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FDA overhaul legislation could reach president by July 4
House and Senate leaders continue to try to work out differences between each chamber’s version of the Prescription Drug User Fee Act, which will authorize a 5-year extension of the FDA’s authority to assess user fees on prescription drug and medical device manufacturers.
The two bills — which significantly overhaul the FDA — also contain provisions intended to help prevent drug shortages, accelerate federal approval of generic drugs and speed up reviews of breakthrough therapies for life-threatening diseases.
The Senate approved its version May 24 by a 96-1 vote. Six days later, the House of Representatives approved a similar bill by a 387-5 margin. Members of each chamber designated to reconcile the differences between the two pieces of legislation hope to forward a compromise measure to President Obama for his approval by July 4. The FDA’s current authority to assess user fees expires Sept. 30.
The Senate and House bills would require drugmakers to notify the government about potential drug shortages, including when they plan to discontinue or interrupt production. The federal government would have the ability to alleviate shortages by finding new suppliers, either within the country or overseas.
Dale Shepard, MD, PhD, an associate staff physician in solid tumor oncology at Taussig Cancer Institute at Cleveland Clinic, called the congressional action “a start” but expressed concern that the legislation does not go far enough or address the underlying problems that trigger shortages.
For example, profit margins on several drugs are small, Shepard said.
“If a company tells the FDA, ‘We’re not making enough money on this drug, so we’re going to stop making it,’ what is the incentive for someone else to make it?” Shepard said. “The very reasons one company may discontinue production aren’t going to make it attractive for someone else to pick up the slack, and there is still a shortage. How are you filling that shortage? We know about it, but what can we do about it?”
Drug shortages reached record highs in recent years. The number of drug shortages tripled, from 61 in 2005 to 178 in 2010, according to an FDA report released last fall. An estimated 230 to 270 drugs went into shortage last year.
Oncology and hematology have been particularly hard hit.
In January, a survey of 204 oncologists conducted by National Analysts Worldwide revealed that about 80% of respondents had been “unable to provide some patients with appropriate or timely treatment due to drug shortages.” Nearly 50% of respondents reported observing tumor recurrence and 40% reported observing early death among their patients due to shortages.
Many of the drugs most vulnerable to shortages — older, generic sterile injectables — are used to treat blood disorders, according to a statement released by the American Society of Hematology.
In its statement, ASH expressed concern that the House version of the legislation exempts products used to treat hemophilia and other bleeding disorders — specifically, products derived from human plasma proteins and recombinant products replacing human tissue — from the early reporting mandate.
The society is working to ensure those products are included in the final legislation.
The organization also has recommended several possible ways to prevent or mitigate shortages. Those suggestions include increasing FDA authority, requiring that information about drug shortages be disseminated to patient and provider organizations, enhancing inter- and intra-agency coordination on drug shortages, and providing economic incentives to manufacturers of critical drugs.
“ASH will continue to monitor all hematologic drug shortages closely and will continue to work with policymakers to further strengthen the [Prescription Drug User Fee Act] to protect the health of Americans,” the ASH statement read.
The legislation would give hospitals the ability to redistribute medication within their own networks, an action prohibited under current law.
“I think that [provision] — the ability to actually utilize the drugs you have — certainly helps patients,” Shepard said.
The compromise legislation is expected to require drugmakers and medical device companies to pay $6.4 billion over 5 years to help cover the costs incurred by the FDA during its review of their products.
The measure also would introduce fees for the review of generic drugs and less expensive versions of biotechnology products. The generic drug fees are expected to reduce review times to a third of current levels and improve the availability of generic products for patients, according to sponsors of the legislation.
The Congressional Budget Office said the legislation would allow earlier marketing of lower-cost drugs, which would reduce the average price of drugs on the market. The CBO also estimated the legislation would save more than $750 million over 10 years in Medicaid, Medicare and federal subsidies for private insurance.
Perspective
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It is encouraging to see near-unanimous bipartisan support for something so vital to our health care system. The proposed legislation enables better access to critical drugs without changing the pathways used in the United States to approve drugs for usage in humans. It should enable better and faster approval of drugs, most notably generic drugs. However, it does not address a core element of the current dilemma — the fact that increasingly off-patent drugs are not “profitable,” or maybe not “sufficiently profitable.” It will therefore be interesting to see whether the envisioned shortening of review times for generic drugs, a laudable goal for certain, will actually translate in sufficient companies engaging in producing such “low-margin” drugs.
Because the demand for such drugs (eg, doxorubicin) is certainly there, one assumes that the supply-and-demand equation will ensure supply. But that remains to be determined. It is reassuring to see that this new law, in fact, foresees the potential for a lack of internal supply by requiring federal officials to help “prevent or mitigate” shortages by finding supplies elsewhere, even abroad if need be. As long as the quality of the drugs made abroad is similar to those manufactured in the United States, allowing drugs made abroad to enter the US market not only makes sense but would be essential to ensure that the gains made in outcome for patients with cancer are retained.