HOUSE OVERWHELMINGLY PASSES BILL TO SPEED FDA DRUG APPROVALS
By Carolyn Johnson July 10
A bipartisan bill that would make significant changes to the process for developing new drugs and medical devices overwhelmingly passed the House in a 344-77 vote Friday morning.
The bill, called 21st Century Cures, was cheered by rare across-the-aisle support from politicians, with 230 co-sponsors nearly evenly split between Democrats and Republicans. The pharmaceutical industry, patient advocacy groups, and medical organizations also support the bill, which calls for an additional $8.75 billion for the National Institutes of Health.
"This bill is about making sure our laws, regulations, and resources keep pace with scientific advances," House Energy and Commerce Committee chairman Fred Upton (R-Mich.) said in a statement.
The bill tries to address the impatience that stems from a major societal problem: despite billions of dollars of research into diseases that range from common cancers to the rarest genetic diseases, we still lack treatments for thousands of conditions. Many of its provisions seek to make the drug approval process less burdensome.
But its laundry list of provisions that tweak the process for approving new drugs or devices have raised significant concern from industry watchdogs and physicians who say the legislation is aimed more at helping drug and device companies than patients. Critics say the bill's regulatory alterations do not address the real problem with the development of new therapies and could lead to the approval of treatments that don't work and could even harm vulnerable sick people.
"The bill unfortunately offers a horse trade," said Vijay Das, a healthcare policy advocate at Public Citizen, a patient advocacy organization. "It increases funding for the world-renowned NIH in exchange for providing perks for the pharmaceutical and medical device industries."
The White House issued a statement that welcomed the additional support for biomedical research, but flagged the part of the bill that would extend a company's exclusive right to market brand name drugs if they are repurposed to treat a rare disease. The Congressional Budget Office estimated that this could increase spending on prescription drugs by $869 million from 2016 to 2025.
"The Administration remains concerned about extending drug exclusivity... and how this provision will affect drug costs," the statement said.
Brielle (left) and Brooke Kennedy of Mattawan, Mich. have Spinal Muscular Atrophy and helped trigger the 21st Century Cures legislation. (Courtesy of Sarah Kennedy)
The passage of the bill brings hope, however, to many people who have watched desperately ill family members struggle with devastating diseases that may have no effective treatments or cures.
Sarah Kennedy was glued to the television in Mattawan, Mich. as the House took its vote. Her experience as a parent illustrates the powerful emotional pull of a bill that promises to increase the development of new treatments for diseases that today have little hope. For years, Kennedy has navigated the frustration and uncertainty of a terrifying disease with no known cure. Her daughters, Brielle, 7, and Brooke, 6, both suffer from Spinal Muscular Atrophy -- a genetic disease that attacks the nerve cells behind voluntary movements. The disease has no cure and eventually robs children of the ability to walk, eat, and breathe.
In many ways, Kennedy's daughters have a life like other kids. Brielle and Brooke are happy and joyful. They love everything Disney, navigate the world in their wheelchairs, take the bus to school, and have tons of friends.
But they are also taking a drug originally developed and approved for seizures that comes with serious potential liver side effects. Although the drug, valproic acid, hasn't been proven and approved for their disease, it seems to help both girls retain their strength and ability to control their movements.
Brielle "had trouble even lifting arm above her head and after we started her on the valproic acid, she could lift her hand above her head," Kennedy said. "We felt right then, 'OK. We can see a difference. Let’s get on it, let’s try it.'"
For Kennedy, as for any parent, it's hard to give children drugs that carry major risks or come with side effects. As a baby, Brooke was on a clinical trial for an experimental drug that tasted so bad that she stopped eating. Eventually, a feeding tube need to be placed. But in their quest to find ways to give their daughters extra strength and more time, the Kennedys are willing to accept far more risk than if they were simply trying to treat an ordinary disease of childhood, like an earache or sore throat.
"When you’re given life or death, you want to do anything you can to give them life," Kennedy said.
While these types of patient experiences are powerful, policy experts worry that such arguments will be used to erode a carefully-wrought standard of evidence that today helps to avoid the tragedies of the past, such as the birth defects caused by thalidomide in the 1960s and infertility caused by the Dalkon intrauterine device in the 1970s.
"We share Congress’ desire to increase funding for NIH, but there are dangerous parts of this bill that many members of Congress did not fully understand," Diana Zuckerman, president of the National Center for Health Research, a nonprofit think tank, said in a statement. "As often happens, well-funded pharma lobbying was more effective than experts’ concerns about patient safety."
One part of the bill that has generated discussion among physicians are changes intended to speed the development of new antibiotics. These allow evidence that would be considered preliminary data today to be considered in the approval of new drugs for a limited population of patients. It also includes a provision that would incentivize the use of new antibiotics by increasing the payments hospitals receive from Medicare for the drugs. To many scientists, this was seen as a problem because overuse of antibiotics has created a crisis of infections that aren't vulnerable to any treatments.
Elizabeth Jungman, director of public health programs at the Pew Charitable Trusts, said that allowing drug approvals for narrower groups of people -- for example people with drug-resistant pneumonia instead of all pneumonia patients -- could help solve that problem. She added that specific language in the bill ensures that the safety and efficacy standards are not lowered for antibiotics.
An amendment that passed Thursday, proposed by Rep. Louise Slaughter (D), called for a study on whether those extra payments resulted in the development of pathogens resistant to those new drugs.
"It's foolish to pay someone to create more antibiotics if they get frittered away the same way," Slaughter said in an interview.
The Senate is working on a parallel track and the Health, Education, Labor and Pensions Committee headed by Sen. Lamar Alexander has held four hearings on the initiative. The hope is to have legislation through the committee by the end of the year, a committee aide said.
Carolyn Johnson is a reporter covering the business of health. She previously wrote about science at The Boston Globe.