How American Families Might Be Helped

“There has to be something that will work.” That phrase is so often said by families facing a devastating or life-threatening illness. When few options seem left to save lives, people hope for anything new that might come along fast enough to make us well again.

New drugs often take decades to make their way through the approval pipeline — at least that’s long been the case.

The 21st Century Cures Act that passed in the Senate on Wednesday afternoon aims to speed up the drug and device approval process, partly by allowing clinical trials to be designed with fewer patients and cheaper, easier-to-achieve goals. The Senate passed the bill 94 to 5.

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The House passed the bill late last week, by a vote of 392 to 26. It now goes to President Obama to sign.

“For the second consecutive year, the Senate is sending the president another Christmas miracle for his signature,” Sen. Lamar Alexander (R-Tenn.) said in a statement. “Last year, it was the Every Student Succeeds Act, and this time, it’s the 21st Century Cures Act — a bill that will help virtually every American family.”

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Sen. Ted Cruz (R-Texas), said in a statement about the bill: “One of the most powerful weapons we have to fight disease is American ingenuity, and I believe the 21st Century Cures Act will help our nation harness its resources to better seek and realize lifesaving cures and therapies. While this bill is far from perfect and Congress should continue to improve on its reforms, this legislation represents an important first step forward in removing barriers to effective medical treatment.”

He added, “I am encouraged that a seed for greater reform of the federal role in research and medical advances has been planted and believe this bill lays an important foundation for next year.”

Critics aren’t so sure. They say the legislation gives massive handouts to the pharmaceutical industry and lowers standards for drug and medical device approvals. “This gift — which 1,300 lobbyists, mostly from pharmaceutical companies, helped sell — comes at the expense of patient safety by undermining requirements for ensuring safe and effective medications and medical devices,” consumer watchdog Public Citizen said in a statement.

The act, sponsored by Rep. Fred Upton (R-Mich.), authorizes $4.8 billion for the National Institutes of Health and $500 million for the Food and Drug Administration.

It also calls for $1 billion over two years to battle the nation’s opioid epidemic.

“I believe this bill could make a difference if it is spent wisely and funding goes to the correct places,” said Dr. David Gortler, a pharmacology expert, drug safety expert, and FDA policy expert at FormerFDA.com.

“Right now, the NIH and FDA have a bloated number of staffers in some departments and have critical shortages in other areas. The FDA in particular has plenty of existing problems they need to fix before they can go on to expediting the drug approval process.”

Gortler told LifeZette he is leery about trying to expedite the time-tested, time-intensive method of testing drugs for safety and efficacy; he said the bill was heavily sponsored and influenced by big pharmaceutical companies and device manufacturers. He believes it is self-serving: They would be able to get their products to market faster, even if the bill does benefit the greater good.

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“I think Congress’ heart is in the right place for wanting to increase the budget for curing diseases. But dramatically speeding up the FDA’s drug approval through the use of surrogate endpoints and biomarkers alone has been shown to be a bad idea,” said Gortler.

Critics note the money described in the bill must be appropriated by separate funding bills and that the money may ultimately never materialize, as Reuters and others have reported. Yet the changes to the clinical trial process, something long sought by the drug industry, will be set in stone regardless of whether money for the research projects is forthcoming.

Among those changes: Greater prominence will be given to “real-world” evidence gathered outside the framework of a randomized, controlled clinical trial, the gold standard for determining whether a drug is safe and effective. Such evidence could be much easier for drug companies to collect.

“The passing of 21st Century Cures Act is a show of extraordinary bipartisan unity after a divisive election that should be celebrated,” Ellen Sigal, chair of the patient advocacy group Friends of Cancer Research, told Reuters.