RE:2quick154Biofinder wrote: My bad, it appears I was thinking of U.K., E.U., Japan, etc., overseas has “conditional approvals”, looks like 2020 it is... https://www.forbes.com/sites/realspin/2016/05/05/the-fda-needs-a-conditional-approval-system/ The Food and Drug Administration (FDA) has long shunned the concept of conditional approvals – the granting of approval to market safe drugs with some indicia of activity for a period of time while additional studies are conducted to support full approval. The FDA’s Accelerated Approval Program allows for earlier approval of drugs that treat serious conditions, and that fill an unmet medical need, based on a surrogate endpoint. And, if mandatory subsequent studies that provide substantial evidence of clinical effectiveness are not positive, the drug is taken off the market.
At the eteplirsen advisory committee meeting last week, the panel voted seven to three against full approval and seven to six against accelerated approval for Sarepta’s drug for patients with a type of Duchenne Muscular Dystrophy (DMD). The FDA maintained that substantial evidence of effectiveness was not provided, and the panel agreed.
So reading this, how does "PLI" achieve "substantial evidence of effectiveness" with a trial in Sweden on 20 people?? Answer is, it can't. Need a FAR larger sample size. Kind of like the IPF study with too few people, then you have to go back and do the REAL P2 or they'll call it "P2/P3" study. I am so tired of the "proof of concept" P2 study like this on a handful of people, at the Swedish Omnio's pace. Meanwhile, a multi-billion opportunity awaits.... bahhh whats a few more financings, eh??? Proof of concept to lure in the big fish??? Don't like these mini trials in foreign countries under the Swedish Drug Health branch and NOT in the US under the FDA.