Senate passes FDA user fee reauthorization

The Senate overwhelmingly passed bipartisan legislation Thursday that reauthorizes U.S. Food and Drug Administration's ability to collect user fees from drug and device makers.

The FDA user-fee agreements, which are renegotiated every five years with the makers of prescription brand drugs, medical devices, generic drugs and biosimilars, fund nearly half of the FDA's operations. The agency would have to layoff more than 5,000 employees if the agreements are not reauthorized before the end of September. A reauthorization delay would also postpone the review of many drugs and devices. The bill passed 94-1, with Sen. Bernie Sanders (I-Vt.) as the lone holdout.

The bill would allow the FDA to hire more staffers, speed up approvals and create a more defined review process of drugs and devices, which could help healthcare companies streamline operations, supporters argue. Critics say that the rising fees disproportionately affect smaller companies, and expediting the review of drugs and medical devices could compromise quality.

The Congressional Budget Office estimates that the FDA would assess about $9 billion in fees over the 2018-2022 period—$8 billion for prescription drugs and $1 billion for medical devices. President Donald Trump lobbied for an even bigger fee hike so that the agency would be entirely funded by user fees, as proposed in his May budget proposal. That proposal has been rebuffed by many policymakers who will now wait and see if Trump signs the bill.

"The legislation we are considering today will help bring lifesaving drugs to the marketplace and ensure the FDA continues to operate smoothly," Sen. Susan Collins (R-Maine) said at the hearing.

The controversial "right-to-try" legislation was tacked onto the bill Thursday, which Sen. Ron Johnson (R-Wis.) argued would allow terminally ill patients receive experimental treatments that haven't received FDA approval. The legislation would prevent regulators from looking at outcomes of products that could "delay or otherwise adversely impact review of approval," which has drawn concern from critics who contend patient safety would be compromised. The House has not approved the right-to-try legislation yet.

Lawmakers also passed by unanimous consent two other bills: one from Sen. Joe Manchin (D-W.V.) that would require hospitals and physicians to note a patient's history of opioid addiction in medical records and another from Sens. Roger Wicker (R-Miss.) and Amy Klobuchar (D-Minn.) that aims to ensure that the patient experience and related data be considered as part of the risk-benefit assessment during a drug's development.

The user fee bill included the Research to Accelerate Cures and Equity for Children Act that would eliminate current exemptions that allow drug and device developers to forgo pediatric studies of certain molecularly targeted cancer drugs. Supporters hope it would create an incentive for the pharmaceutical industry to develop new pediatric oncology treatments. Under the current system, some pediatric cancer drugs take decades to develop and get approved, lawmakers said.

Other provisions in the bill include easing requirements for medical imaging devices, creating a pilot project to evaluate the post-market safety of medical devices, improving access to clinical trials, forming a less expensive regulatory class of hearing aids that could be sold over the counter, adjusting the risk-based classification of medical devices in an effort to improve the predictability of review times, and improving generic drug competition by expediting reviews.

Stephen Ubl, CEO of the Pharmaceutical Research and Manufacturers of America, said the Prescription Drug User Fee Act could have a transformative effect. Building on the 21st Century Cures Act and other initiatives that FDA Commissioner Dr. Scott Gottlieb is undertaking including eliminating the agency's existing orphan designation request backlog hold much promise, he said.

"In terms of overall drug costs, as long as it takes $2.5 billion over 10 years to get a product to market, it is going to be challenging," Ubl said. "So, looking at ways to modernize and reduce those development cycles is really important."

Another provision in the user fee act aims to curb "bad actors'" abusive pricing on pharmaceuticals without generic competition. It provides a six-month exclusive rights period to any generic manufacturers who choose to compete against an off-patent drug that has no competition. Currently, that exclusivity is only available to a first entrant.

"Our purpose is to lower or at least moderate the escalating prescription drug prices that are one of the key cost drivers in our healthcare system," Collins said, adding that she supported Gottlieb's recent strides to increase generic drug competition.

The FDA has posted a list of branded drugs that are not protected by a patent and are not facing looming generic-drug competition in an effort to increase transparency and encourage generic development. It plans to eliminate the existing orphan drug request backlog and create a system for timely response deadlines for all future requests. The FDA also aims to fast-track its review of abbreviated new drug applications for generics for drugs with less than four competitors—a policy move that the FDA called the first of its kind.

As the campaign to repeal and replace the Affordable Care Act fades, policy experts are optimistic similar bills that aim to create a more level pharmaceutical playing field would pass.

"The events leading to the collapse of repeal and replace offers hope that Congress can now push forward with bipartisan reforms to tackle brand-name manufacturer tactics to extend their monopolies and, thus, delay the availability of affordable generic drugs," said Ameet Sarpatwari, an instructor in medicine at Harvard Medical School.