Equivalence and noninferiority trials – are they viable alternatives for registration of new drugs? (III)
Tóm tắt
The scientific community's reliance on active-controlled trials is steadily increasing, as widespread agreement emerges concerning the role of these trials as viable alternatives to placebo trials. These trials present substantial challenges with regard to design and interpretation as their complexity increases, and the potential need for larger sample sizes impacts the cost and time variables of the drug development process. The potential efficacy and safety benefits derived from these trials may never be demonstrated by other methods. Active-controlled trials can develop valuable data to inform both prescribers and patients about the dose- and time-dependent actions of any new drug and can contribute to the management and communication of risks associated with the relevant therapeutic products.
Tài liệu tham khảo
Hutton J, Borowitz M, Oleksy I, Luce BR: The pharmaceutical industry and health reform: lessons from Europe. Health Aff (Millwood). 1994, 13: 98-111. 10.1377/hlthaff.13.3.98.
Williams A: The cost-benefit approach. Br Med Bull. 1974, 30: 252-256.
Drummond MF, Stoddard GL, Torrance GW: Methods for the economic evaluation of health care programmes. 1997, Oxford: Oxford University Press
Maynard A: The design of future cost-benefit studies. Am Heart J. 1990, 119 (3 Pt 2): 761-765.
Spurgeon D: NIH promotes use of lower cost drugs for hypertension. BMJ. 2004, 328: 539-10.1136/bmj.328.7439.539.
Major outcomes in high-risk hypertensive patients randomized to angiotensin-converting enzyme inhibitor or calcium channel blocker vs diuretic: The Antihypertensive and Lipid-Lowering Treatment to Prevent Heart Attack Trial (ALLHAT). JAMA. 2002, 288: 2981-2997.
Dong BJ, Hauck WW, Gambertoglio JG, Gee L, White JR, Bubp JL, Greenspan FS: Bioequivalence of generic and brand-name levothyroxine products in the treatment of hypothyroidism. JAMA. 1997, 277: 1205-1213. 10.1001/jama.277.15.1205.
Oles KS, Penry JK, Smith LD, Anderson RL, Dean JC, Riela AR: Therapeutic bioequivalence study of brand name versus generic carbamazepine. Neurology. 1992, 42: 1147-1153.
Oberlin O, Leverger G, Pacquement H, Raquin MA, Chompret A, Habrand JL: Low-dose radiation therapy and reduced chemotherapy in childhood Hodgkin's disease: the experience of the French Society of Pediatric Oncology. J Clin Oncol. 1992, 10: 1602-1608.
Lefebvre JL, Chevalier D, Luboinski B, Kirkpatrick A, Collette L, Sahmoud T: Larynx preservation in pyriform sinus cancer: preliminary results of a European Organization for Research and Treatment of Cancer phase III trial. EORTC Head and Neck Cancer Cooperative Group. J Natl Cancer Inst. 1996, 88: 890-899. 10.1093/jnci/88.13.890.
Taggart SC, Custovic A, Richards DH, Woodcock A: GR106642X: a new, non-ozone depleting propellant for inhalers. BMJ. 1995, 310: 1639-1640.
Edwards SJ, Lilford RJ, Braunholt DA, Jackson JC, Hewison J, Thornton J: Ethical issues in the design and conduct of randomised controlled trials. Health Technol Assess. 1998, 2: 1-132.
Rothman KJ, Michels KB, Baum B: Declaration of Helsinki should be strengthened: for and against. BMJ. 2000, 321: 442-44514. 10.1136/bmj.321.7258.442.
Mathe G, Brienza S: From methodology to ethics and from ethics to methodology. Biomed Pharmacother. 1988, 42: 143-153.
Food and Drug Administration, Department of Health and Human Services: Guidance for industry: E 10: choice of control group and related issues in clinical trials. Rockville, Md. 2001
Ellenberg SS, Temple R: Placebo-controlled trials and active-control-trials in the evaluation of new treatments. Part 2: practical issues and specific cases. Ann Intern Med. 2000, 133: 464-70.
