Innovation Pharmaceuticals Inc (IPIX)

[baytdr]

came across this regarding temporary marketing approval, FWIW;

"Clinical endpoint ‘validation trials’ that also provide biomarker data for earlier regulatory review under the FDA Accelerated Approval (AA) process.

In 1992, the US Congress created a regulatory process, often referred to as ‘accelerated approval.’ Under this process, a sponsor could receive temporary marketing approval from the FDA for a new treatment regimen that appears to address unmet needs for patients having a life threatening disease, if clinical trials establish the regimen has compelling effects on a biomarker and if such effects are ‘reasonably likely to predict clinical benefit.’ Once AA is achieved, a ‘validation trial’ must be completed in a timely manner to determine whether this new regimen truly provides meaningful benefit on clinical endpoints, i.e., on tangible measures of clinical benefit [11]. To ensure the validation trial will be completed in a timely manner after AA has been granted, a recent practice in the oncology setting has been to design trials with the dual objective of (i) providing interim data on biomarker effects that, if compelling, would be released to sponsors and FDA for consideration of AA; and (ii) serving as the ‘validation trial’ through continued enrollment and follow-up to provide a definite evaluation of effects on tangible measure(s) of clinical benefit. Recognizing that the principal evidence regarding the benefit to risk profile of the experimental regimen is obtained from the longer term second objective, early release of the biomarker data is a breach of the confidentiality principle concerning interim data that can compromise the integrity of the validation trial."