Cures Act ensures U.S. will continue to outperform Canada in approving new medicines
Last month, President Obama signed the last bill of his presidency, the 21st Century Cures Act. The most important part of this bill is reform of the regulatory processes at the Food and Drug Administration (FDA), which will speed up innovation and patient access to new medicines.
In 2013, the Fraser Institute published research showing Health Canada’s Therapeutic Products Directorate, which grants permission for new medicines to be used in Canada, lagged behind its European counterparts and the FDA. Because of the Cures Act, the gap in performance between Canada and the U.S. is likely to get significantly worse.
The Act combines a number of initiatives that Canadian policymakers should study. 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.
In lay terms, it means “learning while doing” will be permitted for researchers. They won’t have to wait until a trial is over to take the next step in winning approval. The FDA’s current leadership has given every sign that it would welcome this opportunity to modernize.
It will be especially beneficial for researchers studying very rare diseases, for which it is all but impossible to recruit enough patients to achieve statistically significant outcomes in a classic clinical trial. The reform will reduce the risk of further catastrophes such as the FDA’s delayed approval of eteplirsen, a drug that allows boys with Duchenne muscular dystrophy to maintain the ability to walk.
The FDA’s “accelerated” approval of etepirlsen happened last September, 100 days after its legally mandated decision deadline. Greater use of adaptive trial design, Bayesian statistics, and patient input—all encouraged by the Act—will ensure more medicines like this get to patients much faster than they do now.
The Act will also greatly improve researcher 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. The Act protects patient privacy while maximizing researcher ability to use Big Datasets for medical research.
The Act will 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 American taxpayers, you ask? Only $1.4 billion over 10 years. And it’s paid for with cuts to other government spending. (The FDA’s budget for 2016 alone is almost $5 billion.)
Canadians should demand Parliament study the 21st Century Cures Act, with a view to importing many of its provisions.
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