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

Abstract

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.

Are Regulators Talking to Each Other Across Borders?

“…I believe these reflections on regulatory science can be condensed into a straightforward message: It is all about the patient . In the drug development arena, there is always a temptation to shift the focus toward local regulatory processes, rules, and regulations, and there is a risk that we forget about the fact that collaborative innovative regulatory science is a key enabler of bringing life‐changing medicines to patients . Sir Alasdair Breckenridge, who passed away in December last year, was a founder and champion of regulatory pharmacology and the field of regulatory sciences owes much to him.…”