Bill to speed drug approvals sitting in Senate’s waiting room
Washington –It was hailed as a rare bipartisan victory when it was approved by the U.S. House of Representatives earlier this month, but legislation that would boost the fortunes of Connecticut’s pharmaceutical industry — and perhaps speed a cure for Lyme disease — faces an uncertain fate.
The 21st Century Cures Act would streamline the process of approving drugs to treat rare diseases. But in its 200 pages, down from an original 400, the legislation would do a lot more.
It would increase the annual budget of the National Institutes of Health by $1.75 billion, money that would be used to fund “high-risk, high-reward research” aimed at discovering new cures.
The bill would also establish a national pediatric research network and fund a new national tracking system for neurological diseases to help the Centers for Disease Control and Prevention collect information on illnesses like Parkinson’s and multiple sclerosis.
It would promote “personalized medicine,” an emerging field that uses an individual’s genetic profile to guide medical decisions concerning the prevention, diagnosis, and treatment of disease. The new Jackson Laboratory for Genomic Medicine in Farmington focuses on personalized medicine.
Connecticut’s pharmaceutical industry and growing biomedical industry support the bill.
But the bill has split Connecticut’s congressional delegation and come under attack by consumer groups and patient advocates concerned some sections would weaken regulations, leading to inadequate testing and the sale of unsafe drugs and medical devices.
One provision that has drawn fire from consumer groups would allow high-risk medical devices to be approved by the Food and Drug Administration based on case studies or medical journal articles alone.
The bill also would streamline the way the FDA approves some drugs. Most approvals are based on the outcomes of highly controlled clinical trials, but the bill would instruct the FDA to study how other data, like information from doctors’ practices or disease registries, could be used to approve new uses for drugs.
The bill would also allow the FDA to grant market approval to a drug by giving it a breakthrough designation based on its early-stage testing for safety and effectiveness. Clinical trials could be delayed until after the drug is on the market.
The bill would also add a new exemption to the Physician Payment Sunshine Act, which requires drug and medical device manufacturers to report payments they make to doctors. The filings are made public in a federal database. The bill would exempt the reporting of speaker fees or gifts to doctors that are intended for “continuing medical education.”
“Rather than addressing the true scientific bottleneck in drug and device development, the bill includes unnecessary, costly, and potentially harmful regulatory changes and financial incentives for pharmaceutical and medical device companies that would put patient safety at risk and undermine public health,” a coalition of groups, including Consumer’s Union, Public Citizen and the American Medical Student Association, wrote in a letter to lawmakers.
Courtney likes it; DeLauro doesn’t
Most Connecticut members of the U.S. House, including Rep. Joe Courtney, D-2nd District, supported the legislation when it was approved by the House on a 344-77 vote on July 13.
One reason Courtney supported the bill is that it includes the “Tick-Borne Disease Research Transparency and Accountability Act,” legislation he co-sponsored that would expand programs designed to speed development of crures for Lyme disease and other tick-related illnesses.
The bill would create a new working group consisting of federal scientists, experienced Lyme disease physicians and patient advocates. It would also require the Department of Health and Human Services to consult with the working group and submit a plan to Congress within three years establishing benchmarks for progress toward eradicating the disease. Lyme disease is named for towns in Courtney’s district where early cases were identified.
“Tick-borne illnesses are especially prevalent in eastern Connecticut, affecting thousands of residents,” Courtney said. “Just last year, more than 1,700 people in our state were diagnosed with confirmed cases of Lyme Disease — which is just one of the many diseases transmitted by ticks in the United States…I am pleased we were able to break through Washington’s partisan gridlock to advance this long-overdue effort…”
Rep. Rosa DeLauro, D-3rd District, voted against the bill, saying the extra money it would provide NIH “is not nearly enough.” DeLauro is the top Democrat on a House Appropriations subcommittee with authority over the NIH budget.
“If we are serious about funding life-saving medical research, we must raise our level of ambition,” said DeLauro, an ovarian cancer survivor.
DeLauro said she is concerned the bill would usher in more lax standards for approval of medical devices and new antibiotics. She also objects to the bill’s $500 million in incentives for certain types of medical research.
“The people best placed to decide which scientific avenues are worth pursuing are scientists and not politicians,” DeLauro said. “We should not substitute our judgment for theirs.”
Senate takes a different approach
The Senate has taken a different path to promoting new cures for rare diseases,
Introduced by Sen. Dick Durbin, D-Ill., the American Cures Act limits itself to steady funding increases at NIH and other federal medical research agencies. There are no policy or programmatic changes in his bill, nor is there any streamlining of regulatory procedures.
While the House’s 21st Century Cures bill would increase biomedical research funding at the NIH by $8.75 billion over the next five years, the Senate bill would increase it by $26.6 billion.
Durbin’s bill has not picked up substantial GOP support, mainly because of its cost. But the 21st Century Cures Act is likely to stumble in the Senate, especially since it includes language that would make permanent a prohibition on the use of federal funds for abortion services. For years, the ban has had to be extended on an annual basis in appropriations bills.
So there’s a stalemate – at least for now – on the effort to promote new cures, even though Congress has a strong interest in Congress doing so. The Senate Health, Education, Labor and Pensions (HELP) Committee has held several hearings over the past year on drug and device innovation and may produce a bill that is closer to the 21st Century Cures Act by the end of the year.
Meanwhile, Durbin has created a bipartisan Senate NIH Caucus to promote federal biomedical research, which counts Sen. Richard Blumenthal, D-Conn., among its 16 members.
Durbin says in 2012, 53 percent of all funding for basic research came from the federal government. Yet, he says, as a percentage of the total federal budget, the federal government spends two-thirds less on research and development today than it did in 1965.
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