The Provisional FDA

  • by: |
  • 08/14/2012

Good omnibus PDUFA V article in the August issue of Nature Biotechnology.

Here’s the opening paragraph:

After months of broad discussion followed more recently by narrower negotiations among legislators, late in June the US House of Representatives and Senate passed the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA), also known as the Prescription Drug User Fee Act V (PDUFA V). With wide bipartisan majorities, President Barack Obama signed FDASIA into law on July 9, ahead of looming deadlines. Whereas some are concerned that Congress sidestepped key issues in its haste to pass the legislation, industry has generally welcomed the new law, particularly its implications for research on treatments for rare diseases.

And some selected quotes:

In terms of fostering product development for rare diseases, FDASIA encapsulates “certainly the strongest language to date, but it's not that strong,” says Peter Pitts, president of the Center for Medicine in the Public Interest in New York. Moreover, the development and review of such products “can't be expedited until the agency becomes more transparent about its risk-benefit analysis...and really explains its decisions so that they can be replicated. We need the agency to be more predictable, even if its heart is in the right place.”

More generally, Pitts says, “I give this version of PDUFA only a gentleman's B. It could have been better.” For one thing, the new law mandates only that FDA work toward developing a “risk-benefit grid” instead of setting a specific date to complete this as well as other comparably challenging projects, he says. “The agency agreed to hold meetings, and it's good to talk. But it won't get to the right place as soon as it might.”

Pitts also points out that language in the new law will not make it easy to “hold [FDA] feet to the fire.” However, on the plus side, “Congress recognized the need for more regular oversight on technical aspects of regulation,” he says. “It's one thing to have hearings on politically expedient issues, but rarely does Congress take on the sophisticated but more boring aspects of drug regulation.”

The article also offers a nice table of PDUFA V highlights:

FDASIA reforms to FDA

* Accelerated Approval: Fast Track designation and accelerated approval for “a broad range of serious or life-threatening diseases or conditions”

* Advisory Committee Conflicts: No cap on conflict-of-interest waivers for advisory committee members

* Breakthrough Therapies: Expedited development and review of drugs for serious and/or life-threatening diseases that show major improvement over the standard of care in phase 1/2 trials

* Biosimilars Performance Goals: Commitment to process 70% of original biosimilar biological product application submissions within 10 months of the receipt date in 2013 (up to 90% in 2017)

* Communication Training: Staff at CDER and CBER to better communicate with drug development teams and manufacturing. Independent auditors to evaluate interactions between sponsors and FDA in discussions on labeling, REMS and other potential issues before submission and during review. FDA to consult with patients during product development and review and to host four meetings with patient groups per year in different diseases

* Critical Path Appropriation: $6 million annually for FY13–FY17 to promote public-private partnerships

* Drug Review Timelines: Extends by two months the PDUFA deadlines for Standard and Priority Review of NMEs and original BLAs

* Foreign Clinical Trials: Encourages collaboration with European and Asian regulators to prevent duplicative trials

* Guidances: FDA to issue guidance on development of abuse-deterrent drugs and on online promotion of regulated products, including social media. Sixty days before issuing any draft or final guidance on laboratory-developed tests, FDA must notify Congress

* Pediatrics: Formalizes the Best Pharmaceuticals for Children Act and Pediatric Research Equity Act and extends Priority Review voucher program to rare pediatric diseases

* Qualified Infectious Disease Products: Priority review and additional five years of exclusivity for novel anti-infective drugs against pathogens with “potential to pose a serious threat to public health”

* Rare Disease Experts: FDA to establish list of rare disease experts whom FDA may consult

The complete Nature Biotechnology article can be found here.

And speaking of fast track authority, Representative Brian Bilbray (R, CA) has drafted “The Patient Choice Act,” which would amend PDUFA V and provide a must swifter fast tract for critical and life-saving medicines.


Subject to the requirements of this subsection, if the Secretary determines that a drug that is designated as a fast track product under this section is adequately safe, the Secretary shall grant provisional approval and the drug may be introduced into interstate commerce on or after the date such provisional approval is granted.

Interestingly, all the power (per the draft language) rests with “the Secretary,” not the FDA. Considering the highly disturbing recent Plan B precedent – do we really want fast track approval authority residing with the Secretary of Health and Human Services?

Mr. Bilbray’s draft bill can be found here.

It is likely the last place you will ever see it.



Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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