Off-label use of drugs and medical devices: A review of policy implications

Clinical Pharmacology and Therapeutics

Volumn 91, Issue 5, 2012, Pages 920-925

  Stafford, R S ^a  

^a STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AMFEBUTAMONE; ANTIINFECTIVE AGENT; CAPTOPRIL; CLONAZEPAM; ESCITALOPRAM; ESOMEPRAZOLE; GABAPENTIN; METFORMIN; MONTELUKAST; OLANZAPINE; QUETIAPINE; RISPERIDONE; SERTRALINE; VENLAFAXINE; ZIPRASIDONE; ZOLPIDEM;

CORONARY STENT; COST EFFECTIVENESS ANALYSIS; DRUG COST; DRUG EFFICACY; DRUG INDUSTRY; DRUG LABELING; DRUG MARKETING; DRUG SAFETY; EVIDENCE BASED PRACTICE; FOOD AND DRUG ADMINISTRATION; HEALTH CARE COST; HEART VALVE PROSTHESIS; HUMAN; MEDICAL DEVICE; OFF LABEL DRUG USE; POLICY; PRIORITY JOURNAL; PRODUCT SAFETY; REVIEW; RISK BENEFIT ANALYSIS;

DRUG AND NARCOTIC CONTROL; EQUIPMENT AND SUPPLIES; HUMANS; OFF-LABEL USE; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84859976904     PISSN: 00099236     EISSN: 15326535     Source Type: Journal    
DOI: 10.1038/clpt.2012.22     Document Type: Review

References (40)

