Ten common questions (and their answers) about off-label drug use

Mayo Clinic Proceedings

Volumn 87, Issue 10, 2012, Pages 982-990

  Wittich, Christopher M ^a   Burkle, Christopher M ^a   Lanier, William L ^a  

^a MAYO CLINIC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ACETYLCYSTEINE; ACETYLSALICYLIC ACID; ALENDRONIC ACID; AMIODARONE; ANTICONVULSIVE AGENT; ANTIDEPRESSANT AGENT; ATENOLOL; ATORVASTATIN; BETA ADRENERGIC RECEPTOR BLOCKING AGENT; COTRIMOXAZOLE; DABIGATRAN; DEXAMETHASONE; DIPHENHYDRAMINE; DONEPEZIL; DOXORUBICIN; ERYTHROMYCIN; ERYTHROPOIETIN; FUROSEMIDE; INDOMETACIN; ISOFLURANE; LINEZOLID; METOPROLOL; NEUROLEPTIC AGENT; OMEPRAZOLE; PROPOFOL; PROPRANOLOL; RITUXIMAB; SALBUTAMOL; SIMVASTATIN; UNINDEXED DRUG;

ANEMIA; ARTICLE; BACTERIAL ENDOCARDITIS; CHEMOTHERAPY INDUCED EMESIS; CLINICAL PRACTICE; CONTRAST INDUCED NEPHROPATHY; DRUG APPROVAL; DRUG COST; DRUG EFFICACY; DRUG INDICATION; DRUG MANUFACTURE; DRUG USE; DYSPNEA; EPILEPTIC STATE; FOOD AND DRUG ADMINISTRATION; FRONTOTEMPORAL DEMENTIA; HEALTH CARE PERSONNEL; HEART ATRIUM FIBRILLATION; HUMAN; HYPERGLYCEMIA; HYPERKALEMIA; HYPERLIPIDEMIA; IDIOPATHIC THROMBOCYTOPENIC PURPURA; INFORMED CONSENT; INSOMNIA; INTRACRANIAL HYPERTENSION; LARYNGITIS; MALPRACTICE; MEDICAL LITERATURE; MEDICATION ERROR; MIGRAINE; MUCOCUTANEOUS LYMPH NODE SYNDROME; MULTIPLE MYELOMA; NAUSEA; NEGLIGENCE; OFF LABEL DRUG USE; PATENT DUCTUS ARTERIOSUS; PRESCRIPTION; SEIZURE; SINUSITIS; STOMACH PARESIS; SUPRAVENTRICULAR TACHYCARDIA; VENOUS THROMBOEMBOLISM; WALDENSTROEM MACROGLOBULINEMIA; DOCTOR PATIENT RELATION; DRUG LABELING; FEMALE; HEALTH PERSONNEL ATTITUDE; MALE; PATIENT EDUCATION; SAFETY; STATISTICS; UNITED STATES;

ATTITUDE OF HEALTH PERSONNEL; DRUG LABELING; DRUG PRESCRIPTIONS; FEMALE; HUMANS; MALE; OFF-LABEL USE; PATIENT EDUCATION AS TOPIC; PHYSICIAN'S PRACTICE PATTERNS; PHYSICIAN-PATIENT RELATIONS; SAFETY MANAGEMENT; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84871911140     PISSN: 00256196     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.mayocp.2012.04.017     Document Type: Review

References (59)

