BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

175 related articles for article (PubMed ID: 34247834)

  • 1. Using real world data to support regulatory approval of drugs in rare diseases: A review of opportunities, limitations & a case example.
    Gross AM
    Curr Probl Cancer; 2021 Aug; 45(4):100769. PubMed ID: 34247834
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Use of real-world evidence in regulatory decisions for rare diseases in the United States-Current status and future directions.
    Wu J; Wang C; Toh S; Pisa FE; Bauer L
    Pharmacoepidemiol Drug Saf; 2020 Oct; 29(10):1213-1218. PubMed ID: 32003065
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Innovative design and analysis for rare disease drug development.
    Chow SC; Huang Z
    J Biopharm Stat; 2020 May; 30(3):537-549. PubMed ID: 32065047
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Raising orphans: how clinical development programs of drugs for rare and common diseases are different.
    Orfali M; Feldman L; Bhattacharjee V; Harkins P; Kadam S; Lo C; Ravi M; Shringarpure DT; Mardekian J; Cassino C; Coté T
    Clin Pharmacol Ther; 2012 Aug; 92(2):262-4. PubMed ID: 22739137
    [TBL] [Abstract][Full Text] [Related]  

  • 5. The US Food and Drug Administration's expedited approval programs: Evidentiary standards, regulatory trade-offs, and potential improvements.
    Wallach JD; Ross JS; Naci H
    Clin Trials; 2018 Jun; 15(3):219-229. PubMed ID: 29871509
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Use of Real-World Evidence to Support FDA Approval of Oncology Drugs.
    Feinberg BA; Gajra A; Zettler ME; Phillips TD; Phillips EG; Kish JK
    Value Health; 2020 Oct; 23(10):1358-1365. PubMed ID: 33032780
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Real-world evidence-What is it and does it matter for approval of drugs?
    Eaglstein WH
    J Am Acad Dermatol; 2018 Aug; 79(2):390-391. PubMed ID: 29410344
    [No Abstract]   [Full Text] [Related]  

  • 8. Characteristics of non-randomised studies using comparisons with external controls submitted for regulatory approval in the USA and Europe: a systematic review.
    Goring S; Taylor A; Müller K; Li TJJ; Korol EE; Levy AR; Freemantle N
    BMJ Open; 2019 Feb; 9(2):e024895. PubMed ID: 30819708
    [TBL] [Abstract][Full Text] [Related]  

  • 9. The needs of the few.
    Nature; 2010 Jul; 466(7303):160. PubMed ID: 20613796
    [No Abstract]   [Full Text] [Related]  

  • 10. The Use of External Controls in FDA Regulatory Decision Making.
    Jahanshahi M; Gregg K; Davis G; Ndu A; Miller V; Vockley J; Ollivier C; Franolic T; Sakai S
    Ther Innov Regul Sci; 2021 Sep; 55(5):1019-1035. PubMed ID: 34014439
    [TBL] [Abstract][Full Text] [Related]  

  • 11. 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]  

  • 12. Post-approval Studies for Rare Disease Treatments and Orphan Drugs.
    Maier WC; Christensen RA; Anderson P
    Adv Exp Med Biol; 2017; 1031():197-205. PubMed ID: 29214573
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Protein replacement therapies for rare diseases: a breeze for regulatory approval?
    Gorzelany JA; de Souza MP
    Sci Transl Med; 2013 Mar; 5(178):178fs10. PubMed ID: 23536010
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Real-world evidence to support regulatory decision-making for medicines: Considerations for external control arms.
    Burcu M; Dreyer NA; Franklin JM; Blum MD; Critchlow CW; Perfetto EM; Zhou W
    Pharmacoepidemiol Drug Saf; 2020 Oct; 29(10):1228-1235. PubMed ID: 32162381
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Using Real-World Data to Extrapolate Evidence From Randomized Controlled Trials.
    Wang SV; Schneeweiss S; Gagne JJ; Evers T; Gerlinger C; Desai R; Najafzadeh M
    Clin Pharmacol Ther; 2019 May; 105(5):1156-1163. PubMed ID: 30107034
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Clinical Trial Evidence Supporting US Food and Drug Administration Approval of Novel Cancer Therapies Between 2000 and 2016.
    Ladanie A; Schmitt AM; Speich B; Naudet F; Agarwal A; Pereira TV; Sclafani F; Herbrand AK; Briel M; Martin-Liberal J; Schmid T; Ewald H; Ioannidis JPA; Bucher HC; Kasenda B; Hemkens LG
    JAMA Netw Open; 2020 Nov; 3(11):e2024406. PubMed ID: 33170262
    [TBL] [Abstract][Full Text] [Related]  

  • 17. A personal perspective of orphan drug development for rare diseases: A golden opportunity or an unsustainable future?
    Oo C; Rusch LM
    J Clin Pharmacol; 2016 Mar; 56(3):257-9. PubMed ID: 26211513
    [No Abstract]   [Full Text] [Related]  

  • 18. Trial designs using real-world data: The changing landscape of the regulatory approval process.
    Baumfeld Andre E; Reynolds R; Caubel P; Azoulay L; Dreyer NA
    Pharmacoepidemiol Drug Saf; 2020 Oct; 29(10):1201-1212. PubMed ID: 31823482
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Feasibility of Using Real-world Data to Emulate Postapproval Confirmatory Clinical Trials of Therapeutic Agents Granted US Food and Drug Administration Accelerated Approval.
    Wallach JD; Zhang AD; Skydel JJ; Bartlett VL; Dhruva SS; Shah ND; Ross JS
    JAMA Netw Open; 2021 Nov; 4(11):e2133667. PubMed ID: 34751763
    [TBL] [Abstract][Full Text] [Related]  

  • 20. A note on breast cancer trials with pCR-based accelerated approval.
    Xia Y; Cui L; Yang B
    J Biopharm Stat; 2014; 24(5):1102-14. PubMed ID: 24926729
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 9.