Brilliant
@ddwn - thankyou. I was suggesting that the long term results would make it difficult to refuse the application.
Below is a part of the FDA's guidance on Priority reviews. Although sponsors must ensure the availability of quality product at the time of approval,
FDA
may exercise some flexibility on the type and extent of manufacturing information that is
expected at the time of submission and approval for certain components (e.g., stability updates,
validation strategies, inspection planning, manufacturing scale-up). The level of flexibility will
be
determined on a case-by-case basis after consideration of factors such as the following: (1)
product characteristics, (2)
seriousness of the condition and medical need, (3) manufacturing
processes, (4) the robustness of the sponsor’s quality system, and (5) the strength of the
sponsor’s risk-based quality assessment. FDA’s consideration of the sponsor’s proposal for an
integrated postmarketing plan will also take into account whether elements of the plan may be
appropriately executed as a postmarketing commitment or requirement. For example,
FDA will
consider impacts on clinical performance, such as safety and immunogenicityQualification for Accelerated approval criteriaA drug that treats a serious condition AND generally provides a meaningful advantage over availabletherapies AND demonstrates an effect on a surrogate endpoint that is reasonably likely to predict
clinical benefit or on a clinical endpoint that can be measured earlier than irreversible morbidity ormortality (IMM) that is reasonably likely to predict an effect on IMM or other clinical benefit (i.e., anintermediate clinical endpoint) The new information demonstrates a meaningful advantage over other trials - the FDA's own literature
states flexibility in considering approval for unmet needs. Si is indicating that Survival is the holy grail. I like this description of survival -
Overall survival offers the greatest clinical gain, provided that quality of life is not compromised. As an endpoint, os is easily measured, unambiguous, objective, felt to be clinically significant, and unaffected by the timing of assessment.
As Mesoblast has had an independent review of 2-4 year results showing significant improvement in survival and the FDA has flexibility around approval criteria for unmet needs.........
I topped up
Can't wait to tell the Mrs
Reg.
(20min delay)