1. Radley DC, Finkelstein SN, Stafford RS. Off-label prescribing among office-based physicians. Arch Intern Med. 2006;166(9):1021-1026.
2. Qureshi ZP, Liu Y, Sartor O, Chu YH, Bennett CL. Enforcement actions involving Medicaid fraud and abuse, 1996-2009. Arch Intern Med. 2011;171(8):785-787.
3. Kmietowicz Z. Eli Lilly pays record $1.4bn for promoting off-label use of olanzapine. BMJ. 2009;338:b217.
4. Ratner M. Pfizer settles largest ever fraud suit for off-label promotion. Nat Biotechnol. 2009;27(11):961-962.
5. Thomas K, Schmidt MS. Glaxo agrees to pay $3 billion in fraud settlement. New York Times. July 2, 2012. http://www.nytimes. com/2012/07/03/business/glaxosmithkline-agrees-to-pay-3-billionin- fraud-settlement.html?pagewanted-all. Accessed July 6, 2012.
6. Richardson v Miller, 44 SW3d 1 (Tenn Ct App 2000).
7. Roehr B. Abbott pays $1.6bn for promoting off label use of valproic acid. BMJ. 2012;344:e3343.
8. Stafford RS. Regulating off-label drug use: rethinking the role of the FDA. N Engl J Med. 2008;358(14):1427-1429.
9. About FDA: history. US Food and Drug Administration Web site. http://www.fda.gov/AboutFDA/WhatWeDo/History/ default.htm. Accessed April 10, 2012.
10. Lin H-W, Phan K, Lin S-J. Trends in off-label -blocker use: a secondary data analysis. Clin Ther. 2006;28(10):1736-1746. discussion 1710-1731.
11. Shah SS, Hall M, Goodman DM, et al. Off-label drug use in hospitalized children. Arch Pediatr Adolesc Med. 2007;161(3):282-290.
12. [published correction appears in Arch Pediatr Adolesc Med. 2007;161(7):655].
13. Chen H, Reeves JH, Fincham JE, Kennedy WK, Dorfman JH, Martin BC. Off-label use of antidepressant, anticonvulsant, and antipsychotic medications among Georgia Medicaid enrollees in 2001. J Clin Psychiatry. 2006;67(6):972-982.
14. Lat I, Micek S, Janzen J, Cohen H, Olsen K, Haas C. Off-label medication use in adult critical care patients. J Crit Care. 2011; 26(1):89-94.
15. Loder EW, Biondi DM. Off-label prescribing of drugs in specialty headache practice. Headache. 2004;44:636-641.
16. Dworkin RH, O'Connor AB, Audette J, et al. Recommendations for the pharmacological management of neuropathic pain: an overview and literature update. Mayo Clinic Proc. 2010;85(3, suppl):S3-S14.
17. Drug Facts and Comparisons 4.0. http://online.factsand comparisons.com/index.aspx. Accessed January 16, 2012.
18. Rinke ML, Bundy DG, Shore AD, Colantuoni E, Morlock LL, Miller MR. Pediatric antidepressant medication errors in a national error reporting database. J Dev Behav Pediatr. 2010; 31(2):129-136.
19. Friedman WF, Hirschklau MJ, Printz MP, Pitlick PT, Kirkpatrick SE. Pharmacologic closure of patent ductus arteriosus in the premature infant. N Engl J Med. 1976;295(10):526-529.
20. Schram ME, Borgonjen RJ, Bik CMJM, et al. Off-label use of azathioprine in dermatology: a systematic review. Arch Dermatol. 2011;147(4):474-488.
21. Kerns MJ, Graves JE, Smith DI, Heffernan MP. Off-label uses of biologic agents in dermatology: a 2006 update. Semin Cutan Med Surg. 2006;25:226-240.
22. Berthet S, Charpiat B, Mabrut JY. Erythromycin as a prokinetic agent: risk factors. J Visc Surg. 2010;147(2):e13-e18.
23. Bovio JA, Smith SM, Gums JG. Dabigatran etexilate: a novel oral thrombin inhibitor for thromboembolic disease. Ann Pharmacother. 2011;45(5):603-614.
24. Kerchner GA, Tartaglia MC, Boxer A. Abhorring the vacuum: use of Alzheimer's disease medications in frontotemporal dementia. Expert Rev Neurother. 2011;11(5):709-717.
25. Dobecki DA, Schocket SM, Wallace MS. Update on pharmacotherapy guidelines for the treatment of neuropathic pain. Curr Pain Headache Rep. 2006;10(3):185-190.
26. Hoffmann F, Glaeske G, Steuber C. Did the removal of the indication of nocturnal enuresis for intranasal desmopressin change prescribing practice? Pharmacoepidemiol Drug Saf. 2011;20(1):105-109.
27. Shankar V, Churchwell KB, Deshpande JK. Isoflurane therapy for severe refractory status asthmaticus in children. Intensive Care Med. 2006;32(6):927-933.
28. Maher AR, Maglione M, Bagley S, et al. Efficacy and comparative effectiveness of atypical antipsychotic medications for off-label uses in adults: a systematic review and meta-analysis. JAMA. 2011;306(12):1359-1369.
