For the first time since 1997, the Food and Drug Administration is facing a historic overhaul to change the way it approves drugs and medical devices.
On May 21, the House Energy and Commerce Committee passed the 21st Century Cures Act 51-0, sending it to the House for a full vote. If it passes the House, Senate and is signed into law, the bill’s co-sponsors, Rep. Diana DeGette, D-Colo., and Rep. Fred Upton, R-Mich., think the bill will accelerate the progress of new cures.
The bill also increases the budget of the National Institutes of Health, currently at $30.3 billion per year.
“I can’t think of anyone who goes through life without having a close friend or loved one face a serious illness,” DeGette says. “For all of the remarkable progress we have made to treat medical conditions, far too many patients lack a treatment or cure for the disease they are fighting or will fight soon.”
There are three ways this bill may change medicine as we know it:
Increased NIH ‘Discovery’ Spending
“For years, NIH has faced an erosion of and outright cuts to its research budget, reducing our ability to learn new scientific insights that can lead to medical advancements,” DeGette says.
A large portion of the bill seeks to increase that budget, providing $10 billion in additional funding to NIH over the next 10 years, according to the Regulatory Affairs Professionals Society.
The money is intended to fund more emerging young scientists in drug discovery research and provide more money for those research efforts across the board. The hope is that by providing more grant money for the first stages of development, more novel drug therapies will see FDA approval down the road.
“Last fall, NIH Director Dr. Francis Collins told me that if not for past budget cuts, we would already have developed an Ebola vaccine,” DeGette says.
While we can’t help the patients who’ve already suffered from recent outbreaks, she says she hopes we can prevent similar scenarios from happening again.
Speeding up Drug and Device Approvals
In addition to providing new funding for NIH, the bill would also provide an additional $550 million to the FDA to fund changes in how it approves drugs. By and large, the bill seeks to speed up the general approval process so that patients can receive therapies and devices sooner.
Before gaining approval, manufacturers currently must prove through clinical research that any new drug is both safe and effective in large clinical trials with control groups. For drugs intended to treat a rare disease, however, getting enough patients to participate in large-scale trials is particularly difficult. One provision would allow for surrogate endpoints to be used for approval of some drugs, which means they could be approved based on smaller trials.
Though that would speed up the development of some drugs — particularly antibiotics and cancer medications — opponents say it’s unsafe. “The danger when you’re using only small trials or surrogate endpoints is that you get results that indicate a product is safe and effective, and they turn out not to hold true,” says Sarah Sorscher, an attorney with Public Citizen’s Health Research Group. Public Citizen is an advocacy group that fights on behalf of public rights. “It could be a statistical fluke, or what you’re using might not predict real clinical benefit.”
Increased Access to Drugs
You may have seen a news story or an online petition where a patient with a chronic disease seeks access to a potentially life-saving but experimental treatment. The treatment is often approved for another condition, but sometimes the drug in question merely shows promise in clinical trials.
As a result, several states have passed “Right to Try” laws, which are intended to give chronically ill patients easier access to these drugs that show promise for their condition. The new bill makes that easier, especially for fast-tracked drugs, which include novel or breakthrough therapies and qualified infectious disease products, such as chemotherapies and antibiotics.
Not everybody thinks that’s a good idea; the notion of reducing standards to speed up drug production has Public Citizen worried. “We have concerns antibiotic approvals are already too fast and evidence is already too weak. We think it’s premature to be further reducing the standards,” Sorscher says.
But Degette is unfazed, and says safety won’t be compromised. “I have been working with and listening to stakeholders from the FDA, patient groups, industry leaders and many others,” she says. “When it comes to upholding the FDA as the leader in patient safety, the agency’s biggest needs are greater scientific expertise and additional resources. The 21st Century Cures Act provides more of both.”
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