Drug development in inflammatory bowel disease: The role of the FDA

Inflammatory Bowel Diseases

Volumn 17, Issue 12, 2011, Pages 2585-2593

  Lahiff, Conor ^a   Kane, Sunanda ^b   Moss, Alan C ^a  

^a BETH ISRAEL DEACONESS MEDICAL CENTER   (United States)

^b MAYO CLINIC   (United States)

Author keywords

FDA; inflammatory bowel disease; prescription medications

Indexed keywords

ADALIMUMAB; AMINOSALICYLIC ACID; AZATHIOPRINE; BALSALAZIDE; BUDESONIDE; CERTOLIZUMAB PEGOL; CYCLOSPORIN; INFLIXIMAB; MERCAPTOPURINE; MESALAZINE; METHOTREXATE; NATALIZUMAB; PREDNISOLONE; RIFABUTIN; SALAZOSULFAPYRIDINE; TGN 1412; THALIDOMIDE; TUMOR NECROSIS FACTOR ANTIBODY;

ACUTE LYMPHOCYTIC LEUKEMIA; COLON CROHN DISEASE; CROHN DISEASE; DRUG APPROVAL; DRUG EFFICACY; DRUG MARKETING; DRUG MONITORING; DRUG SAFETY; ENTERITIS; FOOD AND DRUG ADMINISTRATION; HUMAN; KIDNEY GRAFT REJECTION; MULTIPLE ORGAN FAILURE; NONHUMAN; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); POSTMARKETING SURVEILLANCE; PRIORITY JOURNAL; PSORIASIS; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; RHEUMATOID ARTHRITIS; T CELL LYMPHOMA; ULCERATIVE COLITIS;

CLINICAL TRIALS AS TOPIC; DRUG APPROVAL; HUMANS; INFLAMMATORY BOWEL DISEASES; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 80855131608     PISSN: 10780998     EISSN: 15364844     Source Type: Journal    
DOI: 10.1002/ibd.21712     Document Type: Review

References (49)

