What’s In - and Out - of the “Cures” Bill

Congress took a major step towards shoring up FDA operations and biomedical research supported by the National Institutes of Health with House passage of the 21^st Century Cures bill. The measure includes important provisions that can help FDA hire the scientists and experts it needs to evaluate new medical products. And it supports initiatives that encourage pediatric research, patient-focused drug development, qualification of biomarkers, and the development of needed antibiotics and treatments for rare conditions.

An important addition to the package includes provisions to fund and encourage treatment and prevention of opioid abuse, including $1 billion a year for state programs, as requested earlier by the Obama administration. And important additional provisions expand mental health services and support research on regenerative medicine and medical countermeasures.

Key provisions encourage FDA approval of novel adaptive designs and statistical modeling in clinical trials, as well as a more controversial measure that permits wider use of real world evidence to support approval of additional indications for marketed medicines. Medical device makers gain a new approval pathway for “breakthrough” devices, and there’s language to streamline diverse conflict-of-interest reporting requirements for researchers. NIH’s National Center for Advancing Translational Sciences (NCATS) also gains added backing for phase II clinical trials on promising new drugs.

Yet there’s disappointment in the the biomedical research community that the funding for federal research and health care programs is less than originally proposed. NIH gets an additional $4.8 billion over 10 years, half that in the earlier House-passed version. Most of the additional resources will support the current Precision Medicine Initiative, the cancer moonshot, the brain research initiative and regenerative medicine research. FDA gains $500 million, down $50 million from the earlier bill. The added funding, moreover, is authorized, but not appropriated, opening the door to future cuts in promised resources that could hinder efforts at FDA and other agencies to implement the many new requirements in the legislation.

In addition, a provision backed by pharma was dropped to appease critics claiming that the bill is a give-away to industry. The final negotiators removed a change in the Open Payments program that would have exempted from disclosure the value of reprints, textbooks and fees for continuing medical education activities provided by pharma companies to physicians.

The bill retains language clarifying and authorizing manufacturer communication of the economic benefits of drugs to payers and formulary operators. But the measure was weakened by dropping a mandatory timetable for FDA to issue guidance on the policy.

It is no surprise that the legislators also left out language limiting FDA regulation of laboratory-developed tests. FDA recently decided not to proceed with further guidance on this contentious topic, recognizing that Republicans strongly oppose any FDA move to extend its oversight in this area.

Despite these caveats, there is much to applaud in the Cures bill, and hopes it will be approved by the Senate early next week. That would permit President Obama to sign it into law as one of his final acts before leaving office.