The Biotechnology Industry Organization has backed off its calls for a new progressive approval pathway for drugs, instead favoring an expansion and clarification of the existing accelerated approval.

President and CEO Jim Greenwood said the group is moving toward a consensus among its members on the change and is working to finalize the proposal, with hopes for inclusion in the Prescription Drug User Fee Act reauthorization.

While it would not grab as much attention as creating a whole new approval pathway, BIO believes tinkering with accelerated approval to give the regulations more clarity and increase its use still could generate the change it wants at FDA.

Greenwood said during a Jan. 23 media briefing the trade group wants to update the pathway so it “reflects the modern scientific advances that we’ve made.”

Sara Radcliffe, BIO executive vice president of health, said the group still wants accelerated approval to be used for serious and life-threatening diseases and to meet unmet medical needs. It also wants to expand the endpoints FDA can use and let the agency accept a wider range of evidence to extend the pathway to more therapeutic areas.

Radcliffe said adjusting existing regulations also likely will allow FDA to implement the changes faster.

“Among the potential benefits of setting up a separate pathway was to really make clear that from our companies’ point of view there needs to be a genuine departure from the status quo,” she said during the briefing. “One of the advantages of doing it a slightly different way … is that it may enable us to make even clearer the differences between what is the case now and what Congress would intend for FDA to do going forward.”

The progressive approval concept was intended to allow FDA to permit a drug’s marketing if the evidence submitted was likely to predict a clinical benefit in a designated population or use.

BIO pushed the idea as one of its “Big Ideas” for FDA reform announced last year. Sen. Kay Hagan, D-N.C., included it and an “exceptional” approval idea, which would allow FDA marketing permission based on an alternative showing if data could not ethically or feasibly be generated, according to draft legislation Also see "FDA “Progressive Approval” And “Exceptional Approval” Pathways Possible Under Senate Bill" - Pink Sheet, 14 November, 2011..

Greenwood said Hagan’s bill, called the Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act, is expected to be formally introduced soon.

FDA is against any new approval pathway that would lower the standard for drug safety. Greenwood said BIO wants to make sure all parties understand those regulations will remain unchanged. He also said he doesn’t want it to influence reimbursement or become another drain on agency resources.

Greenwood also said there are questions about whether the change should be in PDUFA and whether it would affect the reauthorization.

“All of those questions have been in the mix here, they have been in the mix at [the Pharmaceutical Research and Manufacturers of America], they’ve been in the mix in the Congress, they’ve been in the mix at the FDA,” he said. “I think we’re very much converging towards a consensus that I think will be acceptable to all.”

The new proposal also seems to have at least some support from FDA. Janet Woodcock, director of the Center for Drug Evaluation Research, said during a recent TV appearance the agency believes accelerated approval could be used more and its uses clarified.

She appeared to offer the pathway’s clarification and expanded use as an alternative to creating a new progressive approval pathway Also see "Accelerated Approval Could Expand As Alternative To Progressive Approval" - Pink Sheet, 23 January, 2012..

No Set Formula For Legislative Success

The BIO “Big Ideas” proposal was codified in its report “Unleashing the Promise of Biotechnology,” which was released in June 2011.

In addition to progressive approval, it also called for FDA to be moved out of the Department of Health and Human Services and become an independent agency and for the commissioner to have a mandatory six-year term.

BIO also wanted the rules governing the conflict of interest of advisory committee members loosened and FDA to disclose in a “complete response” letter to sponsors its reasons for not approving a drug Also see "BIO’s "Big Ideas" For FDA Reform Are Too Big For PDUFA" - Pink Sheet, 4 July, 2011..

Hagan’s bill is expected to include many “big ideas”. Greenwood said the mandatory commissioner’s term and moving FDA out of HHS could move in a separate bill.

Greenwood also said legislation including many of BIO’s capital formulation proposals, intended to increase investment in the industry, could be introduced soon.

Brent Del Monte, BIO vice president of federal government relations, said a comprehensive bill or a series of bills could be proposed.

“There is no set formula for legislative success,” he said. “Our goal … is not to jam anything into any package over the objection of major stakeholders.”

It may be difficult for BIO and its allies to get a series of bills passed. Only a small number of bills are expected to be able to garner enough support in both houses of Congress this year, given the existing hyper-partisan atmosphere and the upcoming presidential election.

User fee legislation likely will be the preferred vehicle for many of these proposals because it is must-pass legislation, although it is not expected to be littered with too many additions Also see "Drug Shortage Bills Likely Drivers Of PDUFA Add-ons" - Pink Sheet, 29 November, 2011..

Senate Hearings Not Scheduled, But Expected

FDA has delivered proposed legislative language and final agreements with industry for PDUFA, as well as new user fees for generic drugs and biosimilars, to Capitol Hill.

The Senate Health, Education, Labor and Pensions Committee has not announced any upcoming hearings on the subject, but at least one more is expected.

The House Energy and Commerce Committee scheduled hearings for next month on the three agreements. Health Subcommittee Chairman Joe Pitts, R-Pa., has set a goal to have the bills passed and ready for President Obama’s signature in June Also see "PDUFA V Action Heats Up On Hill With House Hearings In February" - Pink Sheet, 12 January, 2012..

BIO Wants Expanded Accelerated Approval In PDUFA Instead Of Progressive Approval

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