After two years of negotiations, the U.S. House of Representatives and Senate passed the 21st Century Cures Act on December 7, 2016.
More Research, Faster Approvals
Supporters say the Cures Act will speed access to new drugs and devices, in part by allowing clinical trials to be designed with fewer patients and cheaper, easier-to-achieve goals. Critics, including Senators Elizabeth Warren and Bernie Sanders say the bill gives massive handouts to the pharmaceutical industry and will lower standards for drug and medical device approvals. The Senate passed the bipartisan bill on a 94-5 vote.
$6.3 Billion
The $6.3 billion legislation, sponsored by Representative Fred Upton, authorizes $4.8 billion for the National Institutes of Health and $500 million to the FDA. It also calls for $1 billion over two years to battle the opioid epidemic. There is also $1.8 billion in funding for Vice President Biden’s Cancer Moonshot.
Funding for programs in the bill must still be appropriated by separate funding bills. However, the changes to the clinical trial process will be set in stone regardless of whether the funds are appropriated. The FDA will now be able to give greater weight to “real world” evidence gathered outside the framework of a randomized, controlled clinical trial. Patient input will be formally incorporated into the FDA’s review process.
Funding for the Cures Act will be offset by reductions in some Medicaid payments and through the sale of oil from the Strategic Petroleum Reserve.
“We are on the cusp of something special— a once-in-a-generation opportunity to transform how we treat disease, ” said Representative Upton.
AdvaMed, the medtech trade group, praised passage of the bill.
Statnews.com listed the winners and losers of the legislation.
Winners:
Drug Companies
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.
Medical Device Manufacturers
According to AdvaMed, 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.
National Institutes of Health
The 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.
“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.
Losers:
FDA
The $500 million designated for the FDA is meant to pay for the agency’s new responsibilities under the Cures Act. But there is not much 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 Robert Califf, M.D., has said pose a serious problem.
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.
Regenerative Medicine
The final Cures Act draft does not allow new regenerative medicine products, which include stem cell therapies, to skip 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.
Congressional leaders tried to include provisions which 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 White House supported the Cures Act and the President is expected to sign it before leaving office.
Click here for a detailed look at what the Cures Act will mean for the FDA.
