Newly passed 21st Century Cures Act is no panacea
Published 6:00 am Sunday, December 18, 2016
BY TRUDY LIEBERMAN
Will patients benefit from the passage of the 21st Century Cures Act? After listening to politicians and reading the headlines, most people might think it’s the best thing ever to come along for patients.
“A new day for medical research is on the horizon,” proclaimed Rep. Fred Upton, the outgoing chairman of the House Energy and Commerce Committee which had just won a major victory last week. “The House and the Senate have passed this bipartisan legislation which will ensure our health system can keep pace with incredible advances in science and technology,” Upton said in the GOP’s weekly radio address. “We needed to do better. And with 21st Century Cures, we will.”
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Media headlines mirrored Upton’s victory declaration. Headlines like this from the Wall Street Journal, “House Passes Health Bill to Speed Drug Approvals, Boost Biomedical Research” seemed to say it all: faster drug approvals, more money for research, less pesky regulation.
Or was there more to the story? Not everyone thinks the Cures Act will be wonderful for patients. But their voices were drowned out in the slick public relations campaign Upton’s committee waged over the past few years using some 200 or so patient advocacy organizations to push for the bill. Most of those groups have strong ties to the drug and medical device industry.
A recent study by Dr. Vinay Prasad, an oncologist at Oregon Health Sciences University, found that three-quarters of 68 cancer advocacy groups he studied disclosed sponsorship from pharmaceutical companies. Some groups received money from as many as 16 or 17.
Prasad told me, “Ten years from now, someone with a cancer diagnosis will be worse off with this bill. People will be exposed to more things that don’t work.” Prasad and others say the Cures Act actually lowers the regulatory standards for drugs and devices. It’s a step backward for patients.
In a nutshell the Act turns current regulatory practice on its head.
Randomized trials, the gold standard for medical research, may disappear if a drug company wants to sell a medicine for a different condition than the one it’s already approved for. Instead it can use “real world evidence” to show the drug also works for a new indication. Such “evidence” could be observational studies, which are less reliable than randomized trials, but are cheaper and take less time.
The FDA can use patient experience to inform its regulatory decisions – information about the impact of a disease or related therapy on patients’ lives. The data can come from patients, family members, caregivers, patient advocacy organizations, disease research foundations and drug manufacturers.
The agency can approve new drugs on the basis of data summaries rather than requiring the FDA to independently analyze study results for a new drug indication. Drug makers would have to submit all their data, but the FDA would not have to review it.
And although the bill authorizes billions for new medical research at the National Institutes of Health (NIH), the funding is not guaranteed, and the NIH would have to fight Congress for the money.
The Cures Act is the culmination of a 20-year effort by the drug industry and Beltway think tanks to loosen standards and permit new uses for drugs already approved in order to expand their markets. It can be traced back to the 1997 FDA Modernization Act, which was then – as the Cures Act is now – sold on the promise of getting cures to market faster. The 1997 law loosened regulatory standards and reduced the number of clinical trials needed for drug approval.
Are the changes brought about by the earlier law a harbinger of what’s to come?
What have been your experiences with new drugs? Write to Trudy at email@example.com