A bill awaiting introduction in the Senate would create two new approval mechanisms for FDA.

Progressive and Exceptional approval, as they are named in draft bill, would essentially codify existing FDA practices of allowing drugs in certain cases to reach the market without the traditional evidence package.

Both are included in the Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act, which is set to be introduced by Sen. Kay Hagan, D-N.C., and could be included in the Prescription Drug User Fee Act reauthorization in 2012.

A product could qualify for exceptional approval if the data needed “cannot ethically, feasibly, or practicably be generated,” according to the draft legislation obtained by “The Pink Sheet.” FDA would be allowed to approve the drug based on an “alternative showing.”

Progressive approval would be allowed if the evidence submitted is likely to predict a clinical benefit for a designated population and use. Drugs using the pathway would be entitled to priority review and fast track status, if requested by the sponsor.

A Risk Evaluation and Mitigation Strategy or other risk management tool could be used to allow a progressive approval, according to the draft legislation.

Sponsors would be required to reach an agreement with FDA to derive the data needed to confirm the expected clinical benefit in the intended population, as well as submit all promotional material for review at least 30 days before dissemination until the agreement is satisfied.

Sponsors using either pathway also would have to submit biennial reports that include detailed summaries of new evidence that has been found since approval that could affect drug safety, efficacy or labeling.

The legislation also allows for withdrawal of either approval if the sponsor fails to comply with FDA agreements or approval requirements. The agency also could pull a progressive or exceptional approval if the sponsor disseminates false or misleading promotional material about the drug.

If the bill does go forward, that withdrawal process could receive more attention in light oRoche’s efforts to retain future of Avastin’s (bevacizumab) first-line metastatic breast cancer claim Also see "Avastin’s Breast Cancer Claim: Will FDA’s Hamburg Take A Middle Road?" - Pink Sheet, 4 July, 2011..

The emphasis on quality advertising is also likely to draw more scrutiny, especially since industry is waiting for guidances related to the proper use of social media in drug promotion.

Several drug companies closed their Facebook pages after the social networking site said it would no longer allow them to block consumer comments, partly because they did not know how FD A may view off-label and other comments Also see "Firms Navigate Social Media Safely Following Loss Of Facebook Protection" - Pink Sheet, 14 September, 2011..

Certainty By Formalized Process

Both new pathways seem to formalize practices FDA has used to approve some rare disease drugs as well as medical countermeasures. They also could create more certainty about the approval process, something industry and investors have requested in order to drive more drug development.

The Biotechnology Industry Organization and National Venture Capital Association have called for a progressive approval pathway as a way to drive investment and interest in the industry.

BIO included the concept in its “Big Ideas” for FDA reform that it announced earlier this year Also see "BIO’s "Big Ideas" For FDA Reform Are Too Big For PDUFA" - Pink Sheet, 4 July, 2011..

An industry source said a number of congressional offices have been contacted in an effort to get the proposals into legislation, including progressive approval, but the source did not know whether they would all end up in a single package.

Interest in the issue appears evident. Sen. Michael Bennet, D-Colo., called for an expanded and strengthened accelerated approval pathway to help patients with unmet medical needs, according to written remarks delivered Nov. 10 at the Clinical Cancer Research Conference. It could result in support to Hagan’s upcoming bill.

Kelly Slone, NVCA vice president of federal life science policy, said the legislative campaign is just beginning, but the progressive approval concept is the issue the group thinks could make the most impact in terms of driving more industry investment.

FDA appears to be open to an expedited drug development pathway and included it in a recent report outlining its biomedical innovation initiative as a way to spur more treatments targeting serious diseases with unmet needs Also see "FDA Biomedical Innovation Initiative Draws On Familiar Themes" - Pink Sheet, 10 October, 2011..

The agency already exercises what it considers quite a bit of regulatory flexibility to approve drugs for orphan indications, including through trial design. However, industry and venture capital groups continue to believe the agency doesn’t exercise it consistently Also see "Regulatory Flexibility Staring Contest: Will Industry Or FDA Blink First?" - Pink Sheet, 24 October, 2011..

The “animal rule” also allows for animal trials when human clinical trials are determined to be impractical or unethical and already has been used in vaccine development Also see "Animal Rule's Study Designs For Anthrax Vaccine Show Utility For Other Products" - Pink Sheet, 22 November, 2010..

Another PDUFA Add-On?

One of the most likely vehicles for the progressive and exceptional approval legislation is PDUFA. The reauthorization traditionally has included several FDA reforms along with the negotiated agreement between FDA and industry, although observers and stakeholders have indicated the bill likely will not include a long list of reforms from Congress as the previous reauthorization in 2007, which became the FDA Amendments Act.

Changes to conflict of interest rules for advisory committees, increased authority for FDA to protect drug supply chains and incentives for antibiotics development have been mentioned as other potential issues to be included in the legislation Also see "Not Another FDAAA – Observers Expect Fewer FDA Reforms In PDUFA V" - Pink Sheet, 11 July, 2011..

FDA “Progressive Approval” And “Exceptional Approval” Pathways Possible Under Senate Bill

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