(A version of this Health Alert was published by Forbes.)
President Obama has an opportunity to win a positive legacy in health care. Although his attempt at payment reform, Obamacare, has failed in public opinion, he is also encouraging important initiatives in medical innovation. The Cancer Moonshot and Precision Medicine Initiative represent investments in innovation that can bring big payoffs. However, they will not succeed fully unless the Food and Drug Administration allows patients access to new therapies. Legislation modernizing the FDA, the 21st Century Cures Act, is being fumbled inches away from the Congressional end zone. Presidential leadership is needed.
21st Century Cures combines a number of initiatives. The most important is fundamental reform of the FDA’s approval process for new drugs. The Act would require the FDA to issue guidance on the use of adaptive designs for clinical trials and more sophisticated statistical techniques to determine a drug’s efficacy. The FDA’s current leadership has given every sign that it would welcome this opportunity to modernize.
Reforming the FDA’s regulatory approach would reduce the risk of further catastrophes like the FDA’s delayed approval of eteplirsen, a drug that allows boys with Duchenne muscular dystrophy to maintain the ability to walk. This is a very rare disease, so enrolling enough patients to conduct a classical double-blind, randomized-control trial that satisfies the FDA’s statistical requirements is all but impossible.
The FDA’s “accelerated” approval of etepirlsen happened this month, one hundred days after its legally mandated decision deadline. Greater use of adaptive trial design, Bayesian statistics, and patient input – all encouraged by 21st Century Cures – would ensure more medicines like this get to patients much faster than they do now.
21st Century Cures would also greatly improve researchers’ access to patient data. Some of the obstacles researchers face getting access to data are imposed by federal privacy law. Developments in artificial intelligence and machine learning mean the opportunity cost of keeping clinical data locked up in siloes is becoming much too great. 21st Century Cures protects patient privacy while maximizing researchers’ ability to use Big Datasets for medical research.
21st Century Cures would also allow real-world data to be used to support the FDA’s approval of new indications for medicines already in use. This is important because manufacturers are not allowed to promote their medicines for benefits not already approved by the FDA. Even though such “off-label” communications are banned, doctors are free to prescribe medicines for off-label use. (Indeed, many states require insurers to reimburse for off-label prescriptions.)
The bill contains a number of other provisions that will improve the FDA’s performance with respect to allowing patients access to medical innovation. How much would all this cost, you ask? Virtually nothing! Only $1.4 billion over 10 years. And it is more than paid for with some spending cuts and increased revenue. Some comes from strange places, like the Strategic Petroleum Reserve. However, the dollar figures are so small any changes to tidy up the funding sources should be easy to find.
Further, 21st Century Cures is a bill that exemplifies how Congress is supposed to work. So open and deliberative was the process (which included roundtable discussions around the country for one year before Congressional hearings took place) that the House Energy & Commerce Committee approved the bill unanimously in early 2015. The House of Representatives approved the bill by a vote of 344 to 77 a few weeks later.
Then, things stalled in the U.S. Senate, where the sticking point appears to be funding for the National Institutes of Health (NIH). NIH funding has suffered in recent years, having peaked at $27.1 billion in 2003. By 2014, it was $31.3 billion. However, because of inflation, that comprised a real reduction of almost one fifth over the period. Further, authority for funding subject to future appropriations expired in 2009. Nevertheless, Congress has appropriated about $30 billion annually for NIH since then. 21st Century Cures would authorize $100 billion over three years, which would then have to be appropriated annually.
This is clearly a positive step for NIH funding, and restores it to the normal budgeting and appropriations process. This would give researchers dependent on NIH grants confidence to invest time and energy in their projects. Some in the Senate, however, seem to want to go farther, and add NIH funding to mandatory federal payments – like Medicare or Social Security.
This would be a step in the wrong direction. Trying to get around the regular authorizations and appropriations procedures was a feature (or bug) of the 2010 Affordable Care Act. In one instance, the Administration asserted a belief that the Act funded payments to health insurers (in a program called risk corridors) for three years. However, Congress did not appropriate those funds. As a result, insurers are losing money in Obamacare’s exchanges and exiting them, likely crippling the exchanges beyond repair.
Using his powers of moral suasion to get Congress to agree on a clean 21st Century Cures bill that funds the NIH through regular order, instead of getting hung up on “money for Democratic priorities,” would ensure a positive health care legacy for President Obama.