Patients as key partners in rare disease drug development

Rare disease drug development could benefit substantially from increased patient engagement and input to enhance understanding of the key aspects of disease impact, ways to measure these impacts and patients' perspectives on the benefit–risk profile of potential therapies. Rare disease drug dev...

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Veröffentlicht in:	Nature reviews. Drug discovery 2016-11, Vol.15 (11), p.731-732
Hauptverfasser:	Bronstein, Max G., Kakkis, Emil D.
Format:	Artikel
Sprache:	eng
Schlagworte:	631/154/109 631/154/155 692/308/153 692/53 692/699 Analysis Biomedical and Life Sciences Biomedicine Biotechnology Cancer Research Clinical Trials as Topic - methods comment Drug Discovery - methods Drug therapy Humans Medicinal Chemistry Molecular Medicine Patient Participation - methods Pharmaceutical research Pharmacist-patient relations Pharmacology/Toxicology Rare diseases Rare Diseases - diagnosis Rare Diseases - drug therapy Social aspects
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Beschreibung
Zusammenfassung:	Rare disease drug development could benefit substantially from increased patient engagement and input to enhance understanding of the key aspects of disease impact, ways to measure these impacts and patients' perspectives on the benefit–risk profile of potential therapies. Rare disease drug development could benefit substantially from increased patient engagement and input to enhance understanding of the key aspects of disease impact, ways to measure these impacts and patients' perspectives on the benefit–risk profile of potential therapies.
ISSN:	1474-1776 1474-1784
DOI:	10.1038/nrd.2016.133