A major proposed law that alters the way the Food and Drug Administration (FDA) approves drugs and medical devices has been wending its way through Congress since 2014. Momentum is building on Capitol Hill to pass the legislation in the current “lame-duck” session of Congress.
That shouldn’t happen.
The House passed its version of the legislation—the 21st Century Cures Act (hereafter the Cures bill) —in July 2015. The Senate health committee created and passed 19 related bills, under the banner “Innovation for Healthier Americans,” this past spring.
Sen. Lamar Alexander (R-Tenn) pushed, without success, to get the legislation to the Senate floor this past summer. Now, he and proponents—which include the pharmaceutical and medical device industries and dozens of research and patient groups who get support from those industries—seek to pass the legislation in the lame-duck session.
The election of Donald Trump may add to momentum to pass the legislation now, or undermine its chances both now and in 2017. It’s not yet clear.
Proponents’ main arguments for lame-duck passage are:
(a) The need to foster innovation at the FDA and bring new drugs and devices to market sooner;
(b) The House bill would increase funding for NIH, by $9 billion over 5 years;
(c) The Obama administration’s “cancer moonshot” and precision medicine initiatives dovetail with the intent of the House legislation, and funding for both may be folded into any final legislative package;
(d) Americans expressed anger at their government in the election and Congress should pass this bipartisan legislation to demonstrate that lawmakers can work together to get things done.
Some provisions of the House and Senate bills take steps in the right direction. And I join the overwhelming chorus of voices supporting increased funding for medical research and FDA.
But enough problems remain with this complex legislation to caution strongly against its hasty passage in lame-duck session.
First, a major premise of the legislation is, in fact, wrong—namely that the FDA stifles innovation and advances in treatment by approving drugs and devices too slowly compared to other countries. In fact, at least two-thirds of the novel drugs approved each year from 2012 to 2015 in the U.S. were approved here before they were in any other country. That’s because many newly approved drugs are currently being reviewed through existing FDA expedited review programs.
Ignoring those facts, the Cures bill and Senate legislation seek to speed-up drug and device approval by weakening many of FDA’s safety and effectiveness standards, without strengthening post-market tracking and evaluation of products. The Senate bills are a significant improvement over the House’s Cures Act in this regard, but they, too weaken current FDA approval standards.
The second reason to delay consideration of this legislation until 2017 is that must-pass FDA funding is up for renewal next year. This is the mandatory every-five-year process that sets “user fees” for companies applying for approval of prescription drugs and biologics, medical devices, generic drugs, and biosimilar treatments (“generic” versions of biologics).
And there’s a third, equally compelling reason to delay consideration to 2017. Over the past six months it’s become clear that the public is fed up with rising costs of prescription drugs and other medical products. A series of price hikes has amplified the outrage—most recently, over the 5-fold increase in the price of life-saving EpiPens.
A survey of 1,205 consumers in October by the Kaiser Family Foundation, for example, found that prescription drug prices are the public’s #1 health policy concern. Sixty-three percent of respondents agreed that the government should take action to lower drug prices.
Thus, if President Trump and newly elected lawmakers want to be responsive to one of the public’s main healthcare concerns—and demonstrate the ability to work together—they should commit to tackling the issue of prescription drug prices and costs.
Consumer groups weigh in
Three consumer coalitions wrote letters this month to Congressional leadership calling for consideration of the FDA legislation to be put off until next year.
- AFL-CIO and the American Federation of State, County and Municipal Employees joined the Center for American Progress to tell lawmakers: “there is no justification for moving forward with legislation that provides substantial benefits to the drug industry without asking for something in return.”
- 18 nonprofit organizations representing the Patient, Consumer, and Public Health Coalition expressed opposition to numerous provisions in the legislation and said it has the potential to raise drug and device prices.
- Public Citizen, Consumers Union, and 11 other groups criticized the legislation for lowering FDA standards.
I agree that spending is very likely to increase when standards for approval are lowered without ensuring that unsafe or ineffective (and thus money-wasting) medical products can be much more quickly taken off the market.
In addition, new funding for the cancer moonshot and precision medicine initiatives, while laudatory, could yield new and expensive drugs that are not as rigorously evaluated for effectiveness before approval and not thoroughly assessed after they are approved.
A recent study of cancer medicines painfully illustrates that dilemma and the need for more linkage between the results a drug yields and its value in the marketplace—one of the major policy solutions under discussion to restrain drug prices and spending.
Researchers at the National Cancer Institute and Oregon Health & Science University reviewed all the cancer drugs approved by the FDA from 2008 to 2012. Two-thirds were approved based on biomarkers or other surrogate endpoints, usually tumor shrinkage over a relatively short period of time. As a condition of approval, the makers of these medicines were required to conduct post-market studies to assure their medicines were actually extending lives.
The answer, unfortunately: many were not. After a median follow-up of 4.4 years, only five drugs that had been approved on the basis of a surrogate endpoint were proven to help patients live longer. Eighteen drugs (50%) failed to extend life and 13 (36%) have unknown impact on survival because no data on them are publicly available.
The National Center for Health Research looked at the cost of those cancer drugs and found something that lawmakers and the Trump administration need to know: new cancer drugs that are not proven to work cost just as much as the ones that are effective – up to $170,000 per patient. Meanwhile, the ineffective cancer drugs remain on the market and Medicare and insurers are still paying for them.
The new administration and Congress should combine the elements of the Senate bills that improve the quality of medical care with new measures that make substantial progress on constraining drug prices and spending. Initial focus should be on three issues:
- Price gouging by companies that have created new “profit centers” by buying, repurposing or repackaging older medical products and boosting their prices 3 to 10-fold or more;
- The rising cost of new specialty, biologic, and cancer drugs that have not proven to be as effective or more effective than older drugs, and which are leading to substantial increases in Medicaid and Medicare costs and private sector insurance premiums; and
- Rising out-of-pocket costs resulting from insurance coverage design that puts consumers at risk for hundreds or thousands of dollars in annual expenses for very expensive medicines.
FDA reform legislation presents a unique opportunity for a new administration and Congress to demonstrate good will and bipartisanship—and achieve an early win in 2017 for the American people and public health. Changes are needed, however, to make sure this legislation does more good than harm.
Diana Zuckerman is president of the National Center for Health Research in Washington, D.C.