Developing New Immunosuppression for the Next Generation of Transplant Recipients: The Path Forward

American Journal of Transplantation

Volumn 16, Issue 4, 2016, Pages 1094-1101

  Stegall, M D ^a   Morris, R E ^b   Alloway, R R ^c   Mannon, R B ^d  

^a MAYO CLINIC   (United States)

^b STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c UNIVERSITY OF CINCINNATI COLLEGE OF MEDICINE   (United States)

^d University of Alabama at Birmingham   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALEMTUZUMAB; ANTIBODY; AZATHIOPRINE; BIOLOGICAL MARKER; CYCLOSPORIN; DACLIZUMAB; FINGOLIMOD; IMMUNOSUPPRESSIVE AGENT; MYCOPHENOLATE MOFETIL; RAPAMYCIN; TACROLIMUS; TOFACITINIB; TRANSCRIPTOME;

ARTICLE; AUTOIMMUNE DISEASE; COMORBIDITY; DRUG COST; DRUG EFFICACY; DRUG INDUSTRY; DRUG MARKETING; DRUG SAFETY; FOOD AND DRUG ADMINISTRATION; GOVERNMENT REGULATION; GRAFT FAILURE; GRAFT RECIPIENT; GRAFT REJECTION; GRAFT SURVIVAL; HEALTH CARE NEED; HEALTH CARE QUALITY; HEART TRANSPLANTATION; HIGH RISK PATIENT; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; KIDNEY FAILURE; KIDNEY TRANSPLANTATION; MEDICARE; OFF LABEL DRUG USE; PREVENTIVE MEDICINE; PRIORITY JOURNAL; QUALITY OF LIFE; RECURRENT DISEASE; REHABILITATION CARE; RETURN TO WORK; RISK FACTOR; RISK MANAGEMENT; SURVIVAL; VIRUS INFECTION; DRUG DEVELOPMENT; IMMUNOLOGICAL TOLERANCE; IMMUNOLOGY; ORGAN TRANSPLANTATION; PROGNOSIS;

DRUG DISCOVERY; GRAFT REJECTION; HUMANS; IMMUNE TOLERANCE; IMMUNOSUPPRESSION; IMMUNOSUPPRESSIVE AGENTS; ORGAN TRANSPLANTATION; PROGNOSIS; TRANSPLANT RECIPIENTS;

EID: 84953247205     PISSN: 16006135     EISSN: 16006143     Source Type: Journal    
DOI: 10.1111/ajt.13582     Document Type: Article

References (38)

