"(Reuters) - Experimental drugs that show a big effect early in development for treating serious or life-threatening diseases would get a faster and cheaper path to U.S. approval, under a proposal likely to become law this year.
"U.S. drug regulators would be able to label such treatments "breakthrough" therapies, and work with companies to speed up clinical trials, for example by testing the drugs for a shorter time or enrolling fewer patients.
"Jeff Allen, executive director of Friends of Cancer Research, said the time was right for another avenue to speed innovative treatments to patients.
"Allen said new understanding of human biology and of diseases meant companies could create more effective drugs, but with fewer side effects, because they would be more targeted to specific patients or disease types.
"He said a good example was Roche Holding AG's skin cancer drug Zelboraf, approved last year; people taking the pill were 63 percent less likely to die from melanoma than people on standard chemotherapy.
"There was also Pfizer Inc's targeted lung cancer drug Xalkori, which could shrink or eliminate tumors in 10 to 12 months for people with a specific genetic mutation.
"'The most promising drugs show an effect early,' Allen said. '(But) there's a mentality among drug developers or FDA reviewers that you have to go through this multi-step approach' to get a drug to market.
"'We're hoping to encourage getting away from that,' he said.
"Allen said the FDA would retain its power to require companies to do post-approval studies, or withdraw drugs from the market if initial evidence of benefit was not shown in follow-up trials."
Could this mean that if the randomized P2 trials for 427 show very strong results, OGXI might be able to get approval without going through P3? That would mean they and similar companies might not need to partner with big pharma, so you'd think big pharma would oppose this legislation. On the other hand, they'd get drugs they're developing on their own to market sooner, small biotechs might still need them to fund P2 trials or to distribute approved drugs, and the drugs would be on the market longer before their patents expire.
Without more information on how exactly the legislation would work it's hard to predict its impact.
I'm not really knowledgeable enough to comment on the distinctions among existing FDA procedures for expedited review but I think that in general none of them eliminate the requirement of a phase 3 (or at least an almost as big and expensive phase 2b) trial. The link you provided goes into this in more detail, but it's my understanding that fast track mostly provides for rolling submissions of data (e.g., it allows you to submit manufacturing data while clinical trials are still ongoing), priority review shortens the time for the FDA to issue its decision following submission of a post-P3 NDA from 10 months to 6 months, and accelerated approval is post-P3 approval that is subject to the requirement that you conduct additional clinical studies.
In any case, the pending legislation that according to the link I posted is likely to pass this year would create the potential for faster approval than is currently possible. The link says the idea is to allow approval of "breakthrough" drugs following testing for a shorter time or with fewer patients, based on earlier-stage results. That's what made me think it could potentially permit approval of 427 based on strong randomized P2 results. Of course, that's just conjecture based on what little the article says about the legislation.
As far as the patent for 427 is concerned, according to OGXI's most recent 10-K it's derived from a license from the University of British Columbia and a license of Isis' 2nd-generation antisense technology. According to p.25 of the 10-K, "Our licensed UBC patent estate, based on those patents and applications existing now and expected by us to issue, will expire in years ranging from 2020 to 2024, which dates do not include extensions that may be available....Patent term extensions, specifically to make up for regulatory delays, are available in the United States, Europe and Japan. Although we believe that some or all of our product candidates will meet the criteria for patent term extensions, we can provide no assurance that we will obtain such extensions."