29. Liebowitz MR, Gorman JM, Fyer AJ, Klein DF. Social phobia: a review of a neglected anxiety disorder. Arch Gen Psychiatry. 1985;42:729-736.
30. Feldhaus-Dahir M. Treatment options for female sexual arousal disorder: part II. Urol Nurs. 2010;30(4):247-251.
31. Phan H, Leder M, Fishley M, Moeller M, Nahata M. Off-label and unlicensed medication use and associated adverse drug events in a pediatric emergency department. Pediatr Emerg Care. 2010;26(6):424-430.
32. Institute of Medicine. Addressing the Barriers to Pediatric Drug Development: Workshop Summary. Washington, DC: National Academies Press; 2008.
33. Alexander GC, Gallagher SA, Mascola A, Moloney RM, Stafford RS. Increasing off-label use of antipsychotic medications in the United States, 1995-2008. Pharmacoepidemiol Drug Saf. 2011;20(2):177-184.
34. Stone KJ, Viera AJ, Parman CL. Off-label applications for SSRIs. Am Fam Physician. 2003;68(3):498-504.
35. Lowenstein E. The birth of opioid anesthesia. Anesthesiology. 2004;100:1013-1015.
36. Gan TJ, Meyer T, Apfel CC, et al. Consensus guidelines for managing postoperative nausea and vomiting. Anesth Analg. 2003;97(1):62-71.
37. Hartle A, Malhotra S. The safety of propofol. BMJ. 2009;339:928-929.
38. Helm KA. Protecting public health from outside the physician's office: a century of FDA regulation from drug safety labeling to off-label drug promotion. Fordham Intellectual Property Media Entertainment Law J. 2007;18:117-187.
39. Riley JB, Basilius PA. Physicians' liability for off-label prescriptions. Hematol Oncol News Issues. 2007;May/June:24-27. 37. http://www.mcguirewoods. com/news-resources/publications/ health-care/Off-Label.pdf. Accessed April 10, 2012.
40. Bax Global Inc v Brenneman, Ohio 695 (Ohio Ct App 2007).
41. Wilkes M, Johns M. Informed consent and shared decisionmaking: a requirement to disclose to patients off-label prescriptions. PLoS Med. 2008;5(11):1553-1556.
42. Canterbury v Spence, 464 F2d 772 (DC Cir 1972).
43. Klein v Biscup, 109 Ohio App3d 855 (Ohio Ct App 1996).
44. U.S. adults ambivalent about the risks and benefits of off-label prescription drug use. HarrisInteractive Web site. http://www. harrisinteractive.com/news/printerfriend/index.asp?NewsID- 1126. Accessed April 10, 2012.
45. State Board of Registration for the Healing Arts v McDonagh, 123 SW3d 146 (Mo 2003).
46. Committee on Drugs, American Academy of Pediatrics. Uses of drugs not described in the package insert (off-label uses). Pediatrics. 2002;110(1):181-183.
47. Dressler R, Frader J. Off-label prescribing: a call for heightened professional and government oversight. J Law Med Ethics. 2009;37(3):476-486.
48. "Off-label" and investigational use of marketed drugs, biologics, and medical devices: Information sheet. US Food and Drug Administration Web site. http://www.fda.gov/Regulatory Information/Guidances/ ucm126486.htm. Accessed April 10, 2012.
49. ClinicalTrials.gov: clinical trial phases. US National Library of Medicine Web site. http://www.nlm.nih.gov/services/ctphases. html. Accessed April 10, 2012.
50. US National Institues of Health. ClinicalTrials.gov Web site. http://ClinicalTrials.gov. Accessed April 10, 2012.
51. Zarin DA, Tse T, Williams RJ, Califf RM, Ide NC. The Clinical Trials.gov results database: update and key issues. N Engl J Med. 2011;364(9):852-860.
52. Katz KA. Time to nip "seeding trials" in the bud. Arch Dermatol. 2008;144(3):403-404.
53. Accreditation Council for Continuing Medical Education Web site. http://www.accme.org/. Accessed April 10, 2012.
54. Mello MM, Studdert DM, Brennan TA. Shifting terrain in the regulation of off-label promotion of pharmaceuticals. N Engl J Med. 2009;360(15):1557-1566.
55. Nightingale SL. From the Food and Drug Administration. JAMA. 1998;279(5):346.
56. [published correction appears in JAMA. 1998;279(12):913]
57. Tanne JH. FDA allows drug firms to distribute journal articles about unapproved uses. BMJ. 2009;338:b187.
58. Kesselheim AS, Mello MM, Studdert DM. Strategies and practices in off-label marketing of pharmaceuticals: a retrospective analysis of whistleblower complaints. PLoS Med. 2011;8(4): e1000431.
59. Lavine G. Orphan Drug Act passes quarter-century milestone in fight against rare diseases. Am J Health-Syst Pharm. 2008; 65(13):1205-1210.