Urquhart J: Demonstrating effectiveness in post-placebo era. Clin Pharmacol Ther. 2001, 70: 115-120. 10.1067/mcp.2001.117285.
Rothman KJ, Michels KB: The continuing unethical use of placebo controls. N Engl J Med. 1994, 331: 394-398. 10.1056/NEJM199408113310611.
Agostoni A: Placebo and EU guidelines [Letter]. Lancet. 1995, 346: 1710-10.1016/S0140-6736(95)92876-6.
Aspinall RL, Goodman NW: Denial of effective treatment and poor quality of clinical information in placebo controlled trials of ondansetron for postoperative nausea and vomiting: a review of published trials. BMJ. 1995, 311: 844-846.
Henry D, Hill S: Comparing treatments [Editorial]. BMJ. 1995, 310: 1279-
Freedman B, Weijer C, Glass KC: Placebo orthodoxy in clinical research. I: Empirical and methodological myths. J Law Med Ethics. 1996, 24: 243-251.
Freedman B, Weijer C, Glass KC: Placebo orthodoxy in clinical research. II: Ethical, legal, and regulatory myths. J Law Med Ethics. 1996, 24: 252-259.
Angell M: The ethics of clinical research in the Third World [Editorial]. N Engl J Med. 1997, 337: 847-849. 10.1056/NEJM199709183371209.
Garattini S, Bertele V: Adjusting Europe's drug regulations to public health needs. Lancet. 2001, 358: 64-67. 10.1016/S0140-6736(00)05258-2.
Jones B, Jarvis P, Lewis JA: Trials to assess equivalence: the importance of rigorous methods. BMJ. 1996, 313: 36-39.
Ware JH, Antman EM: Equivalence trials. N Engl J Med. 1997, 337: 1159-1161. 10.1056/NEJM199710163371610.
Ebbutt AF, Frith L: Practical issues in equivalence trials. Stat Med. 1998, 17: 1691-1701. 10.1002/(SICI)1097-0258(19980815/30)17:15/16<1691::AID-SIM971>3.3.CO;2-A.
Fleming TR: Design and interpretation of equivalence trials. Am Heart J. 2000, 139: S171-S176. 10.1016/S0002-8703(00)90067-X.
Snapinn SM: Noninferiority trials. Curr Control Trials Cardiovasc. 2000, 1: 19-21. 10.1186/CVM-1-1-019.
McAlister FA, Sackett DL: Active-control equivalence trials and antihypertensive agents. Am J Med. 2001, 111: 553-558. 10.1016/S0002-9343(01)00900-7.
Wang S-J, Hung HMJ, Tsong Y: Utility and pitfalls of some statistical methods in active controlled clinical trials. Control Clin Trials. 2002, 23: 15-28. 10.1016/S0197-2456(01)00155-6.
Greene Wl, Concato J, Feinstein AR: Claims of equivalence in medical research: are they supported by the evidence?. Ann Intern Med. 2000, 132: 715-722.
Tramer MR, Reynolds DJ, Moore RA, McQuay HJ: When placebo controlled trials are essential and equivalence trials are inadequate. BMJ. 1998, 317: 875-880.
Max MB: Divergent traditions in analgesic clinical trials. Clin Pharmacol Ther. 1994, 56: 237-241.
Yusuf S, Peto R, Lewis J, Collins R, Sleight P: Beta blockade during and after myocardial infarction: an overview of the randomized trials. Prog Cardiovasc Dis. 1985, 27: 335-71.
Ernst E, Resch KL: Concept of true and perceived placebo effects. BMJ. 1995, 311: 551-553.
Siegel JP: Equivalence and noninferiority trials. Am Heart J. 2000, 139: S166-70. 10.1016/S0002-8703(00)90066-8.
Alderson P: Absence of evidence is not evidence of absence. BMJ. 2004, 328: 476-477. 10.1136/bmj.328.7438.476.
Djulbegovic B, Clarke M: Scientific and ethical issues in equivalence trials. JAMA. 2001, 285: 1206-1208. 10.1001/jama.285.9.1206.
