Innovation Pharmaceuticals Inc (IPIX)

[slcimmuno]

Endpoints: Yep – and folks arguing the contrary should note the increasing push for Surrogate Endpoints as “validation” of a drug’s efficacy, more wiggle room given to those under “accelerated approval”

If B-OM results pan-out in Primary—Yipee
But the Secondary/Exploratory measures should not be discounted, esp in a Ph2 (non-pivotal trial)

Summary [slide 19]
• For marketing approval, there must be substantial evidence (consisting of adequate and well-controlled investigations) of something that matters
• The primary endpoint(s) of confirmatory Phase 3 trials should represent (directly or through a validated surrogate) something that matters to a patient.
• There is a pathway for approval based on something that probably matters (an incompletely validated surrogate), but this comes with certain commitments. (“Subpart H” “accelerated approval”)
• Patient reported outcome (PRO) instruments and composite endpoints may be used to establish a benefit

https://www.fda.gov/downloads/Training/ClinicalInvestigatorTrainingCourse/UCM337268.pdf

Multiple Endpoints in Clinical Trials (Draft Guidance)(2017)
“Because most diseases have more than one consequence, many trials are designed to examine the effect of a drug on more than one endpoint. In some cases, efficacy cannot be adequately established on the basis of a single endpoint. In other cases, an effect on any of several endpoints could be sufficient to support approval of a marketing application.”

https://www.federalregister.gov/documents/2017/01/13/2017-00695/multiple-endpoints-in-clinical-trials-draft-guidance-for-industry-availability

https://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm536750.pdf