Gatekeepers and enablers: How drug regulators respond to a challenging and changing environment by moving toward a proactive attitude

Clinical Pharmacology and Therapeutics

Volumn 93, Issue 5, 2013, Pages 425-432

  Ehmann, F ^a   Papaluca Amati, M ^a   Salmonson, T ^a,b   Posch, M ^c   Vamvakas, S ^a   Hemmings, R ^a,d   Eichler, H G ^a   Schneider, C K ^a,e  

^a EUROPEAN MEDICINES AGENCY   (United Kingdom)

^b Swedish Medicines Agency (MPA)   (Sweden)

^c MEDICAL UNIVERSITY OF VIENNA   (Austria)

^d MEDICINES AND HEALTHCARE PRODUCTS REGULATORY AGENCY   (United Kingdom)

^e Danish Health and Medicines Authority   (Denmark)

Author keywords

[No Author keywords available]

Indexed keywords

ARTICLE; COST BENEFIT ANALYSIS; DRUG CONTROL; DRUG INDUSTRY; DRUG REGULATORY; DRUG RESEARCH; DRUG SURVEILLANCE PROGRAM; EPIGENETICS; GENOMICS; GOOD CLINICAL PRACTICE; HUMAN; LICENSING; METABOLOMICS; PERSONALIZED MEDICINE; PHARMACOGENOMICS; PRIORITY JOURNAL; PRODUCT DEVELOPMENT; PROTEOMICS; PUBLIC-PRIVATE PARTNERSHIP;

DRUG AND NARCOTIC CONTROL; DRUG APPROVAL; DRUG DESIGN; DRUG INDUSTRY; HEALTH SERVICES ACCESSIBILITY; HUMANS; ORGANIZATIONAL INNOVATION; PHARMACEUTICAL PREPARATIONS;

EID: 84876690294     PISSN: 00099236     EISSN: 15326535     Source Type: Journal    
DOI: 10.1038/clpt.2013.14     Document Type: Article

References (45)

