WASHINGTON – Why is the nation's capital suddenly obsessed with biomedical innovation?
And why is the fruit of that obsession, the proposed 21st Century Cures Act, moving like a freight train through the usually deadlocked congressional process—even though some experts warn it could undermine the Food and Drug Administration's ability to protect the public from unproven and potentially unsafe new drugs and medical devices?
The House Energy & Commerce Committee is scheduled to hold a legislative markup session Thursday on the bill, which would overhaul federal regulation of prescription drugs and devices and provide significantly more funding for National Institutes of Health research. Congressional Democrats have jumped on the bandwagon, primarily out of eagerness to boost NIH support, which has slipped over the past decade.
Leaders of the Energy & Commerce Committee previously circulated a discussion draft of the 21st Century Cures bill that would raise NIH funding to $31.8 billion in fiscal 2016 and to $34.85 billion in fiscal 2018. Congress appropriated $30.3 billion for the NIH in fiscal 2015, and President Barack Obama requested $31.3 billion for fiscal 2016.
Consumer advocacy group Public Citizen called the NIH funding a horse trade “providing perks to the pharmaceutical and medical device industries to approve medications and devices faster based on weaker evidence.”
Observers say nearly every sector of the healthcare industry—even those that seem only tangentially related to the subject of the bipartisan legislation—has gotten involved in lobbying the bill. For instance, an earlier version of the House bill eliminated the requirement for medical manufacturers to disclose payments for doctors' continuing medical education programs.
“It became a Christmas tree,” said Billy Wynne, of Thorn Run Partners, who has lobbied the bill.
One lobbyist, who did not want to be named, said the bill's “popularity has grown commensurate to the attention it promises to the different industries represented.” The lobbyist added that “many people see it as the moonshot opportunity for lots of stuff” ranging from pharmaceuticals to genomics to lab testing to telehealth to dietary supplements.
According to the Senate's lobbying database, the number of groups listing 21st Century Cures as a lobbying issue has grown from 62 in the second quarter of last year to 222 in the first quarter of this year.
The feeding frenzy has attracted some unexpected groups in the fourth quarter of 2014, such as the American Association of Homeopathic Pharmacists and the Natural Products Association, which lobbies on dietary supplement issues. Daniel Fabricant, CEO of the Natural Products Association, said his group's lobbying was intended to get the association into the “four corners” of the bill. The bill “was still a great unknown,” he said.
His group hopes to get Congress to approve coverage for his member companies' products under tax-sheltered health savings accounts and flexible spending accounts.
Other groups appear to have lost their bid to get their pet items into the legislation, though it ain't over til it's over in congressional horsetrading. Lab-developed tests, for example, were one area that the House Energy & Commerce Committee held a hearing on but didn't include in the bill, said Lakshman Ramamurthy, director of Food and Drug Administration regulatory policy at Avalere Health. “We thought they'd do lab-developed testing but they haven't touched that,” Ranamurthy said.
What has given the bill legs is the enthusiastic support of patient advocacy groups. A December 2014 analysis by Avalere found that 43% of comments submitted during the drafting process were from patient advocacy groups, said Craig Burton, Avalere's director of life science.
There's a perception that biomedical innovation is not quite where it needs to be, said Dr. Gregory Daniel, head of the Brookings Institution's pharmaceutical and device policy group. “Particularly in the pharmaceutical and medical device areas, we're making a lot of benchtop, pre-clinical breakthrough discoveries,” he said. “But we don't see a lot of breakthroughs translating into therapies.”
Under the bill, the FDA would be required to increase its consideration of “real-world evidence” about the efficacy of drugs. Manufacturers would be able to expand a drug's indications based on observational data or registries indicating how it performs in the field.
Critics say these provisions would lead the government to endorse drugs for new uses without adequate testing for safety and efficacy.
The FDA also would be required to develop more surrogate endpoints to allow shorter clinical trials. For example, the FDA might accept evidence that a new cancer drug effectively shrinks tumors rather than requiring a longer and more expensive clinical trial showing reduced mortality.
