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What You Need To Know About Congress’ Effort To Speed Up Drug Approval

CREDIT: SHUTTERSTOCK
CREDIT: SHUTTERSTOCK

The House recently passed the 21st Century Cures Act (H.R. 6) by an overwhelming 344–77 vote. This legislation — which aims to improve the Food and Drug Administration’s drug and medical device development system and spur the creation of treatments for thousands of conditions — has support among a bipartisan coalition of lawmakers, pharmaceutical companies, patient advocacy groups, and medical organizations. Still, some detractors have expressed concern about Big Pharma’s growing influence and the potential of deadly mistakes.

Here’s what you need to know about what’s in the bill and where advocates stand:

What would the 21st Century Cures Act do?

Under the 21st Century Cures Act, the Food and Drug Administration would be able to directly incorporate the experiences of patients with certain disorders and their reactions to new therapies in a manner that protects privacy. The bill also advances the development of personalized medicine and provides guidance on the use of biomarkers, a drug development tool that can assess the effects of new treatments earlier. Drug developers will also receive economic incentives for the treatment of rare diseases. The legislation would also create an “Innovation Fund” to allow for more investment into younger scientists and protect against congressional overspending with an annual reserve of $1.75 billion to the FDA and $110 million to the NIH for five years.

The House Committee on Energy and Commerce says the 21st Century Cures Act will reduce the deficit by $500 million.

How did we get to this point?

Drug recalls and allegations of misconduct within the drug approval process in recent years has highlighted shortcomings of the national food and medicine regulatory system, leading critics to agree that the drug approval process needs to be expedited. Efforts to create patient-tailored treatments for chronic illnesses further strengthened this resolve.

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Last April, the House Energy and Commerce Committee kicked off its 21st Century Cures Initiative as part of an effort to speed up the Food and Drug Administration drug approval process. Subsequent roundtables, congressional hearings, and white papers have brought forth a nearly 200-page document that delves into matters of NIH funding and the efficiency of research programs. The original draft included an amendment to a federal law that would facilitate accelerated drug approvals. Legislators later added a section about precision medicine in which patients could disclose health information for the purposes of scientific research. Patients would also be able to opt into experimental treatments under what’s called the “compassionate use” provision.

The bill found support among various groups. Early on, experts predicted the FDA would most likely show support for the changes since they fell in line with its goals to better engage patients in its research. Legislators too have weighed in, mulling over the use of biomarkers as tools in accelerating the approval of new products to high-need populations. Calls to strengthen quality of research gained support, particularly with provisions in 21st Century Cures that proposes the broader use of innovative statistical methods and better use of the Sentinel system — a database that identifies adverse events with medical products.

But no group was perhaps happier than the family members of those suffering from chronic illnesses. For instance, Sarah Kennedy of Mattawan, MI told the Washington Post that, if passed, the 21st Century Cures bill would make the development of a treatment for her daughters’ Spinal Muscular Atrophy all the more probable. Right now, both girls, ages 6 and 7, receive a drug approved for seizures that strengthens them and helps them control their muscle movement.

Who’s opposed to this bill?

Critics say that the 21st Century Cure Act does control quality of new medicines. They assert, rather, that it enables pharmaceutical misconduct and could lead to the release of treatment that harms vulnerable patients.

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In his recent New England Journal of Medicine piece, Dr. Jerry Avorn, professor of medicine at Harvard Medical School, described the quicker clinical trials as “problematic” and implored lawmakers to reconsider the use of “evidence from clinical experience” in place of randomized, controlled trials for new drug approvals. The White House, too, has weighed in with a statement that derides a provision allowing pharmaceutical companies to market a drug’s brand name when it’s marketed to treat a different disease. Such a move, the Congressional Budget Office estimates, could increase prescription drug spending by $869 million between 2016 and 2025.

Amid the threat of antibiotic-resistant superbugs, some people have also pointed to parts of the bill that incentivize the use of new antibiotics by increasing Medicare payments to hospitals for their use. Rep. Louise Slaughter (D- NY) proposed an amendment calling for a study of whether those incentivize induced the creation of more pathogens.

The funding strategy for the 21st Century Cures Act has also raised eyebrows. Legislators said they want to pay for it through the sale of 64 million barrels of crude oil from the Strategic Petroleum Reserve, reducing it by 10 percent.

What’s next?

Now that it’s passed the House, the 21st Century Cures Act heads to the Senate, where it may encounter opposition, particularly from fiscally conservative lawmakers and those concerned about the effects of looser regulation. Shortly after its passage in the House, health care expert Alex Lash predicted more changes to come for the bill on its way to the White House, noting that the original version had been reduced by 200 pages.