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The FDA passes new legislation for user fees and right-to-try applications

Monday, August 14, 2017

On August 3, the Senate quickly passed two new healthcare bills: user fee and right-to-try. The user fee bill is in line to be signed into law by President Trump.

Once signed into law, the user fee legislation will reauthorize user fees for the FDA’s review of drugs and medical devices. Companies that produce these products will pay the FDA for these reviews. Those fees will then provide additional funding for the organizations’ therapy-related activities. The FDA utilizes user fees to fund more than a quarter of its annual budget, and the financing directly received from drug manufacturers are meant to help the FDA work toward approving life-saving therapies with a higher level of efficiency. 

In addition to providing funding through the drug review process, parts of the new legislation will also speed generic drug application reviews if those products have little to no competition. Ultimately, the new drug user fee agreement is aimed toward setting a standard within the industry that may facilitate the swift approval of drugs to market.

According to Steve Ubl, the president and CEO of Pharmaceutical Research and Manufacturers of America (PhRMA), “[The agreement] better incorporates real-world evidence and patient perspectives into the drug development and approval process, and provides the FDA with new tools and resources to keep up with the latest scientific advances.” Ubl also believes this new legislation will bolster competition and ultimately translate to lower costs.

But when it comes to new or updated healthcare policies, critics are often at the forefront. There are some in the industry—as well as in the Senate—who are not fully supportive of the user fee bill. Senator Bernie Sanders, for example, was the lone holdout to the bill’s approval. Executive director of the Project on Government Oversight, Danielle Brian, voiced one of the first critical viewpoints of the new bill.

According to an article in BioWorld, Brian believes the fast-tracking of the bill was one of the first major issues. In addition, Brian stated that patients “could be left in danger as malfunctions mount,” citing the bill’s terms, which states that prompt reporting of medical device malfunctions as adverse events will not be required. Influence of industry lobbyists in the crafting and approval of the bill was also a concern to Brian, “The agency negotiated the deal with a budgetary gun to its head.”

That same day, the Senate also passed the right-to-try legislation, which allows terminally ill patients to gain access to investigational drugs from drug companies without FDA approval. The right-to-try bill will expand patient access to unapproved medications that have yet to move to or beyond phase II clinical trials.

Also referred to as “compassionate use,” the right-to-try law specifically helps patients with terminal illness gain access to medications that have successfully made it through phase I studies, but have yet to be studied for safety, toxicity and side effects. Most of these patients are ineligible for participation in drug trials.

The FDA reports that it currently approves 99 percent of compassionate use requests. The new right-to-try law will prevent the U.S. government from denying investigational therapies. Additionally, this bill seeks to set national standards that will protect both doctors and drug companies from potential lawsuits filed by patients who have been injured by experimental medications.

Additionally, the FDA will be able to gather and use information from patients who have gained access to these unapproved medications, incorporating this information into future approval decisions.

Starlee Coleman of the Goldwater Institute, a Phoenix-based libertarian organization, is in full support of the bill and believes its details to be mostly ironed out, aside from a few potential caveats. “[Although] the Senate passed the bill by unanimous consent,” said Coleman, “getting any piece of legislation through Congress, no matter how uncontroversial, is always a challenge, and we anticipate there will be significant debate in the House when the bill is considered.”

One of the biggest issues with compassionate drug requests is that many patients have a difficult time accessing these therapies. Expanded access programs, while helpful in this regard, often involve the patient having to accomplish several steps in order to access the unapproved drug. Even then, the drug company may not grant access.

Introducing new bills, like the right-to-try legislation, isn’t without its challenges. Medical ethicists have been critical of this legislation for years. Undermining the FDA and allowing drug companies to issue experimental drugs directly to patients represents one example why many within the industry feel uneasy about the potential law. The consumer group Public Citizen is opposed to the bill and believes it will introduce “false hope to patients.”

Dr. R. Adams Dudley, director of University of California San Francisco’s Center for Healthcare Value, previously stated his concerns about the right-to-try legislation by asking, “We know some people try to take advantage of our desperation when we’re ill. . . how do we protect people from physicians or drug companies that will want to sell them things and will want to prey on their desperation?” Despite this, it can be difficult to voice these criticisms to dying patients. “It’s extremely hard to articulate a position against this because it sounds like you’re not sympathetic to a patient’s plight,” according to a quote by Beth E. Roxland, a senior adviser on law, ethics and policy at NYU Langone Medical School.

While the right-to-try legislation remains under debate, the new user fee bill, once signed into law, will be in full force until 2022. At this time, the law’s impact will be reviewed to determine its working utility in improving healthcare outcomes across the board.

 

This article was reprinted from Volume 21, Issue 32, of CWWeekly, a leading clinical research industry newsletter providing expanded analysis on breaking news, study leads, trial results and more. Subscribe »

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