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Sheila Kaplan, STAT
Sheila Kaplan, STAT
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WASHINGTON — Sponsors of the 21st Century Cures Act call it a win-win: a fusion of funds for medical research with new rules that direct the Food and Drug Administration to approve drugs and devices with greater urgency. There’s also money to help states fight the opioid epidemic, and a boost for mental health care.
But this is Washington, so even this bipartisan bill, which the House of Representatives approved in a landslide and which the Senate is expected to pass Tuesday, leaves a trail of winners and losers.
Winner: Drug companies
More than 1,300 lobbyists roamed the halls of Congress on the Cures Act, and disclosure reports show most of them were working for pharmaceutical companies.
Their work has paid off.
Among other measures, the legislation calls for the use of “data summaries” to support the approval of certain drugs for new indications, rather than full clinical trial data. The Cures Act will also allow drug companies to promote off-label uses to insurance companies, allowing them to expand their markets.
They did not get everything they asked for, however. Among the provisions that the Democrats beat back was longer market exclusivity for certain drugs.
READ MORE: House approves the 21st Century Cures Act, sending landmark bill to Senate
Winner: Medical device manufacturers
According to the Advanced Medical Technology Association, which represents 300 medical device companies, the Cures Act builds on FDA’s current programs to allow a quicker path for breakthrough medical technologies for patients with life-threatening or irreversibly debilitating diseases or conditions, and limited alternatives.
“Passage of this important legislation is a milestone in improving the innovation ecosystem for medical technology and ensuring the availability of new lifesaving, life-enhancing devices and diagnostics for patients,” said the group’s president, Scott Whitaker.
Critics of the measure, such as Public Citizen’s Health Research Group, say the designation of “breakthrough” devices is too broad, and could lead to clearance of devices that aren’t ready for the market.
Winner: National Institutes of Health
An earlier House version of the legislation called for the NIH to get an $8.75 billion boost in its budget. The current legislation includes just more than half that amount for the agency: $4.8 billion. But, with an incoming president who has called the nation’s medical research agency “terrible,” it could have been worse.
The landmark legislation provides funding for three signature Obama administration research programs, over the next 10 years: the “moonshot” against cancer, the BRAIN Initiative, and the Precision Medicine Initiative.
READ MORE: 21st Century Cures Act will distort the meaning of ‘FDA approved’
The $500 million designated for the FDA is meant to pay for the agency’s new responsibilities under the Cures Act. There is virtually nothing in it to improve some of FDA’s longtime problems.
The Government Accountability Office considers two of the FDA’s areas of focus especially “high-risk”: food safety and post-approval oversight of medical products that went through the fast track.
There’s also no money in the Cures Act for aging, deteriorating labs, outside of headquarters, which Commissioner Dr. Robert Califf has said pose a serious problem.
Winner: ‘Real world evidence’
The Cures Act directs the FDA to evaluate the use of “real world evidence” for approval of new indications for FDA-approved drugs. Proponents see this as a way to apply useful and timely information learned from observational studies, patient input, anecdotal data, and other research to drug and device approval.
Loser: Randomized clinical trials
Currently the gold standard for testing drugs and devices for safety, the adoption of real world evidence standards may indicate that randomized clinical trials will become less important for drug and device approval.
Winners: Representatives Fred Upton (R-Mich.), Diana DeGette (D-Colo.) and Tim Murphy (R-Pa.)
For Upton, who must step down in January as chairman of the House Energy and Commerce Committee, passage of the Cures Act has been a near obsession. For more than three years, Upton, along with DeGette, has herded patient advocacy groups, the drug and device industry, and even rock star Roger Daltrey to support his cause. The payoff: a 392-to-26 vote.
Upton also got a bonus: More than $1.3 million in donations from health care political action committees during the time he was crafting the bill.
Murphy, a psychologist, is an unexpected victor. Merely days before the vote, the House leadership attached his mental health legislation to the Cures package.
Dr. Maria A. Oquendo, president of the American Psychiatric Association, praised the mental health provisions in the Cures Act, and said it will greatly benefit patients. The legislation includes money to create suicide-prevention programs and to improve mental health services for children. It also allocated the $1 billion in state grants for the opioid epidemic, and seeks to strengthen laws that require the government and insurers to treat mental illness like other diseases.