1. European Medicines Agency: Annual report 2011, Overview of the Agency's contribution to science, medicines and health in the European Union 〈http://www.ema.europa.eu/docs/en-GB/document-library/Annual-report/2012/ 06/WC500128162.pdf〉.
2. European Medicines Agency. Annual report 2011 shows continuously high level of activities 〈http://www.ema.europa.eu/ema/index.jsp?curl=pages/ news-and-events/news/2012/06/news-detail-001523.jsp&mid=WC0b01ac 058004d5c1〉.
3. Mullard, A. 2011 FDA drug approvals. Nat. Rev. Drug Discov. 11, 91-94 (2012).
4. Ratner, M. Big pharma upheavals cast shadow across biotech sector. Nat. Biotechnol. 30, 119-120 (2012).
5. Scherer, F.M. R&D costs and productivity in biopharmaceuticals. HKS Faculty Research Working Paper Series RWP11-046, John F. Kennedy School of Government, Harvard University (2011).
6. IAGG, WHO, SFGG joint 3rd workshop on 'Promoting access to innovation and clinical research for frail old persons' 〈http://www.iagg.info/iagg-who/ workshops〉.
7. Scannell, J.W., Blanckley, A., Boldon, H. & Warrington, B. Diagnosing the decline in pharmaceutical R&D efciency. Nat. Rev. Drug Discov. 11, 191-200 (2012).
8. Woollett, G.R. Innovation in biotechnology: current and future states. Clin. Pharmacol. Ther. 91, 17-20 (2012).
9. Eichler, H.G., Aronsson, B., Abadie, E. & Salmonson, T. New drug approval success rate in Europe in 2009. Nat. Rev. Drug Discov. 9, 355-356 (2010).
10. Ledford, H. Biosimilar drugs poised to penetrate market. Nature 468, 18-19 (2010)
11. Coté, T., Kelkar, A., Xu, K., Braun, M.M. & Phillips, M.I. Orphan products: an emerging trend in drug approvals. Nat. Rev. Drug Discov. 9, 84 (2010).
12. Committee for Orphan Medicinal Products and the European Medicines Agency Scientifc Secretariat. European regulation on orphan medicinal products: 10 years of experience and future perspectives. Nat. Rev. Drug Discov. 10, 341-349 (2011).
13. Dolgin, E. Personalized investigation. Nat. Med. 16, 953-955 (2010).
14. Maciulaitis, R., D'Apote, L., Buchanan, A., Pioppo, L. & Schneider, C.K. Clinical development of advanced therapy medicinal products in Europe: evidence that regulators must be proactive. Mol. Ther. 20, 479-482 (2012) (doi:10.1038/mt.2012.13).
15. Regnstrom, J. et al. Factors associated with success of market authorisation applications for pharmaceutical drugs submitted to the European Medicines Agency. Eur. J. Clin. Pharmacol. 66, 39-48 (2010).
16. Eichler, H.G. et al. Bridging the efcacy-efectiveness gap: a regulator's perspective on addressing variability of drug response. Nat. Rev. Drug Discov. 10, 495-506 (2011).
17. Scientifc Advice at the European Medicines Agency (EMA) 〈http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/ general-content-000050.jsp&mid=WC0b01ac05800229bb〉.
18. Innovation Task Force (ITF) at the European Medicines Agency (EMA) 〈http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/ general-content-000334.jsp&mid=WC0b01ac05800ba1d9〉.
19. Becker, R.E. & Greig, N.H. Increasing the success rate for Alzheimer's disease drug discovery and development. Expert Opin. Drug Discov. 7, 367-370 (2012).
20. European Medicines Agency. Workshop on clinical-trial data and transparency 〈http://www.ema.europa.eu/ema/index.jsp?curl=pages/news-and- events/events/2012/07/event-detail-000656.jsp&mid=WC0b01 ac058004d5c3〉.
21. Allison, M. Reinventing clinical trials. Nat. Biotechnol. 30, 41-49 (2012).
22. Chiang, A. & Million, R.P. From the analyst's couch: personalized medicine in oncology: next generation. Nat. Rev. Drug Discov. 10, 895-896 (2011).
23. Innovative Medicines Initiative (IMI) 〈http://www.imi.europa.eu/ 〉.
24. Final Conclusions on the pilot joint EMEA/FDA VXDS Experience on Qualifcation of Nephrotoxicity Biomarkers 〈http://www.ema.europa. eu/docs/en-GB/document-library/Regulatory-and-procedural-guideline/2009/10/ WC500004205.pdf〉.
25. Bai, J.P. et al. Translational biomarkers: from preclinical to clinical a report of 2009 AAPS/ACCP Biomarker Workshop. AAPS J. 13, 274-283 (2011). Erratum in: AAPS J. 13, 493 (2011).
26. European Medicines Agency: Qualifcation of novel methodologies for drug development 〈http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/ document-listing/document-listing-000319.jsp&mid=WC0b01a c0580022bb0〉.
27. European Medicines Agency. Small and medium-sized enterprise ofce 〈http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/ general-content-000059.jsp&mid=WC0b01ac05800240cc〉.
28. Interactions between the European Medicines Agency and U.S. Food and Drug Administration, September 2009-September 2010 〈http://www.ema. europa.eu/docs/en-GB/document-library/Report/2011/06/WC500107900.pdf〉.
29. Katsnelson, A. FDA and EMEA pool scientifc advice. Nat. Biotechnol. 22, 1490-1491 (2004).
30. EMA-FDA clusters (regular telephone conferences around specifc topics) 〈http://www.ema.europa.eu/docs/en-GB/document-library/Report/2011/06/ WC500107900.pdf〉.
31. Asia Pacifc Economic Cooperation (APEC); 〈http://www.APEC-rhsc. org〉.
32. Eichler, H.G., Abadie, E., Raine, J.M. & Salmonson, T. Safe drugs and the cost of good intentions. N. Engl. J. Med. 360, 1378-1380 (2009).
33. Breckenridge, A., Woods, K. & Walley, T. Medicines regulation and health technology assessment. Clin. Pharmacol. Ther. 87, 152-154 (2010).
34. Oortwijn, W., Mathijssen, J. & Banta, D. The role of health technology assessment on pharmaceutical reimbursement in selected middle-income countries. Health Policy 95, 174-184 (2010).
35. European Medicines Agency's scientifc guidance documents on biosimilar medicines 〈http://www.ema.europa.eu/ema/index. jsp?curl=pages/regulation/ general/general-content-000408.jsp&mid=WC0 b01ac058002958c〉.
36. Eichler, H.-G. et al. Adaptive licensing: taking the next step in the evolution of drug approval. Clin. Pharmacol. Ther. 91, 426-437 (2012) (doi:10.1038/clpt.2011.345).
37. EMA Beneft-Risk Methodology Project: Development and testing of tools and processes for balancing multiple benefts and risks as an aid to informed regulatory decisions about medicinal products. 〈http://www.ema.europa.eu/ docs/en-GB/document-library/Report/2011/07/WC500109477.pdf〉.
38. European Medicines Agency's beneft-risk methodology project 〈http://www.ema.europa.eu/ema/index.jsp?curl=pages/special-topics/ document-listing/document-listing-000314.jsp&mid= WC0b01ac0580223ed6〉.
39. Ashby, D. A Beneft-Risk Analysis of Using Formal Beneft-Risk Approaches for Decision-Making in Drug Regulation 22 August 2012. 〈http://www.imi- protect.eu/documents/AshbyDISCB33August2012.pdf〉.
40. European Medicines Agency/European Federation of Pharmaceutical Industries and Associations frst workshop on adaptive designs in confrmatory clinical trials. 〈http://www.ema.europa.eu/ema/index.jsp?curl=pages/news- and-events/events/2009/11/event-detail-000056.jsp &mid= WC0b01ac058004d5c3〉.
41. Refection paper on methodological issues in confrmatory clinical trials planned with an adaptive design, EMA 2007. 〈http://www.ema.europa.eu/docs/ en-GB/document-library/Scientifc-guideline/2009/09/WC500003617.pdf〉.
42. Arrowsmith, C.H., Bountra, C., Fish, P.V., Lee, K. & Schapira, M. Epigenetic protein families: a new frontier for drug discovery. Nat. Rev. Drug Discov. 11, 384-400 (2012).
43. Knight, R. et al. Unlocking the potential of metagenomics through replicated experimental design. Nat. Biotechnol. 30, 513-520 (2012).
44. Park, B.K. et al. Managing the challenge of chemically reactive metabolites in drug development. Nat. Rev. Drug Discov. 10, 292-306 (2011).
45. European Medicines Agency/Committee for Advanced Therapies and Medical Devices Notifed Body (EMA/CAT-NB) Collaboration Group 〈http://www.ema. europa.eu/ema/index.jsp?curl=pages/contacts/CAT/people-listing-000086. jsp&mid=WC0b01ac058029021c〉.