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Evaluation of Therapeutics for Advanced‐Stage Heart Failure and Other Severely‐Debilitating or Life‐Threatening Diseases
Author(s) -
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
Publication year - 2017
Publication title -
clinical pharmacology and therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.941
H-Index - 188
eISSN - 1532-6535
pISSN - 0009-9236
DOI - 10.1002/cpt.730
Subject(s) - life expectancy , intensive care medicine , medicine , context (archaeology) , disease , quality of life (healthcare) , heart failure , cancer , drug development , risk analysis (engineering) , pharmacology , drug , environmental health , population , paleontology , nursing , biology
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 .