Evaluation of Therapeutics for Severely Debilitating or Life-Threatening Diseases or Conditions: Defining Scope to Enable Global Guidance Development.

Autor: Liu M; Drug Safety Research and Development, Pfizer Inc, La Jolla, California, USA., Fields FO; Worldwide Global Regulatory Affairs, Collegeville, Pennsylvania, USA., Prescott JS; Safety Assessment and Laboratory Animal Resources, Merck & Co., Inc., West Point, Pennsylvania, USA., Bello A; Clinical Pharmacology and Pharmacometrics, Bristol-Myers Squibb, Princeton, New Jersey, USA., Bower N; Global Nonclinical Regulatory, Eisai Inc., Woodcliff Lake, New Jersey, USA., Darakjy S; Worldwide Medical and Safety, Pfizer, Inc, New York, New York, USA., Hartke J; Nonclinical Development, Celgene, La Jolla, California, USA., Kadambi V; Quantitative Pharmacology & Drug Safety Evaluation, Blueprint Medicines, Cambridge, Massachusetts, USA., Lapadula D; Preclinical Safety, Novartis Institutes for Biomedical Research, East Hanover, New Jersey, USA., Stoch A; Translational Pharmacology, Merck & Co., Inc, Kenilworth, New Jersey, USA., Derzi M; Drug Safety Research and Development, Pfizer Inc, Cambridge, Massachusetts, USA.
Jazyk: angličtina
Zdroj: Clinical pharmacology and therapeutics [Clin Pharmacol Ther] 2020 Mar; Vol. 107 (3), pp. 514-520. Date of Electronic Publication: 2019 Nov 22.
DOI: 10.1002/cpt.1673
Abstrakt: A significant regulatory gap exists to facilitate global development of therapeutics for nononcology severely debilitating or life-threatening diseases or conditions (SDLTs). In a 2017 publication, a streamlined approach to the development of treatments for SDLTs was proposed to facilitate earlier and continued patient access to new, potentially beneficial therapeutics. 1 However, a major hindrance to broad adoption of this streamlined approach has been the lack of universally accepted, objective criteria to define SDLTs. This article serves to extend the 2017 publication by further addressing the challenge of defining SDLT scope in order to stimulate broader discussion and facilitate development of regional and ultimately international guidelines on the development of therapeutics for SDLTs. Using case examples, we describe key attributes of SDLTs and provide criteria for consideration of an SDLT scope definition.
(© 2019 The Authors Clinical Pharmacology & Therapeutics © 2019 American Society for Clinical Pharmacology and Therapeutics.)
Databáze: MEDLINE
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