Did Big Pharma scuttle an attempt to radically restructure the approval pathway?

Posted under Blog, Companies, Diagnostics, Funding, Kay Hagan, Medical Devices, Medical Supply, Pharmaceuticals, Phase II, PhRMA, regulatory approvals, Startups, Universities, Videos by John Carroll

Sen. Kay Hagan's proposal to set up an accelerated approval pathway for experimental medications hasn't been universally embraced by everyone in the biopharma industry. And a contributing Forbes columnist claims that he knows why: Big Pharma forces helped scuttle an initial proposal that could have triggered a major retooling of the approval process, significantly restructuring the relationship that exists between pharma companies and the biotech companies that often deliver their most innovative treatments.  

In an in-depth article, the News & Observer notes that the National Organization for Rare Disorders, The Friends of Cancer Research, Parkinson's Action Network and the Biotechnology Industry Organization all lined up behind the TREAT Act, which promises to accelerate the approval process for developers who can prove that they can potentially treat an unmet medical need or significantly advance the standard of care for people suffering from deadly diseases. But PhRMA and its Big Pharma allies appear to be standing on the sidelines for now.

In a column in Forbes today, though, Avik Roy--a frequent critic of government bureaucracy and left-wingers of all stripes--maintains that the new TREAT Act is a pale version of its original form. Initially, he writes, Hagan, a North Carolina Democrat, had sought to offer a proposal that would have allowed developers to go after a provisional approval with Phase II data. 

"Sen. Hagan's proposal would have been devastating to the big pharma R&D oligopoly," writes Roy. "If small biotech companies could get their drugs tentatively approved after inexpensive phase II studies, they would have far less need to partner those drugs with big pharma. They could keep the upside themselves and attract far more interest from investors. Big pharma, on the other hand, would be without its largest source for innovative new medicines: the small biotech farm team."

So PhRMA and the big companies moved to water the TREAT Act down, Roy claims. The new act "merely allows for the existing, surrogate marker-driven accelerated approval process to address 'highly targeted therapies for distinct subpopulations.'" 

Erik Gordon, a biomedical expert and business professor at the University of Michigan, told the News & Observer that the bill still has the potential to make biotechs less dependent on Big Pharma, which typically picks up a program and carries it through the expensive late-stage study. 

"Smaller biotech companies often can't raise enough money on their own to do the currently-required safety trials, so they want to dilute the rules to let them get approvals without having to give big pharma a cut of the action in return," Gordon told Bloomberg a few weeks ago. "Big pharma says 'don't dilute safety measures for new drugs.' That sounds better than 'we want to keep our cut of the action.'"

- here's the column from Roy
- read the story from the News & Observer

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Senator launches campaign for speedier approvals of targeted drugs

Posted under Blog, Companies, congress, Diagnostics, Drug approvals, Funding, Kay Hagan, Medical Devices, Medical Supply, PDUFA, Pharmaceuticals, Startups, Universities, Videos by Ryan McBride

In an already politically charged year for the FDA, U.S. Senator Kay Hagan is spearheading legislation that would expedite reviews and approvals of drugs against serious medical conditions, her home state's North Carolina News Network reported.

The report says the legislation aims to give speedier reviews to drugs that deliver major benefits to patients, including targeted meds for patients with serious diseases and therapies that address underserved medical needs. The details of the North Carolina Democrat's plan were scant, and it's unclear exactly how quick she wants reviews to be. The FDA, of course, already has practices in place to speed new drugs to market if they offer superior benefits to patients, among other things.

“But for patients suffering today from some diseases for which there are no current treatments or for those patients who are clutching to the hope that a better treatment will be developed for their rare diseases, medical advances cannot come fast enough," said Hagan, as quoted by NCNN. She pointed to successful programs that sped new HIV treatments to patients in the 1990s to bolster her case.

Some lawmakers and biopharma backers have blasted the FDA for slowing the advance of innovative therapies to the market. FDA boss Margaret Hamburg has answered critics by emphasizing the agency hit a 7-year high in approvals last year, so the regulator can indeed act quickly to green light drugs when warranted. Take its recent speedy approval of Vertex's ($VRTX) cystic fibrosis drug Kalydeco, which the agency blessed last month ahead of schedule. Still, agency brass will need to gain support from U.S. lawmakers to reauthorize PDUFA this year, making 2012 a good time for members of Congress to voice their hopes and concerns about how the FDA conducts business.

- check out Hagan's release
- here’s the report from NCNN

Special Report: FDA approvals of 2011

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