Altman DG, Bland JM: Absence of evidence is not evidence of absence. BMJ. 1995, 311: 485-
Probstfield JL: The clinical trial prerandomization compliance (adherence) screen. In: Patient compliance in medical practice and clinical trials. Edited by: Cramer JA, Spilker B. 1991, New York: Raven Press, 323-334.
Knipschild P, Leffers P, Feinstein AR: The qualification period. J Clin Epidemiol. 1991, 44: 461-464. 10.1016/0895-4356(91)90208-Q.
Urquhart J, de Klerk E: Contending paradigms for the interpretation of data on patient compliance with therapeutic drug regimens. Stat Med. 1998, 17: 251-267. 10.1002/(SICI)1097-0258(19980215)17:3<251::AID-SIM762>3.3.CO;2-1.
Vrijens B, Geotghebeur E: The impact of compliance in pharmacokinetic studies. Stat Methods Med Res. 1999, 8: 247-262. 10.1191/096228099669221405.
Paterson DL, Swindells S, Mohr J, Brester M, Vergis EN, Squier C: Adherence to protease inhibitor therapy and outcomes in patients with HIV infection. Ann Intern Med. 2000, 133: 21-30.
Burt VL, Whelton P, Roccella EJ, Brown C, Cutler JA, Higgins M: Prevalence of hypertension in the US adult population. Results from the Third National Health and Nutrition Examination Survey, 1988–1991. Hypertension. 1995, 25: 305-313.
Walley T, Duggan AK, HAycox AR, Noziol CJ: Treatment for newly diagnosed hypertension: patterns of prescribing and antihypertensive effectiveness in the UK. J R Soc Med. 2003, 96: 525-531. 10.1258/jrsm.96.11.525.
Rand CS: I took the medicine like you told me, doctor: self-report of adherence to medical regimens. In The science of self-report: implications for research and practice. Edited by: Stone AA, Turkkan JS, Bachrach CA, Jobe JB, Kurtzman HS, Cain VS. 2000, Lawrence Erlbaum Associates: Mahwah, NJ
Vaur L, VAisse B, Genes N, Elkik F, Legrand C, Poggi L: Use of electronic pill boxes to assess risk of poor treatment compliance: results of a large-scale trial. Am J Hypertens. 1999, 12 (4): 374-380.
Burke LE: Electronic measurement. In: Compliance in healthcare and research. Edited by: Burke LE, Ockene IS. 2001, Armonk, NY: Futura Publishing, 117-138. American Heart Association Monograph Series
Pledger G, Hall DB: Active control equivalence studies: do they address the efficacy issue?. In: Statistical Issues in Drug Research and Development. Edited by: Peace KE. 1990, New York: Marcel Dekker, 226-238.
Glaser S, Steinbach M, Opitz C, Wruck U, Kleber FX: Torsades de pointes caused by Mibefradil. Eur J Heart Fail. 2001, 3: 627-630. 10.1016/S1388-9842(01)00159-3.
Weir MR, Sperling RS, Reicin A, Gertz BJ: Selective COX-2 inhibition and cardiovascular effects: a review of the rofecoxib development program. Am Heart J. 2003, 146 (4): 591-604. 10.1016/S0002-8703(03)00398-3.
Konstam MA: Matters of the heart: Assessing the cardiovascular safety of new drugs. Am Heart J. 2003, 146: 561-562. 10.1016/S0002-8703(03)00400-9.
Biostatistical methodology in clinical trials in applications for marketing authorizations for medicinal products. CPMP Working Party on Efficacy of Medicinal Products Note for guidance III/3630/92-EN. Stat Med. 1995, 14: 1658-1682.
Committee for Proprietary Medicinal Products: Points to consider on switching between superiority and non-inferiority. Br J Clin Pharmacol. 2001, 52: 223-228. 10.1046/j.1365-2125.2001.01397-3.x.
Gomberg-Maitland M, Frison L, Halperin JL: Active-control clinical trials to establish equivalence or noninferiority: methodological and statistical concepts linked to quality. Am Heart J. 2003, 146: 398-340. 10.1016/S0002-8703(03)00324-7.
