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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Pharma defeats biotech push for rapid approval program

Posted under Biotechnology Industry Organization, biotechs, Blog, Clinical Trials, Companies, Diagnostics, drug development, FDA, Funding, Jim Greenwood, Medical Devices, Medical Supply, Pharmaceuticals, regulatory approvals, Startups, Universities, Videos by Mark Hollmer

Biotechs butted heads with their far-larger pharmaceutical allies over a key lobbying issue, and lost. As Bloomberg reports, biotechnology companies threw their weight behind a plan drafted by North Carolina Democratic Sen. Kay Hagan to launch a more rapid FDA approval program, using fewer human trials, for orphan drugs or treatments that address fatal diseases. Pharmaceutical companies beat back the measure, however, apparently not wanting to dilute existing safety measures.

In a word: awkward. Both industry sectors lobby as part of the Biotechnology Industry Organization, a group that theoretically represents both sides' interests equally as drug development increasingly overlaps between each side. BIO board members representing the far smaller biotechs supported the proposal, but their pharmaceutical industry counterparts nixed the idea, Acorda Therapeutics ($ACOR) CEO Ron Cohen told Bloomberg. (His company, of course, is a biotech.)

Biotechs want the plan because they'd gain a more rapid approval timeline by redefining what efficacy means, using things like biomarkers that measure a drug's potential effectiveness, early trial results, or interim data to support regulatory approval. The idea arguably reflected a more innovative approach typical for biotechs, but they are also still relatively small and not generating significant revenue, so they'd welcome anything that could help better manage the financially punishing cost of traditional, extensive clinical trials. On the other hand, larger pharmaceutical companies want to preserve the existing system, citing safety concerns, but the status quo also helps preserve an important financial stake. Pharmas often help fund biotech clinical trials and then share the revenue once a drug hits the market, University of Michigan business professor Erik Gordon noted to Bloomberg.

A GlaxoSmithKline ($GSK) spokeswoman told Bloomberg that her company (one of the Big Pharma BIO members) preferred changes to existing programs with which regulators and regulated companies already work. Along those lines, BIO, perhaps not surprisingly, is pushing a compromise plan that would add other orphan drug or fatal disease-treatments to an existing program that accelerates approval for both cancer and HIV drugs, BIO CEO Jim Greenwood said in the story. Hagan will submit the new proposal to Congress later this year, Greenwood said, and it may be folded into larger legislation that renews the drug industry fee system that funds FDA regulatory reviews.

The compromise throws biotechs another bone: the use of surrogate endpoints, which show how a drug affects biological activity. But regulators would still require companies to prove clinical outcomes at some point.

- here's the Bloomberg story

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