From Congress’s shelves to a vote seemingly in a heartbeat, a major US bill to fund biomedical research and streamline FDA approvals looks like it will pass Congress and be signed into law within days.
The conclusion of a long presidential election campaign appears to have been the catalyst to advance the 21st Century Cures bill, which would fund the National Institutes of Health’s precision medicine and cancer “moonshot” initiatives. The FDA, meanwhile, would be prodded to streamline approvals of drugs and devices, which has stimulated Democratic opposition.
More money, but is it enough?
The House of Representatives voted 392-26 yesterday to send the bill to the Senate for a final congressional decision. If it passes the Senate as expected – a vote is expected next week – the White House has said President Barack Obama will sign the legislation into law.
The headline of the bill is a $4.8bn, 10-year boost to the National Institutes of Health to fund precision medicine, the cancer moonshot, brain research and regenerative medicine. This would help to cushion, though not reverse, a budgetary flat-lining of the world’s largest health research organisation.
Congress appropriated $32.3bn for NIH in the 12 months ended September 30, 2016, only slightly above the $31.2bn of 2010. To match the inflation rate, the agency would have needed to receive $34.6bn in 2016, and these shortfalls have been felt throughout the sector (As pharma R&D engine surges US research backing sputters, January 2, 2015).
A major carrot to getting the legislation through Congress has been a $1bn pot states can access to fund prevention of opioid abuse. This issue has left its mark in almost every congressional district and has been particularly devastating in many parts of the rural South, Midwest and West.
Finding budgetary offsets for all the new spending also helped. The legislation takes money from programmes authorised by the Affordable Care Act, including more than $3bn over the next decade from a prevention and public health fund, as well as selling some of the US government’s strategic petroleum reserve to raise $1bn over the next three years.
Faster, reviewers: cure, cure
The legislation has become a buffet table for multiple interest groups in the life sciences field, with a focus on speeding drug and device approvals. These proposals have sparked some of the biggest opposition from certain Democratic lawmakers and interest groups.
On drugs, the legislation would require the FDA to consider “real-world evidence” from drugs already on the market for purposes of evaluating pharma companies’ request for label changes. The FDA also would be obligated to develop a review pathway for more biomarker endpoints and other tools that could speed drug development and reduce failure rates; issue guidance on adaptive trial designs and novel statistical modelling; and offer accelerated review for regenerative advanced therapies.
The legislation also would require the agency to develop ways to incorporate patient experience data in drug reviews. Guidance on how to collect and analyse these data would be issued within 18 months of the bill’s signing (Vantage point – Patient-reported data gain traction in FDA decisions, June 8, 2016).
In the medical device sphere, the bill establishes a “breakthrough therapy” designation much like that for drugs, eases limits on the humanitarian device exemption, and requires that FDA reviewers consider the “least burdensome” method for assuring safety and effectiveness during pre-market approval.
The focus on faster approvals has not gone without criticism from interest groups and lawmakers who warn that FDA reviewers will feel pressure to wave new drugs through without sufficient safety evaluation. The agency has its work cut out for it averting the next Avandia or Vioxx – this may not make its job any easier.