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Regulatory Focus™ > News Articles > FDA, Absent From Hearing, Makes the Case That it's Increasingly Innovative

FDA, Absent From Hearing, Makes the Case That it's Increasingly Innovative

Posted 21 May 2014 | By Alexander Gaffney, RAC

FDA, Absent From Hearing, Makes the Case That it's Increasingly Innovative

Yesterday, US legislators in the House Energy and Commerce Committee held a hearing to discuss how the federal environment—including the US Food and Drug Administration's (FDA) regulations—might be improved to bolster drug innovation. But while the pharmaceutical industry, device industry and patient groups found themselves well represented at the meeting, one prominent stakeholder found itself conspicuously excluded from the meeting: The US Food and Drug Administration.

Hearing Details

As explained by the committee in a meeting notice, the intent of the hearing was to follow up on a 2012 report by the President's Council of Advisors on Science and Technology (PCAST), which contained several regulatory proposals intended to accelerate drug approvals at FDA.

Those eight recommendations were the focus of the House Energy and Commerce committee meeting on 20 May 2014:

  • Recommendation 1: Support Federal Initiatives to Accelerate Therapeutics
  • Recommendation 2: Catalyze the Creation of a Broad-Based Partnership to Accelerate Therapeutics
  • Recommendation 3: Expand the Use in Practice of FDA’s Existing Authorities for Accelerated Approval and Confirmatory Evidence
  • Recommendation 4: Create a New Pathway for Initial Approval of Drugs Shown to be Safe and Effective in a Specific Subgroup of Patients
  • Recommendation 5: Explore Approaches for Adaptive Approval Via Pilot Projects Under Existing Pathways, but Do Not Create New Adaptive Approval Pathways Through Legislation
  • Recommendation 6: Improve FDA’s Tools for Monitoring and Communication of Clinical Benefits and Risks
  • Recommendation 7: Reform Management Practices at FDA
  • Recommendation 8: Study Current and Potential Economic Incentives to Promote Innovation in Drug Development

Those recommendations explicitly mention FDA five times, and the agency's absence from the meeting was therefore conspicuous. Instead, the committee called witnesses from the Biotechnology Industry Organization (BIO), the National Organization for Rare Disorders (NORD), Friends of Cancer Research (FoCR), the Center for Medical Technology (CMT) and Medgenics. Their respective testimony may be found on the Committee's website.

FDA Response

But despite FDA's absence at the hearing, it decided to use the occasion to highlight some of its recent successes on its blog. In a 20 May 2014 posting, FDA's Janet Woodcock, the director of the Center for Drug Evaluation and Research (CDER), said the agency has made "significant progress" in addressing the recommendations of the PCAST report.

Some of the highlights:

  • FDA has received 178 requests for breakthrough product designation, and granted 44 of those requests thus far. Six of those drugs have received full marketing approval from the agency.
  • FDA is involved in 22 public-private partnerships to develop research tools, platforms, clinical databases and predictive models.
  • Nearly half of all approved drugs obtained approval using one of FDA's expedited review pathways (fast track, accelerated approval, priority review, breakthrough product designation).
  • FDA supports (at least in concept) the creation of a "Special Medical Use" (SMU) or Limited Population pathway to encourage the development of drugs for small medical populations. Those pathways would prevent the drugs from being prescribed to those outside of the limited population. FDA has been soliciting comments on this idea since January 2013, but has only received cautious support from industry.
  • FDA is working on implementing a "structured Benefit-Risk Assessment framework," which is intended to better communicate the decision-making behind product approvals.

While none of these are necessarily a response to the testimony heard by legislators at the hearing, which included proposals for major overhauls of how FDA approves drugs, it indicates that FDA is paying attention and wants to let legislators know that it's working within the confines of its resources and authority.


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