Medical devices and drugs could reach your hands faster if a massive House bill is approved, but leading experts question whether they will be safe.
Two medical experts are concerned several provisions in the bipartisan 21st Century Cures Act may unnecessarily speed up product approvals at the expense of thorough reviews for safety and effectiveness.
“Changing the Food and Drug Administration’s standards to get more drugs on the market is inherently risky to patients,” Dr. Aaron S. Kesselheim, associate professor of medicine with Harvard Medical School, told the Washington Examiner.
Kesselheim, with Harvard professor Dr. Jerome Avorn, wrote an editorial in the New England Journal of Medicine that criticizes parts of the measure.
The bill was unanimously approved by the House Energy and Commerce Committee last month and awaits a vote from the full chamber. That vote could happen as early as next month, Committee Chairman Rep. Fred Upton, R-Mich., and Rep. Diana DeGette, D-Colo., told the Examiner.
A big impetus for the legislation is lawmakers’ worries that it takes too long to get a drug to market and is effectively impeding access to new cures.
But that may not be the case, Kesselheim and Avorn wrote.
More than two-thirds of new drugs are approved based on data from trials that last six months or less, according to the opinion piece, which cited a separate study of clinical trial data from 2005-12.
In addition, a third of new drugs are approved on the basis of a single trial, the average size of which is about 760 patients, the authors wrote.
“Once the FDA starts its review, it approves new medications about as quickly as any regulatory agency in the world, evaluating nearly all new drug applications within six to 10 months,” the article reads.
That evidence juxtaposes a study from Tufts University that found it costs more than $2 billion and takes a decade to get a drug to market.
Kesselheim and Avorn said the FDA already has several pathways that enable a company to get faster approval.
For instance, a company seeking approval for a new kind of cancer drug could get it reviewed in six months instead of 10 if it addresses an unmet need or is an improvement over an existing therapy.
Several provisions in the bill seek to enhance these powers.
If a company wants to get an existing drug approved for a new use, it could submit existing evidence on the current use of the drug instead of conducting a new clinical trial.
But that method is not as “rigorous or valid as randomized trials in assessing efficacy,” according to the article.
The bill also creates a new pathway for accelerated approval of a device that treats an unmet need. The pathway would encourage device makers to use smaller clinical trials than current guidelines, Avorn and Kesselheim wrote.
The authors were pleased, however, that the House gives the National Institutes of Health $10 billion and the FDA $550 million in new funding over five years.
Upton and DeGette vigorously defended the bill and rejected notions it would hoist unsafe devices or drugs onto the American public.
The lawmakers told the Examiner that they worked hard to ensure that the new powers helped to accelerate development while promoting safety.
“These concepts are not mutually exclusive,” they said in a joint statement. “This is why we took our time drafting the 21st Century Cures Act, worked very closely with the top officials at FDA and included leaders from across all facets of healthcare innovation in our effort.”
Many patient and doctor advocacy groups and pharmaceutical companies have applauded the measure.
The legislation contains a “number of critical provisions that can jumpstart new treatments for brain tumors and many other cancers,” wrote Sally Davis, CEO of the National Brain Tumor Society, in an editorial in The Hill newspaper.
Healthcare correspondent Paige Winfield Cunningham contributed to this report.
