Cross-sector sponsorship of research in eosinophilic esophagitis: A collaborative model for rational drug development in rare diseases

Journal of Allergy and Clinical Immunology

Volumn 130, Issue 3, 2012, Pages 613-616

  Fiorentino, Robert ^a   Liu, Gumei ^a   Pariser, Anne R ^a   Mulberg, Andrew E ^a  

^a CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

Author keywords

Clinical outcome assessment; eosinophilic esophagitis; Study Endpoints and Labeling Development; the International Gastrointestinal Eosinophil Researchers; US Food and Drug Administration

Indexed keywords

MEPOLIZUMAB;

ARTICLE; CAREGIVER; CLINICAL EFFECTIVENESS; CLINICAL RESEARCH; CORRELATION ANALYSIS; DISEASE ACTIVITY; DRUG RESEARCH; ENDOSCOPIC BIOPSY; ENDOSCOPY; EOSINOPHILIC ESOPHAGITIS; ESOPHAGUS BIOPSY; FOOD AND DRUG ADMINISTRATION; HISTOPATHOLOGY; HUMAN; MEDICAL SOCIETY; ORGANIZATION AND MANAGEMENT; OUTCOME ASSESSMENT; PATHOPHYSIOLOGY; PATIENT MONITORING; PHENOTYPE; PRIORITY JOURNAL; RISK BENEFIT ANALYSIS; SYMPTOM;

COOPERATIVE BEHAVIOR; DRUG DISCOVERY; EOSINOPHILIC ESOPHAGITIS; HUMANS; RARE DISEASES;

EID: 84865687859     PISSN: 00916749     EISSN: 10976825     Source Type: Journal    
DOI: 10.1016/j.jaci.2012.07.011     Document Type: Article

References (20)

1. S.E. Attwood, T.C. Smyrk, T.R. DeMeester, and J.B. Jones Esophageal eosinophilia with dysphagia. A distinct clinicopathologic syndrome Dig Dis Sci 38 1993 109 116
2. T.H. Taft, E. Kern, L. Keefer, D. Burstein, and I. Hirano Qualitative assessment of patient-reported outcomes in adults with eosinophilic esophagitis J Clin Gastroenterol 45 2011 769 774
3. C.A. Liacouras, G.T. Furuta, I. Hirano, D. Atkins, S.E. Attwood, and P.A. Bonis Eosinophilic esophagitis: updated consensus recommendations for children and adults J Allergy Clin Immunol 128 2011 3 20
4. R.J. Noel, P.E. Putnam, and M.E. Rothenberg Eosinophilic esophagitis N Engl J Med 351 2004 940 941
5. J.P. Franciosi, K.A. Hommel, C.W. DeBrosse, A.B. Greenberg, A.J. Greenler, and J.P. Abonia Development of a validated patient-reported symptom metric for pediatric eosinophilic esophagitis: qualitative methods BMC Gastroenterol 11 2011 126
6. Orphan Drug Act. Pub L no. 97-414.96 Stat 2049. Amended in 1984 by Pub L no. 98-551 to add a numeric prevalence threshold to definition of rare diseases.
7. R.C. Griggs, M. Batshaw, M. Dunkle, R. Gopal-Srivastava, E. Kaye, and J. Krischer Clinical research for rare disease: opportunities, challenges, and solutions Mol Genet Metab 96 2009 20 26
8. Institute of Medicine Committee on accelerating rare diseases research and orphan product development Summary, introduction and profile of rare diseases M.J. Field, T.F. Boat, Rare diseases and orphan products: accelerating research and development 2010 National Academies Press Washington (DC) 1 72
9. M. Posada de la Paz, and S.C. Groft Rare diseases epidemiology research. Rare diseases epidemiology. 1st ed. Springer Netherlands Advances in experimental medicine and biology 686 2010 17 39
10. A. Straumann, H.P. Spichtin, L. Grize, K.A. Bucher, C. Beglinger, and H.U. Simon Natural history of primary eosinophilic esophagitis: a follow-up of 30 adult patients for up to 11.5 years Gastroenterology 125 2003 1660 1669
11. P. Hruz, A. Straumann, C. Bussmann, P. Heer, H.U. Simon, and M. Zwahlen Escalating incidence of eosinophilic esophagitis: a 20-year prospective, population-based study in Olten County, Switzerland J Allergy Clin Immunol 128 2011 1349 1350
12. I. Hirano, N. Moy, M.G. Heckman, C.S. Thomas, N. Gonsalves, and S.R. Achem Endoscopic assessment of the oesophageal features of eosinophilic oesophagitis: validation of a novel classification and grading system Gut 2012 [Epub ahead of print]
13. E.S. Dellon, A. Aderoju, J.T. Woosley, R.S. Sandler, and N.J. Shaheen Variability in diagnostic criteria for eosinophilic esophagitis: a systematic review Am J Gastroenterol 102 2007 2300 2313
14. A.H. Assa'ad, S.K. Gupta, M.H. Collins, M. Thomson, A.T. Heath, and D.A. Smith An antibody against IL-5 reduces numbers of esophageal intraepithelial eosinophils in children with eosinophilic esophagitis Gastroenterology 141 2011 1593 1604
15. R. Dohil, R. Newbury, L. Fox, J. Bastian, and S. Aceves Oral viscous budesonide is effective in children with eosinophilic esophagitis in a randomized, placebo-controlled trial Gastroenterology 139 2010 418 429
16. J.M. Spergel, M.E. Rothenberg, M.H. Collins, G.T. Furuta, J.E. Markowitz, and G. Fuchs III Reslizumab in children and adolescents with eosinophilic esophagitis: results of a double-blind, randomized, placebo-controlled trial J Allergy Clin Immunol 129 2012 456 463
17. A. Straumann, S. Conus, P. Grzonka, H. Kita, G. Kephart, and C. Bussmann Anti-interleukin-5 antibody treatment (mepolizumab) in active eosinophilic oesophagitis: a randomised, placebo-controlled, double-blind trial Gut 59 2010 21 30
18. N. Gonsalves, G.Y. Yang, B. Doerfler, S. Ritz, A.M. Ditto, and I. Hirano Elimination diet effectively treats eosinophilic esophagitis in adults; food reintroduction identifies causative factors Gastroenterology 142 2012 1451 1459
19. US Food and Drug Administration. Drug Development Tools (DDT) Qualification Programs. Available at: http://www.fda.gov/Drugs/ DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/default.htm. Accessed April 18, 2012.
20. D.C. Landy, M.A. Brinich, M.E. Colten, E.J. Horn, S.F. Terry, and R.R. Sharp How disease advocacy organizations participate in clinical research: a survey of genetic organizations Genet Med 14 2012 223 228