1. Longobardi T, Jacobs P, Bernstein CN,. Utilization of health care resources by individuals with inflammatory bowel disease in the United States: a profile of time since diagnosis. Am J Gastroenterol. 2004; 99: 650-655. (Pubitemid 38607968)
2. Improving Public Health Through Human Drugs. 2007. US Department of Health and Human Services, Food and Drug Administration.
3. The Pharmaceutical Research and Manufacturers of America (PhRMA). New Medicines Database. 2011.
4. Crohn's Disease - Pipeline Assessment and Market Forecasts to 2017. 2010. GlobalData.
5. Hamburg MA,. Shattuck lecture. Innovation, regulation, and the FDA. N Engl J Med. 2010; 363: 2228-2232.
6. Ballentine, C., Sulfanilimide Disaster. FDA Consumer Magazine. 1981. Office of Public Affairs, Food and Drug Administration.
7. Speirs AL,. Thalidomide and congenital abnormalities. Lancet. 1962; 1: 303-305.
8. Berndt ER, Gottschalk AH, Philipson TJ, et al. Industry funding of the FDA: effects of PDUFA on approval times and withdrawal rates. Nat Rev Drug Discov. 2005; 4: 545-554. (Pubitemid 40975708)
9. Food & Drug Administration. Prescription Drug User Fee Rates for Fiscal Year 2011. Federal Register. 2010; 75.
10. Hennessy S, Strom BL,. PDUFA reauthorization-drug safety's golden moment of opportunity? N Engl J Med. 2007; 356: 1703-1704. (Pubitemid 46658700)
11. Moss AC, Cheifetz AS,. The role of the FDA in drug development in inflammatory bowel disease. In:, Lichtenstein GR, ed. Ulcerative Colitis; The Complete Guide to Medical Management. Thorofare, NJ: Slack; 2011: 33-39.
12. Meadows M,. The FDA's drug review process: ensuring drugs are safe and effective. FDA Consum. 2002; 36: 19-24.
13. (CDER (Center for Drug Evaluation and Research). Immunotoxicology evaluation of investigational new drugs. 2002. US Department of Health and Human Services, Food and Drug Administration.
14. Lesko LJ, Salerno RA, Spear BB, et al. Pharmacogenetics and pharmacogenomics in drug development and regulatory decision making: report of the first FDA-PWG-PhRMA-DruSafe Workshop. J Clin Pharmacol. 2003; 43: 342-358. (Pubitemid 36368777)
15. Weaver JL, Staten D, Swann J, et al. Detection of systemic hypersensitivity to drugs using standard guinea pig assays. Toxicology. 2003; 193: 203-217. (Pubitemid 37352942)
16. Suntharalingam G, Perry MR, Ward S, et al. Cytokine storm in a phase 1 trial of the anti-CD28 monoclonal antibody TGN1412. N Engl J Med. 2006; 355: 1018-1028. (Pubitemid 44343545)
17. Vos JG, Van Loveren H,. Markers for immunotoxic effects in rodents and man. Toxicol Lett. 1995; 82-83: 385-394. (Pubitemid 26057513)
18. Kaitin KI, DiMasi JA,. Pharmaceutical innovation in the 21st century: new drug approvals in the first decade, 2000-2009. Clin Pharmacol Ther. 2011; 89: 183-188.
19. Flieger K,. FDA finds new ways to speed treatment to patients. FDA Consum. 1993; 27: 14-18.
20. Okie S,. Raising the safety bar-the FDA's coxib meeting. N Engl J Med. 2005; 352: 1283-1285. (Pubitemid 40411491)
21. Lasser KE, Allen PD, Woolhandler SJ, et al. Timing of new black box warnings and withdrawals for prescription medications. JAMA. 2002; 287: 2215-2220. (Pubitemid 34441937)
22. Bakke OM, Manocchia M, de Abajo F, et al. Drug safety discontinuations in the United Kingdom, the United States, and Spain from 1974 through 1993: a regulatory perspective. Clin Pharmacol Ther. 1995; 58: 108-117.
23. Keane J, Gershon S, Wise RP, et al. Tuberculosis associated with infliximab, a tumor necrosis factor alpha-neutralizing agent. N Engl J Med. 2001; 345: 1098-1104.
24. Van Assche G, Van Ranst M, Sciot R, et al. Progressive multifocal leukoencephalopathy after natalizumab therapy for Crohn's disease. N Engl J Med. 2005; 353: 362-368. (Pubitemid 41132340)
25. Institute of Medicine. The Future of Drug Safety: Promoting and Protecting the Health of the Public. 2006. Washington DC: National Academic Press.
26. Rosh JR, Gross T, Mamula P, et al. Hepatosplenic T-cell lymphoma in adolescents and young adults with Crohn's disease: a cautionary tale? Inflamm Bowel Dis. 2007; 13: 1024-1030. (Pubitemid 47309975)
27. DiMasi JA, Hansen RW, Grabowski HG,. The price of innovation: new estimates of drug development costs. J Health Econ. 2003; 22: 151-185. (Pubitemid 36279392)
28. Department of Health and Human Services. Orphan Drugs. 2011.
29. Office of Orphan Products Development. Orphan Products Grant Program. 2011.
30. Asacol (mesalamine) Tablets Drug Approval Package Medical Review (Part 1)., 1-25. 1997.
31. Lialda (meslamine) Delayed Release Tablets Drug Approval Package Medical Review. 2007.
32. Cooney RM, Warren BF, Altman DG, et al. Outcome measurement in clinical trials for ulcerative colitis: towards standardisation. Trials. 2007; 8: 17.
33. Fernandez-Becker NQ, Moss AC,. Improving delivery of aminosalicylates in ulcerative colitis: effect on patient outcomes. Drugs. 2008; 68: 1089-1103. (Pubitemid 351693677)
34. Lawlor G, Ahmed A, Moss AC,. Once-daily mesalamine granules for ulcerative colitis. Expert Rev Clin Immunol. 2010; 6: 521-526.
35. Renna S, Orlando A, Mocciaro F, et al. Placebo therapy in Crohn's disease. Eur J Intern Med. 2009; 20: 572-578.
36. Jones J, Loftus EV Jr, Panaccione R, et al. Relationships between disease activity and serum and fecal biomarkers in patients with Crohn's disease. Clin Gastroenterol Hepatol. 2008; 6: 1218-1224.
37. Sandborn WJ, Feagan BG, Stoinov S, et al. Certolizumab pegol for the treatment of Crohn's disease. N Engl J Med. 2007; 357: 228-238. (Pubitemid 47080387)
38. Baert F, Moortgat L, Van Assche G, et al. Mucosal healing predicts sustained clinical remission in patients with early-stage Crohn's disease. Gastroenterology. 2010; 138: 463-468.
39. Kane S,. Endoscopic healing should be a goal for everyone with ulcerative colitis. Inflamm Bowel Dis. 2009; 15: 796-800.
40. Mercaptopurine Tablets 50mg, Package Insert. 2009. Par Pharmaceutical Companies.
41. Kesselheim AS, Solomon DH,. Incentives for drug development-the curious case of colchicine. N Engl J Med. 2010; 362: 2045-2047.
42. Grody WW, Getzug T,. Colchicine's other indication-effect of FDA action. N Engl J Med. 2010; 363: 2267-2268.
43. Unapproved Drugs Initiative. 1-5- 2011.
44. Woodcock J,. Re: Docket Nos. FDA-2010-P-0111 and FDA-2008-P-0507. Hara I, Jonas J. 8-20- 2010
45. Schellekens H,. Follow-on biologics: challenges of the "next generation." Nephrol Dial Transplant. 2005; 20 (suppl 4): iv31-iv36. (Pubitemid 40711823)
46. Jelkmann W,. Biosimilar epoetins and other "follow-on" biologics: update on the European experiences. Am J Hematol. 2010; 85: 771-780.
47. Biologics Price Competition and Innovation Act. 2010.
48. Mullard A,. Hearing shines spotlight on biosimilar controversies. Nat Rev Drug Discov. 2010; 9: 905-906.
49. Franson TR,. Has the FDA amendments act of 2007 impaired drug development? Clin Pharmacol Ther. 2011; 89: 169-171.