Challenges of biomarkers in drug discovery and development

被引:13
|
作者
Goodsaid, Federico [1 ]
机构
[1] Vertex Pharmaceut, Strateg Regulatory Intelligence, Washington, DC 20024 USA
关键词
biomarkers; clinical studies; qualification; DUCHENNE MUSCULAR-DYSTROPHY; GAUCHER-DISEASE; QUALIFICATION;
D O I
10.1517/17460441.2012.679615
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
Biomarker data are essential in the discovery and development of new drugs. However, pathways needed to make sure that biomarker data are accepted by regulatory agencies may be considered an unnecessary burden on the critical path for drug development. There is the need to consider early in discovery and development that these pathways for biomarker acceptance or qualification not only are necessary, but may also enhance the success of novel therapies through regulatory review and clinical use. There also needs to be a focus on the challenge in the application of biomarkers as these approach regulatory evaluation. Regulatory guidance is needed on how a patient population may be defined by the molecular phenotype classification associated with specific mutations in patient genomes. Enzyme replacement therapies have been implicitly approved in the past assuming a molecular phenotype of a defective enzyme, but these and other precedents have not yet been translated into regulatory guidance. A second regulatory pathway for biomarkers is a biomarker qualification process. Biomarker data may be submitted, in the context of a specific NDA, but the biomarker qualification process has added a path through which efficacy and safety biomarkers useful in product development across multiple companies may be qualified through pre-competitive collaboration between these companies.
引用
收藏
页码:457 / 461
页数:5
相关论文
共 50 条
  • [32] Biomarkers in pharmacology and drug discovery
    Anderson, D. C.
    Kodukula, Krishna
    BIOCHEMICAL PHARMACOLOGY, 2014, 87 (01) : 172 - 188
  • [33] Biomarkers: Opportunities and Challenges for Drug Development in the Current Regulatory Landscape
    Gromova, Mariya
    Vaggelas, Annegret
    Dallmann, Gabriele
    Seimetz, Diane
    BIOMARKER INSIGHTS, 2020, 15
  • [34] The challenges of drug discovery and drug development for cognitive aging and Alzheimer's disease
    Fillit, H
    O'Connell, A
    DRUG DISCOVERY AND DEVELOPMENT FOR ALZHEIMER'S DISEASE, 2000, 2002, : 1 - 4
  • [35] New Challenges and Inspired Answers for Anticancer Drug Discovery and Development
    Utsugi, Teruhiro
    JAPANESE JOURNAL OF CLINICAL ONCOLOGY, 2013, 43 (10) : 945 - 953
  • [36] Development of functional protein microarrays for drug discovery: Progress and challenges
    Zhou, FX
    Bonin, J
    Predki, PF
    COMBINATORIAL CHEMISTRY & HIGH THROUGHPUT SCREENING, 2004, 7 (06) : 539 - 546
  • [37] Drug discovery and development in idiopathic pulmonary fibrosis: challenges and opportunities
    Cruwys, Simon
    Hein, Peter
    Humphries, Bob
    Black, Darcey
    DRUG DISCOVERY TODAY, 2020, 25 (12) : 2277 - 2283
  • [38] Metabolic stability for drug discovery and development - Pharmacokinetic and biochemical challenges
    Masimirembwa, CM
    Bredberg, U
    Andersson, TB
    CLINICAL PHARMACOKINETICS, 2003, 42 (06) : 515 - 528
  • [39] Leishmaniasis drug discovery: recent progress and challenges in assay development
    Zulfigar, Bilal
    Shelper, Todd B.
    Avery, Vicky M.
    DRUG DISCOVERY TODAY, 2017, 22 (10) : 1516 - 1531
  • [40] Promise and challenges in drug discovery and development of hybrid anticancer drugs
    Gediya, Lalji K.
    Njar, Vincent C. O.
    EXPERT OPINION ON DRUG DISCOVERY, 2009, 4 (11) : 1099 - 1111