메뉴 건너뛰기




Volumn 17, Issue 15-16, 2012, Pages 898-904

Characteristics of rare disease marketing applications associated with FDA product approvals 2006-2010

Author keywords

[No Author keywords available]

Indexed keywords

ADVANCED CANCER; DRUG MARKETING; FOOD AND DRUG ADMINISTRATION; GASTROINTESTINAL STROMAL TUMOR; HUMAN; KIDNEY CARCINOMA; MULTIVARIATE LOGISTIC REGRESSION ANALYSIS; MYELOID LEUKEMIA; POSTMARKETING SURVEILLANCE; PRODUCT DEVELOPMENT; PRODUCT SAFETY; RARE DISEASE; REVIEW;

EID: 84864628576     PISSN: 13596446     EISSN: 18785832     Source Type: Journal    
DOI: 10.1016/j.drudis.2012.04.011     Document Type: Review
Times cited : (16)

References (33)
  • 3
    • 57649109461 scopus 로고    scopus 로고
    • For the rare diseases clinical research network. Clinical research for rare disease: Opportunities, challenges, and solutions
    • R.C. Griggs For the rare diseases clinical research network. Clinical research for rare disease: opportunities, challenges, and solutions Mol. Genet. Metab. 96 2009 20 26
    • (2009) Mol. Genet. Metab. , vol.96 , pp. 20-26
    • Griggs, R.C.1
  • 4
    • 77954243603 scopus 로고    scopus 로고
    • Emergence of orphan drugs in the United States: A quantitative assessment of the first 25 years
    • M.M. Braun Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years Nat. Rev. Drug Discov. 9 2010 519 522
    • (2010) Nat. Rev. Drug Discov. , vol.9 , pp. 519-522
    • Braun, M.M.1
  • 7
    • 84872428219 scopus 로고    scopus 로고
    • US Food and Drug Administration (Accessed, 25 April, 2012, at:)
    • US Food and Drug Administration (2011). Search orphan drug designation and approvals. (Accessed, 25 April, 2012, at: http://www.accessdata.fda.gov/ scripts/opdlisting/oopd/index.cfm)
    • (2011) Search Orphan Drug Designation and Approvals
  • 8
    • 32044463568 scopus 로고    scopus 로고
    • Adopting orphan drugs - Two dozen years of treating rare diseases
    • DOI 10.1056/NEJMp058317
    • M.E. Haffner Adopting orphan drugs - two dozen years of treating rare diseases N. Engl. J. Med. 354 2006 445 447 (Pubitemid 43200295)
    • (2006) New England Journal of Medicine , vol.354 , Issue.5 , pp. 445-447
    • Haffner, M.E.1
  • 9
    • 79958036418 scopus 로고    scopus 로고
    • Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer
    • A.S. Kesselheim Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer JAMA 305 2011 2320 2326
    • (2011) JAMA , vol.305 , pp. 2320-2326
    • Kesselheim, A.S.1
  • 10
    • 70149120097 scopus 로고    scopus 로고
    • Pivotal studies of orphan drugs approved for neurological diseases
    • J. Mitsumoto Pivotal studies of orphan drugs approved for neurological diseases Ann. Neurol. 66 2009 184 190
    • (2009) Ann. Neurol. , vol.66 , pp. 184-190
    • Mitsumoto, J.1
  • 13
    • 84859313068 scopus 로고    scopus 로고
    • Regulatory framework for drugs for rare diseases
    • Institute Of Medicine Committee On Accelerating Rare Diseases Research And Orphan Product Development Board On Health Sciences Policy M.J. Field, T.F. Boat, The National Academies Press
    • Institute of Medicine Committee on Accelerating Rare Diseases Research and Orphan Product Development, Board on Health Sciences Policy Regulatory framework for drugs for rare diseases M.J. Field, T.F. Boat, Rare Diseases and Orphan Products: Accelerating Research and Development, 2010 The National Academies Press 73 110
    • (2010) Rare Diseases and Orphan Products: Accelerating Research and Development , pp. 73-110
  • 14
    • 84872428765 scopus 로고    scopus 로고
    • Federal Food, Drug, and Cosmetic Act (FD&C Act), Section 505(b) [21 USC §355] As amended through December 31
    • Federal Food, Drug, and Cosmetic Act (FD&C Act), Section 505(b) [21 USC §355]. New Drugs. As amended through December 31, 2004
    • (2004) New Drugs
  • 15
    • 84872426975 scopus 로고    scopus 로고
    • US Food and Drug Administration July 9 (Accessed, 25 April, 2012, at:)
    • US Food and Drug Administration. News and Events, July 9, 2008. (Accessed, 25 April, 2012, at: http://www.fda.gov/NewsEvents/Newsroom/ PressAnnouncements/2008/ucm116920.htm)
    • (2008) News and Events
  • 16
    • 84872427143 scopus 로고    scopus 로고
    • US Food and Drug Administration (Accessed, 25 April, 2012, at:)
    • US Food and Drug Administration. Complete Response Letter Final Rule, July 9, 2008. (Accessed, 25 April, 2012, at: http://www.fda.gov/Drugs/ GuidanceComplianceRegulatoryInformation/LawsActsandRules/ucm084138.htm)
    • (2008) Complete Response Letter Final Rule, July 9
  • 18
    • 84872428435 scopus 로고    scopus 로고
    • 21968 renal cell cancer (RCC). Search US Food and Drug Administration Drugs@FDA. (Accessed, 25 April, 2012, at: ['sunitinib'])