1. U.S. Department of Health and Human Services. 2012 annual data report. [cited 2 Sept 2015 ]. Available from: http://srtr.transplant.hrsa.gov/annual-reports/2012/Default.aspx.
2. Rao PS, et al., Survival on dialysis post-kidney transplant failure: Results from the Scientific Registry of Transplant Recipients. Am J Kidney Dis 2007; 49: 294-300.
3. Surveillance Epidemiology and End Results Cancer Statistics Review 1975-2014 [cited 2015 Apr]. Available from: http://seer.cancer.gov/csr/1975-2012/
4. Kaji AH, Lewis RJ., Noninferiority trials: Is a new treatment almost as effective as another ? JAMA 2015; 313: 2371-2372.
5. Landias P, Daures J-P,. Clinical trials, immunosuppression and renal transplantation: New trends in design and analysis. Pediatr Nephrol 2002; 17: 573-584.
6. Lachenbruch PA, Rosenberg AS, Bonvini E, et al., Biomarkers and surrogate endpoints in renal transplantation: Present status and considerations for clinical trial design. Am J Transplant 2004; 4: 451-457.
7. Hariharan S, Kasiski B, Matas A, et al., Surrogate markers for long-term renal allograft survival. Am J Transplant 2004; 4: 1179-1183.
8. Wiebe C, Gibson IW, Blydt-Hansen TD, et al., Rates and determinants of progression to graft failure in kidney allograft recipients with de novo donor-specific antibody. Am J Tranplant 2015; 15: 2921-2930.
9. Amur S, LaVange L, Zineh I, et al., Biomarker qualification: Toward a multiple stakeholder framework for biomarker development, regulatory acceptance, and utilization. Clin Pharmacol Ther 2015; 98: 34-46.
10. U. S. Food and Drug Administration. Developing products for rare diseases & conditions. [cited 2015 Apr]. Available from: http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/ucm2005525.htm.
11. Howie LJ, Hirsch BR, Abernethy AP., A comparison of FDA and EMA drug approval: Implications for drug development and cost of care. Oncology (Williston Park) 2013; 27: 1195, 1198-1200, 1202 passim.
12. Trotta F, Leufkens HG, Schellens JH, et al., Evaluation of oncology drugs at the European Medicines Agency and US Food and Drug Administration: When differences have an impact on clinical practice. J Clin Oncol 2011; 29: 2266-2272.
13. Begley CG, Ioannidis JPA., Reproducibility in science. Improving the standard of basic and preclinical research. Circ Res 2015; 116: 116-126.
14. Begley CG., Six red flags for suspect work. Nature 2013; 497: 433-434.
15. Begley CG, Ellis LM., Raise standards for preclinical cancer research. Nature 2012; 483: 531-533.
16. Prinz F, Schlange T, Asadullah K., Believe it or not: How much can we rely on published data on potential drug targets ? Nat Rev Drug Disc 2011; 10: 712-713.
17. Boumpas DT, Bertsias GK, Balow JE., A decade of mycophenolate mofetil for lupus nephritis: Is the glass half-empty or half-full ? Ann Rheum Dis 2010; 69: 2059-2061.
18. Heydendael VM, Spuls PI, Opmeer BC, et al., Methotrexate versus cyclosporine in moderate-to-severe chronic plaque psoriasis. N Engl J Med 2003; 349: 658-665.
19. Liu Z, Zhang H, Liu Z, et al., Multitarget therapy for induction treatment of lupus nephritis: A randomized trial. Ann Intern Med 2015; 162: 18-26.
20. Teachy DT, Greiner R, Seif A, et al., Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome. Br J Haematol 2009; 145: 101-106.
21. Kappos L, Radue EW, O'Conner P, et al., A placebo-controlled trial of oral finglolimod in relapsing multiple sclerosis. N Engl J Med. 2010; 362: 387-401.
22. Burmeister GF, Charles-Schoeman C, Wollenhaut J, et al., Tofacitinib (CD-690-550) in combination with methotrexate in patients with active rheumatoid arthritis with an inadequate response to tumor necrosis factor inhibitors: A randomized phase 3 trial. Lancet. 2013; 381: 451-460.
23. Gold R, Giovannoni G, Selmaj K, et al., Daclizumab high-yield process in relapsing-remitting multiple sclerosis (SELECT): A randomized, double-blind, placebo-controlled trial. Lancet 2013; 381: 2167-2175.
24. Cohen JA, Coles AJ, Arnold DL, et al., Alemtuzumab versus interferon beta 1a as first-line treatment for patients with relapsing-remitting multiple sclerosis: A randomized controlled phase 3 trial. Lancet 2012; 380: 1891-1828.
25. Kocher R, Roberts B., The calculus of cures. N Engl J Med 2014; 370: 1473-1475.
26. Roth M, Keeling P, Smart D., Driving personalized medicine: Capturing maximum net present value and optimal return on investment. Personal Med 2010; 7: 103-114.
27. Cavalla D, Minhas R., Does R&D pay Drug Disc Today 2009; 15: 230-234.
28. Kahan BD., Efficacy of sirolimus compared with azathioprine for reduction of acute renal allograft rejection: A randomized multicenter study. The Rapamune US Study Group. Lancet 1999; 356: 194-202.
29. Global observatory on donation & transplantation. Available from: http://www.transplant-observatory.org/.
30. Vincenti F, Dritselis A, Kirkpatrick P., Belatacept. Nat Rev Drug Disc 2011; 10: 655-656.
31. Talaga SR., Medicare immunosuppressive drug coverage for kidney transplant recipients. 2013; Congressional Research Service; 7-5700. Available from: www.crs.gov.
32. GAO report to congressional requesters. End-stage renal disease. C haracteristics of kidney transplant recipients, frequency, of transplant failures, and cost to Medicare. 2007. GAO-07-1117.
33. Cumming JG, Finlay MR, Giordanetto F, et al., Potential strategies for increasing drug-discovery productivity. Fut Med Chem 2014; 6: 515-527.
34. Hay M, Thomas DW, Craighead JI, et al., Clinical development success rates for investigational drugs. Nat Biotechnol 2014; 32: 40-51.
35. DeMasi J., Pharmaceutical R&D performance by firm size: Approval success rates and economic returns. Am J Therap 2014; 21: 26-34.
36. Khanna I,. Drug discovery in pharmaceutical industry productivity challenges and trends. Drug Disc Today 2012; 17: 1088-1102.
37. Scannell JW, Blanckley A, Boldon H, et al., Diagnosing the decline in pharmaceutical R&D efficiency. Nat Rev Drug Disc 2012; 11: 191-200.
38. Knutsen LJS., Drug discovery management, small is still beautiful: Why a number of companies get it wrong. Drug Disc Today 2011; 11: 476-484.