1. Radley, D.C., Finkelstein, S.N. & Stafford, R.S. Off-label prescribing among office-based physicians. Arch. Intern. Med. 166, 1021-1026 (2006).
2. Ma, J., Lee, K.V. & Stafford, R.S. Depression treatment during outpatient visits by U.S. children and adolescents. J. Adolesc. Health 37, 434-442 (2005).
3. Stafford, RS. Perspective: Regulating off-label drug use: rethinking the role of the FDA. N. Engl. J. Med. 358, 1427-1429 (2008).
4. Chen, D.T., Wynia, M.K., Moloney, R.M. & Alexander, G.C. U.S. physician knowledge of the FDA-approved indications and evidence base for commonly prescribed drugs: results of a national survey. Pharmacoepidemiol. Drug Saf. 18, 1094-1100 (2009).
5. US Food and Drug Administration. Drugs@FDA. 〈http://www.accessdata. fda.gov/Scripts/cder/DrugsatFDA/index.cfm〉. Accessed 3 February 2012.
6. Walton, S.M., Schumock, G.T., Lee, K.V., Alexander, G.C., Meltzer, D. & Stafford, R.S. Prioritizing future research on off-label prescribing: results of a quantitative evaluation. Pharmacotherapy 28, 1443-1452 (2008).
7. Yank, V. et al. Systematic review: benefits and harms of in-hospital use of recombinant factor VIIa for off-label indications. Ann. Intern. Med. 154, 529-540 (2011).
8. Alexander, G.C., Gallagher, S.A., Mascola, A., Moloney, R.M. & Stafford, R.S. Growth and diffusion of antipsychotic medicines, 1994-2009. Pharmacoepidemiol Drug Saf 20, 177-184 (2011).
9. Maher, A.R. et al. Efficacy and comparative effectiveness of atypical antipsychotic medications for off-label uses in adults: a systematic review and meta-analysis. JAMA 306, 1359-1369 (2011).
10. Borchers, A.T., Hagie, F., Keen, C.L. & Gershwin, M.E. The history and contemporary challenges of the US Food and Drug Administration. Clin. Ther. 29, 1-16 (2007).
11. Kesselheim, A.S., Mello, M.M. & Studdert, D.M. Strategies and practices in offlabel marketing of pharmaceuticals: a retrospective analysis of whistleblower complaints. PLoS Med 8, e1000431 (2011).
12. Mello, M.M., Studdert, D.M. & Brennan, T.A. Shifting terrain in the regulation of off-label promotion of pharmaceuticals. N Engl J Med 360, 1557-1566 (2009).
13. US Department of Justice. Eli Lilly and Company Agrees to Pay $1.415 Billion to Resolve Allegations of Off-label Promotion of Zyprexa. 15 January 2009. 〈http://www.usdoj.gov/opa/pr/2009/January/09-civ-038. html〉. Accessed 3 February 2012.
14. US Department of Justice. Pharmaceutical company Pfizer, Inc. to pay $301 million for off-label drug marketing. 2 September 2009. 〈http://www.justice.gov/usao/pae/News/Pr/2009/sep/pfizerrelease.pdf〉. Accessed 3 February 2012.
15. US Department of Justice. Justice Department Announces Nearly $1 Billion Civil and Criminal Resolution with Merck Sharp and Dohme Over Promotion of Vioxx. 22 November 2011. 〈http://www.fda.gov/ICECI/ CriminalInvestigations/ucm280985.htm〉. Accessed 3 February 2012.
16. Edney, A. Drugmaker CEOs May be Targets for U.S. FDA in Off-label Cases, Lawyer Says. Bloomberg News. 14 October 2010. 〈http://www.bloomberg. com/news/2010-10-14/drugmaker-executives-may-become-targets-of-fda-for-off- label-promotions.html〉. Accessed 3 February 2012.
17. Foussias, G. & Remington, G. Antipsychotics and schizophrenia: from efficacy and effectiveness to clinical decision-making. Can. J. Psychiatry. 55, 117-125 (2010).
18. Ray, W.A., Chung, C.P., Murray, K.T., Hall, K. & Stein, C.M. Atypical antipsychotic drugs and the risk of sudden cardiac death. N. Engl. J. Med. 360, 225-235 (2009).
19. Chen, H., Reeves, J.H., Fincham, J.E., Kennedy, W.K., Dorfman, J.H. & Martin, B.C. Off-label use of antidepressant, anticonvulsant, and antipsychotic medications among Georgia Medicaid enrollees in 2001. J. Clin. Psychiatry 67, 972-982 (2006).
20. Beohar, N. et al. Outcomes and complications associated with off-label and untested use of drug-eluting stents. JAMA 297, 1992-2000 (2007).
21. US Food and Drug Administration. Overview of Device Regulation. 〈http:// www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/ Overview〉. Accessed 3 February 2012.
22. Dhruva, S.S., Bero, L.A. & Redberg, R.F. Strength of study evidence examined by the FDA in premarket approval of cardiovascular devices. JAMA 302, 2679-2685 (2009).
23. Kesselheim, A.S. Permitting product liability litigation for FDA-approved drugs and devices promotes patient safety. Clin. Pharmacol. Ther. 87, 645-647 (2010).
24. Bridges, J. & Maisel, W.H. Malfunctions and adverse events associated with off-label use of biliary stents in the peripheral vasculature. Am. J. Ther. 15, 12-18 (2008).
25. A STRAL Investigators, Wheatley, K., Ives, N., & Gray, R. et al. Revascularization versus medical therapy for renal-artery stenosis. N. Engl. J. Med. 361: 1953-1962 (2009).
26. Bax, L. et al. Stent placement in patients with atherosclerotic renal artery stenosis and impaired renal function: a randomized trial. Ann. Intern. Med. 150, 840-848, W150 (2009).
27. Cooper, C.J. et al. Stent revascularization for the prevention of cardiovascular and renal events among patients with renal artery stenosis and systolic hypertension: rationale and design of the CORAL trial. Am. Heart J. 152, 59-66 (2006).
28. US Food and Drug Administration. The Sentinel Initiative: A National Strategy for Monitoring Medical Product Safety 〈http://www.fda.gov/ downloads/Safety/FDAsSentinelInitiative/UCM124701.pdf〉. (2008). Accessed 3 February 2012.
29. Platt, R., Wilson, M., Chan, K.A., Benner, J.S., Marchibroda, J. & McClellan, M. The new Sentinel Network-improving the evidence of medical-product safety. N. Engl. J. Med. 361, 645-647 (2009).
30. A lexander, G.C. & Stafford, R.S. Commentary: Does comparative effectiveness have a comparative advantage? JAMA 301: 2488-2490 (2009).
31. Wilensky, G.R. Consumer-driven health plans: early evidence and potential impact on hospitals. Health Aff. (Millwood). 25, 174-185 (2006).
32. Lindstrom, J.A. Sources of drug information: FDA-approved labeling and other official FDA sources. Dermatol. Ther. 22, 246-256 (2009).
33. Stafford, R.S., Wagner, T.H. & Lavori, P.W. New, but not improved? Incorporating comparative-effectiveness information into FDA labeling. N. Engl. J. Med. 361, 1230-1233 (2009).
34. O'Connor, A.B. Building comparative efficacy and tolerability into the FDA approval process. JAMA 303, 979-980 (2010).
35. Alexander, G.C., O'Connor, A.B., Stafford, R.S. Increasing the societal value of innovation: policy strategies to improve drug development and medication prescribing. Ann. Intern. Med. 154, 833-837 (2011).
36. Mello, M.M. & Messing, N.A. Restrictions on the use of prescribing data for drug promotion. N. Engl. J. Med3651248-12542011
37. Osborne, J.E. Can I tell you the truth? A comparative perspective on regulating off-label scientific and medical information. Yale J. Health Policy Law Ethics 10, 301-356 (2010).
38. Walton, S. et al. Computerized interventions to obtain indication information for inpatient prescriptions: a pilot study in drugs frequently used off-label. App. Clinical Informatics 2, 94-103 (2011).
39. Relman, A.S. Could physicians take the lead in health reform? JAMA 304, 2740-2741 (2010).
40. Curfman, G.D. & Redberg, R.F. Medical devices-balancing regulation and innovation. N. Engl. J. Med. 365, 975-977 (2011).