By Peter Pitts In a rare display of bipartisanship in politically polarized Washington, 344 members of the House of Representatives voted in July to pass the 21st Century Cures Act.
This legislation has come at a pivotal time in medicine. It will help researchers get new and better treatments to patients sooner, which could significantly reduce healthcare costs. At a recent hearing, Sen. Ted Cruz, R-Texas, noted that the treatment of Alzheimer's and dementia could cost the United States $226 billion this year alone, yet funding for research into new treatments for these disease was only a quarter of that level. With more research dollars and access to better medications, people could be living more fulfilling lives at a lower cost to the government.
Over the next five years, the Food and Drug Administration would receive an additional $550 million under the bill. More than just a measure to provide needed funding, though, the 21st Century Cures Act would create a more streamlined drug approval system by revolutionizing procedures for clinical trials. The new rules would make bringing a medication to market less expensive, less time consuming, and more predictable. The result will be that medications will cost less and patients will have access to them sooner.
Currently, pharmaceuticals must undergo three phases of clinical trials before gaining FDA approval. The first phase tests for safety, the second for efficacy in a relatively small number of patients, the third for efficacy on a large scale. According to the Tufts Center for the Study of Drug Development, on average it costs over $2.5 billion and over ten years to get a new drug from the lab to patients.
The new legislation would allow the FDA to cut out the third phase of some clinical trials if the pharmaceutical showed significant effectiveness in phase II. Phase III trials make up 90 percent of the total developmental costs for successful drugs — and get new medications to people who need them sooner.
The legislation makes sure that streamlining the approvals process won't compromise patient safety. Pharmaceuticals that have been fast-tracked by the FDA would still need to meet appropriate safety standards.
The bill would also expand the use of "adaptive clinical trials"—in which researchers assess preliminary data at a specified point in the trial and can then modify its parameters to obtain better information more quickly.
A successful trial is obviously the goal. But there is also a huge advantage in letting proposed new medications that are turning out to be ineffective "fail faster." Rather than complete a failing trial, researchers can move on, potentially saving millions of dollars in reaching a foregone conclusion.
After all, when Thomas Edison was asked why he was so successful he commented, “Because I fail faster than everyone else.”
The age of big data is also coming to pharmaceutical research. The 21st Century Cures Act facilitates new uses for existing medications by allowing evidence from observational studies and patterns of real-world use in the approval process. This could open up the opportunity to monitor patient outcomes directly after phase I trials, possibly eliminating the need for phase II and III trials altogether.
Some companies are already implementing the power of data-sharing to improve patient outcomes. Biopharmaceutical research firm AstraZeneca has partnered with HealthCore, Wellpoint's data and analytics arm, to use real-world data to study the effectiveness of treatments for chronic and common illnesses. The data obtained from HealthCore will help to determine where AstraZeneca will put its R&D investment dollars.
There's no doubt that the 21st Century Cures Act would vastly improve the biopharmaceutical drug development process. In addition to funding more basic research and streamlining approvals, the bill would open up opportunities for further improvements as researchers realize the potential from sharing big data.
Now is the time for Democrats and Republicans in the Senate to follow the lead of their House counterparts and pass this revolutionary bill.
Peter Pitts is a former FDA associate commissioner.
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