“Congress has finally come together in a bipartisan effort to bring serious mental illness out of the shadows,” Murphy said. “It is my pledge to the patients and families in crisis that I will never stop fighting to deliver treatment before tragedy for those most in need.”
READ MORE: The Who’s Roger Daltrey: Teens with cancer deserve better
Winner: Senator Lamar Alexander (R-Tenn.), chairman of the Committee on Health, Education, Labor, and Pensions
Since July 2015, when the House passed a draft of the Cures Act and sent it to Alexander’s committee, the Tennessee lawmaker has had to overcome enormous opposition from both Democrats and Republicans.
His GOP colleagues vowed to reject any legislation that cost money — no matter how noble its goal — unless the cash was cut from another program.
The Democrats did not view the Cures Act’s main purpose — “acceleration” of FDA’s work — as especially noble. Most of them believe the agency already works fast enough, perhaps too fast, and needs to maintain its status as the global regulatory leader in patient protection.
So Alexander devised a plan: He chopped the Cures Act into small “medical innovation” bills, which had committee consensus. These ranged from measures to help NIH hire and retain scientists to developing better ways of managing electronic health records. Then, he took the hard stuff to the Senate and House leadership, who crafted the current compromise.
Loser: Senator Elizabeth Warren (D-Mass.)
Warren took to the Senate floor last week to rail against the Cures Act, saying it had been “hijacked” by the pharmaceutical industry. She said the legislation watered down safety requirements for new drugs and devices and then, as a trade-off, called for research funding — at levels that must be appropriated on an annual basis
“I cannot vote for this bill,’’ Warren said to a largely empty chamber. “I will fight it because I know the difference between compromise and extortion.”
Warren returned the next day with reinforcements: Sen. Dick Durbin of Illinois, and Sen. Jeff Merkley of Oregon, who agreed that the Cures Bill should be stopped. But she could not get majority support, even in her own party.
READ MORE: Elizabeth Warren rips 21st Century Cures bill and vows to fight it
Winner: Vice President Joe Biden
Since the death of his son, Beau Biden, from brain cancer, the vice president has devoted himself to a cancer “moonshot,” designed to speed cures by compressing 10 years of medical research into five.
The initiative faced an uncertain future under the new administration, but the Cures Act saves it, by authorizing $1.8 billion over five years to continue the program.
Congressional leaders praised Biden for his work on the bill, which the White House supports.
Winner: Michael Milken’s FasterCures and a host of patient advocacy groups
Michael Milken, the former junk-bond king turned philanthropist, has hosted retreats and receptions from Lake Tahoe, Nev., to New York, putting lawmakers in front of renowned scientists, patient advocates, Cabinet secretaries, and biomedical industry chief executives — all with the objective of winning support for medical innovation.
His Milken Institute center, FasterCures, was integral to developing support for the Cures bill. Other groups, including the American Cancer Society Action Network and Research!America, all supported the Cures Act — although some expressed disappointment with the reduced funding.
Losers: Consumer advocacy groups
Public Citizen’s Health Research Group and the National Center for Health Research campaigned hard against the Cures Act. Although they were able to make some changes, both organizations believe the Cures Act, as it stands, will endanger public health by weakening FDA standards.
Loser: Regenerative medicine
Senator Mark Kirk of Illinois, who had a stroke in 2012, introduced the REGROW Act earlier this year, to expand treatments for stroke victims and people suffering from Alzheimer’s, Parkinson’s and diabetes. Kirk’s legislation, which GOP leaders tried to include in the Cures Act, would have permitted the FDA to approve stem cell treatments conditionally, without a large, final-stage clinical trial that is usually required.
But many researchers argued that stem cell therapies are still in the early stages of safety and efficacy, and urged Congress to hold off giving FDA such a mandate.
The final Cures draft does not allow new regenerative medicine products, which include stem cell therapies, to skip the Phase 3 trials. But it does permit FDA to grant them accelerated approval if they can show that surrogate endpoints might indicate the drug works, subject to further study.
This article is reproduced with permission from STAT. It was first published on Dec. 5, 2016. Find the original story here.
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