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Evaluation of Therapeutics for Advanced‐Stage Heart Failure and Other Severely‐Debilitating or Life‐Threatening Diseases

Severely‐debilitating or life‐threatening (SDLT) diseases include conditions in which life expectancy is short or quality of life is greatly diminished despite available therapies. As such, the medical context for SDLT diseases is comparable to advanced cancer and the benefit vs. risk assessment and...

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Published in:Clinical pharmacology and therapeutics 2017-08, Vol.102 (2), p.219-227
Main Authors: Prescott, JS, Andrews, PA, Baker, RW, Bogdanffy, MS, Fields, FO, Keller, DA, Lapadula, DM, Mahoney, NM, Paul, DE, Platz, SJ, Reese, DM, Stoch, SA, DeGeorge, JJ
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cited_by cdi_FETCH-LOGICAL-c3170-944089a9edb02446af2fc2fd21ba3e2e59c0e9f4e7be2b7cb32c18a9f2d3a973
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container_title Clinical pharmacology and therapeutics
container_volume 102
creator Prescott, JS
Andrews, PA
Baker, RW
Bogdanffy, MS
Fields, FO
Keller, DA
Lapadula, DM
Mahoney, NM
Paul, DE
Platz, SJ
Reese, DM
Stoch, SA
DeGeorge, JJ
description Severely‐debilitating or life‐threatening (SDLT) diseases include conditions in which life expectancy is short or quality of life is greatly diminished despite available therapies. As such, the medical context for SDLT diseases is comparable to advanced cancer and the benefit vs. risk assessment and development of SDLT disease therapeutics should be similar to that of advanced cancer therapeutics. A streamlined development approach would allow patients with SDLT conditions earlier access to therapeutics and increase the speed of progression through development. In addition, this will likely increase the SDLT disease therapeutic pipeline, directly benefiting patients and reducing the economic and societal burden of SDLT conditions. Using advanced‐stage heart failure (HF) as an example that illustrates the concepts applicable to other SDLT indications, this article proposes a streamlined development paradigm for SDLT disease therapeutics and recommends development of aligned global regulatory guidance.
doi_str_mv 10.1002/cpt.730
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source Wiley-Blackwell Read & Publish Collection
subjects Animals
Communicable Diseases - diagnosis
Communicable Diseases - drug therapy
Communicable Diseases - epidemiology
Disease Progression
Drug Discovery - methods
Drug Discovery - trends
Drug Evaluation - methods
Drug Evaluation - trends
Heart Failure - diagnosis
Heart Failure - drug therapy
Heart Failure - epidemiology
Humans
Neurodegenerative Diseases - diagnosis
Neurodegenerative Diseases - drug therapy
Neurodegenerative Diseases - epidemiology
Severity of Illness Index
title Evaluation of Therapeutics for Advanced‐Stage Heart Failure and Other Severely‐Debilitating or Life‐Threatening Diseases
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