Pharmas have embarked on the long road to developing new ICH guidelines that could expedite patient access to therapies for severe or life threatening diseases by up to three years, calling upon advanced cancer guidance as a model.
The proposed guideline would eliminate or defer to the post-market setting some preclinical toxicity testing, like pharmacology safety, carcinogenicity and fertility tox studies, while shortening the duration of others, like general toxicology tests.
But according to one regulatory official and three industry executives, publication of the final guidance is likely years away, given the glacial pace at which the International Council for Harmonization (ICH) typically operates.
The biggest challenges may be finding consensus on the definition of a severely debilitating/life threatening (SDLT) disease, and the criteria that would be used to determine when an adequate number of effective therapies are available such that a disease no longer qualifies as SDLT.
While regulators have previously implemented streamlined development pathways, these have been limited to specific disease settings.
“It is time to really consider a fresh approach.”
The early days of HIV are a prime example of reduced toxicity testing requirements for a specific SDLT disease. Preclinical and early clinical requirements were stripped to the bare necessities to ensure patient access to new therapeutic options as