These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

148 related articles for article (PubMed ID: 31102646)

  • 1. Premarket safety population size associated with approval of expedited program drugs and orphan drugs.
    Illoh K
    J Clin Epidemiol; 2019 Sep; 113():101-103. PubMed ID: 31102646
    [No Abstract]   [Full Text] [Related]  

  • 2. Marketing authorisation of orphan medicines in Europe from 2000 to 2013.
    Hofer MP; Hedman H; Mavris M; Koenig F; Vetter T; Posch M; Vamvakas S; Regnstrom J; Aarum S
    Drug Discov Today; 2018 Feb; 23(2):424-433. PubMed ID: 29074441
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Survey of Japanese Orphan Drug Program: Factors Related to Successful Marketing Approval.
    Harada K; Toriyabe K; Ono S
    J Clin Pharmacol; 2020 Jan; 60(1):117-124. PubMed ID: 31364772
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Precision Medicines' Impact on Orphan Drug Designation.
    Mueller CM; Rao GR; Miller Needleman KI
    Clin Transl Sci; 2019 Nov; 12(6):633-640. PubMed ID: 31297924
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Regulators adopt more orphan drugs.
    Reardon S
    Nature; 2014 Apr; 508(7494):16-7. PubMed ID: 24695293
    [No Abstract]   [Full Text] [Related]  

  • 6. Beware the increasing cost and number of orphan drugs.
    Silverman E
    Manag Care; 2013 Mar; 22(3):10-4. PubMed ID: 23610800
    [No Abstract]   [Full Text] [Related]  

  • 7. The drug lag and associated factors for orphan anticancer drugs in Japan compared to the United States.
    Nakayama H; Matsumaru N; Tsukamoto K
    Invest New Drugs; 2019 Oct; 37(5):1086-1093. PubMed ID: 29855823
    [TBL] [Abstract][Full Text] [Related]  

  • 8. The impact of the Orphan Drug Act on Food and Drug Administration-approved therapies for rare skin diseases and skin-related cancers.
    Karas L; Lu CY; Agrawal PB; Asgari MM
    J Am Acad Dermatol; 2019 Sep; 81(3):867-877. PubMed ID: 31103566
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Regulatory characteristics and pivotal study design of US Food and Drug Administration approval of drugs for major vs. minor cancer.
    Yamashita K; Kaneko M; Narukawa M
    Eur J Clin Pharmacol; 2019 Sep; 75(9):1193-1200. PubMed ID: 31129731
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Accelerated approval of drugs: ethics versus efficacy.
    Chary KV; Pandian K
    Indian J Med Ethics; 2017; 2(4):244-247. PubMed ID: 28661403
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years.
    Braun MM; Farag-El-Massah S; Xu K; Coté TR
    Nat Rev Drug Discov; 2010 Jul; 9(7):519-22. PubMed ID: 20531273
    [TBL] [Abstract][Full Text] [Related]  

  • 12. The prevalence and cost of unapproved uses of top-selling orphan drugs.
    Kesselheim AS; Myers JA; Solomon DH; Winkelmayer WC; Levin R; Avorn J
    PLoS One; 2012; 7(2):e31894. PubMed ID: 22363762
    [TBL] [Abstract][Full Text] [Related]  

  • 13. What the Orphan Drug Act has done lately for children with rare diseases: a 10-year analysis.
    Thorat C; Xu K; Freeman SN; Bonnel RA; Joseph F; Phillips MI; Imoisili MA
    Pediatrics; 2012 Mar; 129(3):516-21. PubMed ID: 22371464
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Statistical considerations for rare diseases drug development.
    Chow SC; Chang YW
    J Biopharm Stat; 2019; 29(5):874-886. PubMed ID: 31454299
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Five-Year Sales for Newly Marketed Prescription Drugs With and Without Initial Orphan Drug Act Designation.
    Tu SS; Nagar S; Kesselheim AS; Lu Z; Rome BN
    JAMA; 2023 May; 329(18):1607-1608. PubMed ID: 37159041
    [TBL] [Abstract][Full Text] [Related]  

  • 16. A clinical pharmacology-regulatory perspective on the approval of drugs for rare diseases.
    Bashaw ED
    Clin Pharmacol Ther; 2016 Oct; 100(4):327-9. PubMed ID: 27417549
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Patient-Reported Outcomes in Orphan Drug Labels Approved by the US Food and Drug Administration.
    Hong YD; Villalonga-Olives E; Perfetto EM
    Value Health; 2019 Aug; 22(8):925-930. PubMed ID: 31426934
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Therapies for inborn errors of metabolism: what has the orphan drug act delivered?
    Talele SS; Xu K; Pariser AR; Braun MM; Farag-El-Massah S; Phillips MI; Thompson BH; Coté TR
    Pediatrics; 2010 Jul; 126(1):101-6. PubMed ID: 20566615
    [TBL] [Abstract][Full Text] [Related]  

  • 19. The new drug approvals of 1990, 1991, and 1992: trends in drug development.
    Kaitin KI; Manocchia M; Seibring M; Lasagna L
    J Clin Pharmacol; 1994 Feb; 34(2):120-7. PubMed ID: 8163711
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Postmarketing safety of orphan drugs: a longitudinal analysis of the US Food and Drug Administration database between 1999 and 2018.
    Fan M; Chan AYL; Yan VKC; Tong X; Lau LKW; Wan EYF; Tam EYT; Ip P; Lum TY; Wong ICK; Li X
    Orphanet J Rare Dis; 2022 Jan; 17(1):3. PubMed ID: 34983612
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 8.