Accessing the accelerated approval pathway for rare disease therapeutics

Improvements must be made to the qualification process for biomarkers as primary endpoints in pivotal clinical studies of treatments for the rarest of diseases.

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Bibliographic Details
Published in:Nature biotechnology 2016-04, Vol.34 (4), p.380-383
Main Authors: Kakkis, Emil D, Kowalcyk, Sara, Bronstein, Max G
Format: Article
Language:English
Subjects:
631/61
692/308/1892
692/700/565
Agriculture
Bioinformatics
Biomarkers
Biomarkers - analysis
Biomedical Engineering/Biotechnology
Biomedicine
Biotechnology
Chemotherapy
Clinical trials
commentary
Disease
Drug Approval - legislation & jurisprudence
Drug Approval - statistics & numerical data
Humans
Life Sciences
Rare diseases
Rare Diseases - diagnosis
Rare Diseases - drug therapy
Therapy
United States
United States Food and Drug Administration
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Description
Summary:Improvements must be made to the qualification process for biomarkers as primary endpoints in pivotal clinical studies of treatments for the rarest of diseases.
ISSN:1087-0156
1546-1696
DOI:10.1038/nbt.3530