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

Clin Pharmacol Ther. 2017 Aug;102(2):219-227. doi: 10.1002/cpt.730. Epub 2017 Jun 21.


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.

Publication types

  • Review

MeSH terms

  • 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*