The 21st Century Cures Act, legislation that's been alternately hailed as a game changer for medicine and a gift to the pharmaceutical industry, is set to go to a vote in the House Wednesday, Nov. 30.
An earlier version of the bill, at 352-pages, gained House approval last year on a 344-77 vote but was delayed in the Senate. The latest version was rolled out this past weekend by Rep. Fred Upton R-Mich. and Senator Lamar Alexander R-Tenn. with an eye towards passing the bill before the end of the year. Upton and Lamar called the legislation "a transformational bill to bring our health infrastructure light years ahead."
The 996-page bill aims to pave the way for new drugs to gain faster approval from the Food and Drug Administration and provides $4.8 billion in new funding for the National Institute of Health, $500 million to the FDA as well as $1 billion to fund prevention of opioid abuse, and issue that has become critical in states like West Virginia, Kentucky and New Hampshire.
It also alters some of the medical device and drug approval and regulation process so that treatments could come to the market more quickly, in some cases relying more on anecdotal evidence rather than hard scientific proof, especially when there is an unmet medical need. It also opens the door to pharma companies receiving more market exclusivity for existing drugs that could be used for patients suffering from rare diseases, delaying the introduction of generic versions of drugs that are usually cheaper for patients to buy.
Republicans wanted the bill to include a simpler drug approval process for the FDA, while Democrats wanted more funding for the NIH and the FDA.
Drug companies have long charged that gaining approval for a new drug can take a decade and cost more than $1 billion. Likewise, patient advocacy groups have become more vocal in their demands that the FDA find ways to bring drugs to market more quickly, especially in cases where patients have no approved drug to treat what are in some cases fatal diseases.
While Senate Majority Leader Mitch McConnell, R-Ky, has called the 21st Century Cures the most important legislation of the year and pushed hard to get it into the approval process, his enthusiasm is not shared by all. His senate colleague Elizabeth Warren, D-Mass, said on Monday that the bill was simply a way to funnel money to the pharmaceutical industry, terming the cash going to the NIH and FDA a "fig leaf."
Senator Chuck Grassley, R-Iowa, who doesn't have much common political ground with Warren, is opposed to the legislation as well. Grassley has threatened to hold up a vote in the Senate, which is now set for a vote by unanimous consent because he objects to disclosure requirements that would shield doctors from having to disclose publicly any payments the receive from drug companies or medical device makers. Grassley, an advocate for transparency in the health care sector, said in a statement that "With taxpayers and patients paying billions of dollars for prescription drugs and medical devices, and prices exploding, disclosure of company payments to doctors makes more sense than ever."
Former presidential candidate Senator Bernie Sanders, I-Vt., said in a statement Nov. 29 "This is a bad bill which should not be passed in its current form. It's time for Congress to stand up to the world's biggest pharmaceutical companies, not give them more handouts."
The election of President-elect Donald Trump may prove to be a wild card in terms of the fate of the 21st Century Cures Act because Trump has pledged to dump Obamacare and eliminate red tape where medical issues are concerned. Democrats may withhold their blessing for a bill that tells the FDA to work faster if Trump may begin cleaving regulations anyway.
Politics aside, there are others concerned with the bill. Aaron Kesselheim, an associate professor of medicine at Harvard Medical School, did a paper on the 21st Century act last year and questions whether the legislation could weaken the FDA's ability to regulate the drug industry properly.
At the same time, the bill has plenty of advocates. The Center for Responsive Politics reports that 420 different organizations and companies lobbied on the 21st Century Cures Act, including medical insurers such as Blue Cross, pharma companies like Roche Holdings and Amgen Inc., industry advocates like Pharmaceutical Research & Manufactures of America, research facilities such as Indiana University Health and patient advocate organizations like Autism Speaks.
Supporters of the legislation have said that the FDA is too slow to approve new drugs and that is why the bill needs to be approved. But a January report by the FDA showed that the regulator approved 45 novel drugs in 2015, a pace that is well ahead of approvals from 2006 to 2014 which averaged just 28 drugs per year.
Accelerated approvals have not always spelled success for patients receiving the drugs. Kesselheim did a pair of studies last year that showed only a third of the drugs that received faster approvals were the first to treat diseases or had an advantage over drugs already on the market. Kesselheim said the accelerated approval process was being driven by drug availability and not innovation or advancement of treatment.
In recent months, Sarepta Therapeutics (SRPT) gained approval for Exondys 51, a drug to treat a form of muscular dystrophy. Bristol-Myers Squibb Co. (BMY) received an accelerated approval for Opdivo and Merck & Co. (MRK) got the OK for Keytruda. Both drugs are used to treat lung, head and neck cancers.
So far this year, the agency has signed off on at least 15 enhanced approvals in the oncology and hematology sector alone. Generally, when the FDA gives an accelerated approval, it's for drugs that treat serious or life threatening diseases and provide an advantage over existing meds, or where there is no approved treatment available. Accelerated approval candidates are also subject to additional post-approval testing.
The post-approval process may already have issues that a 21st Century approach may exacerbate. An audit last year by the U.S. Government Accountability Office found that expedited drug approvals could be putting patients at risk because of missing or wrong post-approval data.