nnz-2566 trial updates, page-11

Mr Cod

I only know about cancer drugs being approved without phase 3 trials (sometimes just on a Phase 1 trial!). But it would only happen for 'unmet need' scenarios. I have copied and pasted below from this link: http://voiconsulting.com/blogs/news/tagged/cancer
Note the higher probability of 'no Phase 3 trial required' for orphan designated drugs.



As a rule, the FDA recommends that applications include data from “at least two adequate and well-controlled clinical trials” as a condition of standard drug approval.[1] In cancer, however, the high degree of unmet medical need and the urgency of offering new treatments for potentially fatal diseases mean that approvals are based on a wide range of clinical data packages.This is demonstrated in Figure 1 which shows that the “two adequate and well-controlled trials” standard is actually an unusual occurrence in cancer as only 2% of all indications received approval using this approach. Indeed, approved applications have been based on pivotal trials ranging from as little as a single Phase I pharmacokinetic study (asparaginase Erwinia chrysanthemi, approved in 2011 as part of a combination regimen for acute lymphoblastic leukemia) to as much as three separate Phase 3 trials (rituximab’s 2006 approval for use with CHOP chemotherapy in newly diagnosed Non-Hodgkin Lymphoma patients).Despite the variance, certain thresholds are evident. For example, 75% of all applications and 56% of orphan indications were directly supported with at least one Phase 3 trial. Standards are predictably higher for non-orphan indications, where 88% of applications included a Phase 3 trial. Of indications approved without Phase 3 support, most were for patients who had failed or who demonstrated intolerance to earlier lines of therapy which is understandable given the limited treatment options available to these groups.In addition to demonstrating the range of required data, Figure 1 also reveals that 59% of all indications were approved with a single Phase 3 trial. Nearly three-quarters of all non-orphan drugs fall in this category and it is the most common scenario for orphan drugs as well. In the absence of specific guidance from regulatory agencies, this may serve as the best available proxy for establishing the scope of a clinical trial program necessary to gain approval.