The device industry also would benefit. A “breakthrough device” program would provide a faster path to market for devices that might substantially raise the standard of care.
A provisional section would expand the FDA's “humanitarian device exemption” for products that treat rare diseases. Under current law, a device treating a disease affecting fewer than 4,000 patients per year receives less regulatory scrutiny. The provisional section would raise that cap to 8,000. Dr. Jeff Shuren, the head of FDA's device division, praised the raise in the cap during an April 30 hearing.
Dr. Margaret Hamburg, who stepped down as FDA commissioner in March, has said the bill's approach is misguided. "Shortening review times is not going to create the scientific understanding and the research and development that needs to be done to translate exciting opportunities in science into new products," Hamburg told Modern Healthcare in a recent interview. “That is the wrong mechanism to achieve that goal.”
She also cautioned that placing additional requirements on the FDA in terms of its oversight responsibilities will increase the demands on an already overstretched FDA staff, hampering the review process. “Let's really focus on what's going to make a real meaningful, measurable difference in terms of what needs to be done and focus on those activities,” she said.
But some members of Congress argue that more regulatory flexibility is needed to incentivize and stimulate the drug and device industries. Sen. Richard Burr (R-N.C.) said during a May 12 event that for too many patients suffering from too many diseases, their choices were between “nothing and nothing.” Burr said the Senate will need to develop its own bill that “collapses” the FDA's product review times and the CMS' time to approve payment for products. He also said the FDA may need more resources.
“If you haven't got a functional FDA that's ready for the 21st century, if you haven't got a functional CMS … (innovation is) overseas before it's here,” he warned. Burr also mentioned a desire to revise intellectual property provisions, which might incentivize more development – particularly in areas without many treatments.
Earlier versions of the House bill would have let manufacturers apply for longer market exclusivity, thus giving them more protection from generic competition. Manufacturers would be able to apply for up to 15 years of patent protection for a product if it was intended to treat “one or more unmet need.” Currently new biologic drugs can receive the maximum of 12 years of market exclusivity, with five years for new chemical drugs and seven years for orphan drugs. The current status of those provisions, strongly supported by drugmakers, is unclear because they have drawn lots of criticism on the grounds that they potentially could boost healthcare costs.
Some experts question the need for revamping the FDA approval process. Ameet Sarpatwari, a research fellow at Brigham and Women's Hospital, said FDA already has speeded up its approvals and is now faster than European regulatory agencies. “Pharmaceutical companies are, in general, doing great,” he said. The product development problem, he said, is confined to certain areas, such as neurological and antibiotic therapies, for which a more targeted approach is needed.
Sarpatwari also is wary about the potential proliferation of alternative approval choices, which for example would allow FDA to approve drugs and devices based on biomarkers such as reduction of cholesterol rather than an improvement in patient outcomes such as a reduction in heart attacks. Such approaches have promise, he said, but policymakers need to be cautious. “There's an infectiousness of excitement,” he said. “It needs to be grounded with some data.”
But Sarpatwari's caution seems to be falling on deaf ears among both Republicans and Democrats. The major holdup for the legislation is money. The House draft allocates roughly $10 billion in new funding for the NIH, which Democrats pressed heavily for.
Some Democrats, backed by experts, also want additional resources for FDA, which they believe would otherwise be overstressed by meeting new burdens with their present workforce. That's a critique Burr agrees with, saying the FDA needs “a whole separate type of reviewer at the FDA” and that “it's incumbent on us to make sure they have the resources to recruit and retain the talent they need.”
But Rep. Diana DeGette (D-Colo.), one of the sponsors of the House bill, admitted during the most recent Energy & Commerce committee hearing that lawmakers still don't have a firm idea of how to pay for the legislation.
On Tuesday, Burr suggested that Congress could use “dynamic scoring” to cover the cost. He suggested that the biomedical innovations resulting from the regulatory reforms and increased research funding would reduce long-term government costs for treating expensive conditions such as Alzheimer's disease. Those savings should be calculated in establishing the bill's price tag, he argued.
“Wouldn't it be novel to do that?” he said. But, he added, “I don't think Congress is ready to have that debate.”
