21 st Century Cures Act : No Cure for an FDA Patent Bias

BY TERRY G. MAHN O ne of the more controversial provisions in the legislative behemoth known as the 21 Century Cures Act, signed into law by President Obama on December 13, 2016, involves an overhaul of the regulatory approval process for new uses for old drugs, a fertile target for private investment in the important field of precision medicine. To incentivize investment in drugs that have already gone (or soon will go) generic, the Act makes it cheaper and easier for brand manufacturers to negotiate the FDA approval process. Instead of requiring multiple randomized well-controlled clinical trials, the current gold standard for all drug approvals, FDA will be allowed to rely on data from ‘‘observational studies, registries, and therapeutic use’’ to approve new indications for already-approved drugs. In addition, the legislation streamlines and expedites the review process for precision medicines and ‘‘qualified indications,’’ defined by the Act as cancer and other therapeutic uses ‘‘to be determined’’ by FDA. Critics point out that this is Congress, once again, meddling in science; that a forced speeding up of FDA regulatory reviews will artificially short cut an approval process that has served the public for more than half a century; and in the end will lead to compromises in patient safety. But the real question is why these legislative incentives are needed in the first place. Here, some of the blame can be squarely placed on FDA. For years, FDA has promoted policies that discourage investment in new uses for drugs already on the market specifically, albeit unintentionally, undercutting its own efforts to spur advances in precision medicine. These policies target method of use patents and stem from an historic misreading of the Hatch-Waxman Act and FDA’s kowtowing to political pressure to help reduce public health care costs even if it means undermining the patent system on which much of the U.S. pharmaceutical business is based.