    • Dagher, R. and Justice, R. (2006) Summary review of NDA, 21938 gastrointestinal stromal tumors (GIST), 21968 renal cell cancer (RCC). Search US Food and Drug Administration Drugs@FDA. (Accessed, 25 April, 2012, at: http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm ['sunitinib'])
    • (2006) Summary Review of NDA, 21938 Gastrointestinal Stromal Tumors (GIST)
    • Dagher, R.1    Justice, R.2
  • 19
    • 78651361355 scopus 로고    scopus 로고
    • Characteristics of orphan drug applications that fail to achieve marketing approval in the USA
    • H.E. Heemstra Characteristics of orphan drug applications that fail to achieve marketing approval in the USA Drug Discov. Today 16 2011 73 80
    • (2011) Drug Discov. Today , vol.16 , pp. 73-80
    • Heemstra, H.E.1
  • 22
    • 41049085160 scopus 로고    scopus 로고
    • Predictors of orphan drug approval in the European Union
    • H.E. Heemstra Predictors of orphan drug approval in the European Union Eur. J. Clin. Pharmacol. 64 2008 545 552
    • (2008) Eur. J. Clin. Pharmacol. , vol.64 , pp. 545-552
    • Heemstra, H.E.1
  • 23
    • 77954359837 scopus 로고    scopus 로고
    • Therapies for inborn errors of metabolism: What has the Orphan Drug Act delivered?
    • S.S. Talele Therapies for inborn errors of metabolism: what has the Orphan Drug Act delivered? Pediatrics 126 2010 101 106
    • (2010) Pediatrics , vol.126 , pp. 101-106
    • Talele, S.S.1
  • 24
    • 84859266035 scopus 로고    scopus 로고
    • Determinants for successful marketing authorization of orphan medicinal products in the ER
    • M. Putzeist Determinants for successful marketing authorization of orphan medicinal products in the ER Drug Discov. Today 17 2012 352 358
    • (2012) Drug Discov. Today , vol.17 , pp. 352-358
    • Putzeist, M.1
  • 25
    • 76949104143 scopus 로고    scopus 로고
    • Factors associated with success of market authorization applications for pharmaceutical drugs submitted to the European Medicines Agency
    • J. Regnstrom Factors associated with success of market authorization applications for pharmaceutical drugs submitted to the European Medicines Agency Eur. J. Clin. Pharmacol. 66 2010 39 48
    • (2010) Eur. J. Clin. Pharmacol. , vol.66 , pp. 39-48
    • Regnstrom, J.1
  • 26
    • 84859326107 scopus 로고    scopus 로고
    • Rare diseases and orphan drugs
    • I. Melnikova Rare diseases and orphan drugs Nat. Rev. Drug Discov. 11 2012 267 268
    • (2012) Nat. Rev. Drug Discov. , vol.11 , pp. 267-268
    • Melnikova, I.1
  • 27
    • 84872428703 scopus 로고
    • US Food and Drug Administration (Accessed, 25 April, 2012, at:)
    • US Food and Drug Administration (1992). Prescription Drug User Fee Act (PDUFA). Standard Review and Priority Review. (Accessed, 25 April, 2012, at: http://www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocates/ SpeedingAccesstoImportantNewTherapies/ucm128291.htm)
    • (1992) Prescription Drug User Fee Act (PDUFA). Standard Review and Priority Review
  • 28
    • 84872430288 scopus 로고    scopus 로고
    • FDA, CDER Manual of Policies and Procedures (MAPP) 6020.3 July 16 (Accessed, 25 April, 2012, at:)
    • FDA, CDER Manual of Policies and Procedures (MAPP) 6020.3, Priority Review, July 16, 2007. (Accessed, 25 April, 2012, at: http://www.fda.gov/ downloads/AboutFDA/CentersOffices/CDER/ManualofPoliciesProcedures/ucm082000.pdf)
    • (2007) Priority Review
  • 29
    • 79151485604 scopus 로고    scopus 로고
    • Regulatory scientific advice in drug development: Does company size make a difference?
    • M. Putzeist Regulatory scientific advice in drug development: does company size make a difference? Eur. J. Clin. Pharmacol. 67 2011 157 164
    • (2011) Eur. J. Clin. Pharmacol. , vol.67 , pp. 157-164
    • Putzeist, M.1
  • 30
    • 33745172773 scopus 로고    scopus 로고
    • A journey of hope: Lessons learned from studies on rare diseases and orphan drugs
    • DOI 10.1111/j.1365-2796.2006.01666.x
    • M. Wastfelt A journey of hope: lessons learned from studies on rare diseases and orphan drugs J. Intern. Med. 260 2006 1 10 (Pubitemid 43894064)
    • (2006) Journal of Internal Medicine , vol.260 , Issue.1 , pp. 1-10
    • Wastfelt, M.1    Fadeel, B.2    Henter, J.-I.3
  • 32
    • 79851510629 scopus 로고    scopus 로고
    • Research challenges in central nervous system manifestations of inborn errors of metabolism
    • P.I. Dickson Research challenges in central nervous system manifestations of inborn errors of metabolism Mol. Genet. Metab. 102 2010 326 338
    • (2010) Mol. Genet. Metab. , vol.102 , pp. 326-338
    • Dickson, P.I.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.