Drug Industry Fees May Head to Congress by End of 2016

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By Jeannie Baumann

July 20— The FDA may send to Congress ahead of schedule its goals for the next drug industry fees law, an industry representative said July 20.

This expedited schedule would seek to prevent delays in the must-pass bill caused by a change in presidents and a new Congress in 2017.

The sixth authorization of the Prescription Drug User Fee Act (PDUFA VI) marks the first time in the program's nearly 25-year history that negotiations happened during an election year for a new president, Kay Holcombe, senior vice president of science policy for the Biotechnology Innovation Organization, told reporters.

The Food and Drug Administration must submit its letter on PDUFA VI performance goals by Jan. 15, 2017; a new Congress convenes Jan. 3 and a new president will be sworn in Jan. 20.

Holcombe said, “I think FDA’s hopeful that perhaps everything can be ready to send to Capitol Hill maybe before the end of [2016], so that maybe they can avoid falling into that hiatus pit.”

Unique Situation

“I think they recognize the uniqueness of this situation,” Holcombe said about the FDA, adding that many FDA staffers at the agency have been there a long time. “And they recognize that you do frequently have a period of hiatus where big decisions are very difficult to make because people are coming and going, and we don’t know ‘who’s on first.'”

Holcombe said, “As far as what we can do, we as the industry really can’t navigate that particular piece of this. It’s going to happen. We’re going to be in the same boat as everyone else.” But drug and biotechnology lobbyists have spoken with congressional aides about PDUFA and the new agreement to make the case for importance of moving forward. “I think they all recognize that as well.”

Performance Goals

The final user fee laws often contain policy measures affecting how the FDA does its job. The PDUFA program was created by Congress in 1992, and must be renewed every five years, according to the FDA.

Legal and policy leaders at BIO and the Pharmaceutical Research and Manufacturers of America held a joint press briefing on PDUFA VI five days after the FDA released an agreement it entered with the pharmaceutical industry. The agreement sets the stage for congressional action to renew the industry-paid fees that help fund the agency.

Specifically, the July 15 agreement describes the performance goals and procedures of the FDA's Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER). This reauthorization will cover fiscal years 2018-2022 (10 LSLR 15, 7/22/16).


Sascha Haverfield, senior vice president, science and regulatory advocacy at PhRMA, described the new agreement as a transformational effort that builds upon past user-fee agreements.

“There is a very strong focus on ensuring patient safety [and] maintaining the appropriately high FDA standards for safety and efficacy,” Haverfield said. “Ultimately this will promote an environment that can foster innovation and will lead to timely access to safe and effective innovative medicines for patients.”

Some of the general commitment topics in PDUFA VI, Holcombe said, are an attempt to streamline the entire drug development process, which the Tufts Center for the Study of Drug Development now estimates costs $2.6 billion per drug and can take more than a decade.

“Right now getting a drug from the top of the funnel to dripping off the bottom of the funnel, is a long process, of which the FDA portion is a mere drop in the bucket,” she said.


The latest PDUFA agreement's initiatives include:

  •  facilitating the systematic integration of patient perspective into the drug development and review process,
  •  enhancing the biomarker qualification process by the use of novel biomarkers as for new surrogate endpoints,
  •  using of real-world evidence in regulatory decision making either alone or in conjunction with clinical trials,
  •  expanding the breakthrough therapies program,
  •  adopting innovative clinical trial designs that deviate from the randomized, controlled clinical trial, which has generally been the gold standard for testing new medical products,
  •  streaming regulatory review of combination products, and
  •  working on model-informed drug development, which is an approach to drug development that uses the development and application of statistical models derived from preclinical and clinical data sources.

Shaving off three years of clinical development would save tens of millions of dollars, Holcombe said, but questions still need to be answered exactly how much time or money would be saved from using one of these innovative trial designs.

“That’s what we are hoping to learn from these initiatives,” Holcombe said. “We definitely are confident that you have to try something different from what you’ve been doing all along because things are not getting shorter and they’re not getting less expensive. The reverse is true. So we need to try different ways and think about different ways of doing things while still preserving the high standard of safety and the high standard of efficacy that FDA has always had.”

FDA Capacity Building, Public Engagement

Much of the focus of the programs involves building capacity at the FDA through hiring more people with the right expertise, and by holding public forums or workshops on topics such as new approaches to biomarkers, real-world evidence and new clinical trial designs.

“Some of these trial designs are so creative that it’s the first time the agency has ever seen it,” Holcombe said. “What the agency wants to do—and what PDUFA VI resources will allow them to do—is build up its expertise to be able to look at these trial designs and make a determination about whether the data to demonstrate safety and effectiveness are going to be able to be collected if the trial is designed in this way.”

On the real-world evidence, Jeff Francer, vice president and senior counsel at PhRMA, said it's pretty clear the FDA is going to have to go through a public process and take input into how these data can be used to meet existing safety and effectiveness standards.”

“This is very much a walk-before-you-can-run approach,” Francer said. “We're really trying to make sure that FDA has a robust public process, which could include the academic community, the tech community and others to make sure that there's full scientific agreement on the ability of real-world evidence to be used for regulatory decisions.”

There is also a provision in PDUFA VI that focuses on drug safety, specifically by expanding the Sentinel program, which allows the FDA to access data from sources such as electronic health records, insurance claims data and registries, and other data partners. There's also another topic that would build on ongoing efforts to enhance communication between sponsors and the FDA.

The legislative authority for the current PDUFA expires in September 2017. At that time, new legislation will be required for the FDA to continue to collect user fees. PDUFA most recently was reauthorized through FY 2017 (PDUFA V) as Title I of the Food and Drug Administration Safety and Innovation Act (FDASIA, Pub. L. No. 112-144), a 2012 law.

The agency has scheduled a public meeting on the user fees agreement, set for Aug. 15 in Silver Spring, Md.

To contact the reporter on this story: Jeannie Baumann in Washington jbaumann@bna.com

To contact the editor responsible for this story: Randy Kubetin at rkubetin@bna.com

For More Information

The FDA agreement document for FY 2018-2022 is at http://src.bna.com/gX4.

A Federal Register notice about the August meeting is at https://www.gpo.gov/fdsys/pkg/FR-2016-07-19/html/2016-16916.htm.

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