docs/e...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
30 European Medicines Agency. Summary of product characteristics for TYVERB. Available at http://www.emea.europa.eu/docs/e...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
31 Food and Drug Administration. TYKERB (lapatinib) Label. (7 July 2008). Available at http://www.accessdata.fda.gov/dr...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
32 European Medicines Agency. Tyverb: procedural steps taken and scientific information after the authorisation. Available at http://www.emea.europa.eu/docs/e...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
33 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: 848-853.
34 Arnardottir AH, Haaijer-Ruskamp FM, Straus SM, Eichler HG, de, Graeff PA, Mol PG. Additional safety risk to exceptionally approved drugs in Europe? Br J Clin Pharmacol, 2011; 72: 490-499.
35 Carpenter D, Zucker EJ, Avorn J. Drug-review deadlines and safety problems. N Engl J Med, 2008; 358: 1354-1361.
36 Berlin RJ. Examination of the relationship between oncology drug labeling revision frequency and FDA product categorization. Am J Public Health, 2009; 99: 1693-1698.
37 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: 4398-4405.
38 Food and Drug Administration. Summary Minutes of the Oncologic Drugs Advisory Committee Meeting. February 8, 2011. Available at http://www.fda.gov/downloads/Adv...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
39 Cohen J, Faden L, Predaris S, Young B. Patient access to pharmaceuticals: an international comparison. Eur J Health Econ, 2007; 8: 253-266.
40 Eichler HG, Bloechl-Daum B, Abadie E, Barnett D, Konig F, Pearson S. Relative efficacy of drugs: an emerging issue between regulatory agencies and third-party payers. Nat Rev Drug Discov, 2010; 9: 277-291.
41 Boukus ER, Carrier ER. Americans' access to prescription drugs stabilizes, 2007-2010. Track Rep, 2011; 27: 1-5.
42 Canadian Cancer Society. Cancer drug access for Canadians. September 2009. Available at http://www.cancer.ca/canada-wide...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
43 Zheng B, Poulose A, Fulford M, Holbrook A. A pilot study on cost-related medication nonadherence in Ontario. J Popul Ther Clin Pharmacol, 2012; 19: e239-247.
44 Law MR, Cheng L, Dhalla IA, Heard D, Morgan SG. The effect of cost on adherence to prescription medications in Canada. CMAJ, 2012; 184: 297-302.
45 Vernon JA. Drug research and price controls. Regulation, 2002; 25: 22-25. Available at http://www.cato.org/pubs/regulat...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
46 Barros PP. Pharmaceutical policies in European countries. Adv Health Econ Health Serv Res, 2010; 22: 3-27.
47 Office of Trade Trading. Annexe K: international survey of pharmaceutical pricing and reimbursement schemes. The Pharmaceutical Price Regulation Scheme.' (Product code: OFT885), February 2007 (Crown Copyright 2007). Available at http://www.oft.gov.uk/OFTwork/ma...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
48 Wilking N, Jonsson B, Hogberg D, Justo N. Comparator report on patient access to cancer drugs in Europe. Comparator Reports. 15 February 2009. Available at http://www.comparatorreports.se/- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
49 Mason A, Drummond M, Ramsey S, Campbell J, Raisch D. Comparison of anticancer drug coverage decisions in the United States and United Kingdom: does the evidence support the rhetoric? J Clin Oncol, 2010; 28: 3234-3238.
50 Jonsson B, Bergh J. Hurdles in anticancer drug development from a regulatory perspective. Nat Rev Clin Oncol. 2012; 9: 236-243.
51 Shah DR, Shah RR, Morganroth J. Tyrosine Kinase Inhibitors: their on-target toxicities as potential indicators of efficacy. Drug Saf, 2013; (in press).
52 Niraula S, Seruga B, Ocana A, Shao T, Goldstein R, Tannock IF, Amir E. The price we pay for progress: a meta-analysis of harms of newly approved anticancer drugs. J Clin Oncol, 2012; 30: 3012-3019.
53 Eichler HG, Abadie E, Breckenridge A, Flamion B, Gustafsson LL, Leufkens H, Rowland M, Schneider CK, Bloechl-Daum B. Bridging the efficacy-effectiveness gap: a regulator's perspective on addressing variability of drug response. Nat Rev Drug Discov, 2011; 10: 495-506.
54 Food and Drug Administration. Transcript of the Oncologic Drugs Advisory Committee meeting of 8 November. 2005. Available at http://www.fda.gov/ohrms/dockets...- ouverture dans une nouvelle fenêtre (last accessed 10 September 2012).
55 European Medicines Agency. Presentation by Eichler H-G. Adaptive Licensing: a useful approach for drug licensing in the EU? Available at http://www.ema.europa.eu/docs/en...- ouverture dans une nouvelle fenêtre (last accessed 16 January 2013).
56 Food and Drug Administration. New drug review 2009 updates. Presentation by Jenkins JK. FDA/CMS Summit, 3 December 2009. Available at http://www.fda.gov/downloads/Abo...- ouverture dans une nouvelle fenêtre (last accessed 2 September 2012).
|
Language: | English.
|
Document Type: | Cancer Therapeutics Themed Section.
|
Journal Subset: | Pharmacology.
|
ISSN: | 0306-5251
|
NLM Journal Code: | au9, 7503323
|
DOI Number: | https://dx.doi.org/10.1111/bcp.1...- ouverture dans une nouvelle fenêtre
|
Annotation(s) | |
|
|