New Directions for FDA, Part 1 – 21st Century Cures

One of the often stated priorities for lame-duck Congress has been voting on the 21st Century Cures Act. In a press release from Saturday, November 26 from the House Energy and Commerce Committee, the House is set to vote on a new version of the bill following release by House and Senate health committee leaders on a “final” bill.

Originally passed in the House in July 2015 by a lopsided vote of 344-77, all attention then went to the Senate side for passage. And then there was a long wait. This year in April there were statements made that a bill could be out of committee within the week and ready for Senate action. It did not happen. At the beginning of September, there was some talk that it would get attention before the month was over. Now it comes to the lame duck session after the election and media reports that Senate Majority Leader McConnell has termed the bill a priority.

Among other things, the Cures Act seeks to liberalize the way FDA goes about regulating the approval of and access to drugs and biologics through a number of mechanisms. In addition the new final bill has something in it for just about everybody, including funding for the Precision Medicine Initiative for NIH, a boost in funding for FDA, $1 billion in grants to states to combat the opioid epidemic. Many claims are associated with the bill by its proponents – increasing research collaboration, incorporating patient perspective into drug development and the regulatory review process, providing new incentives for the development of drugs for rare diseases and provides support for the “cancer moonshot” initiative. In other words, it would all sound good. For some, too good.

The great milestone in moving FDA to speed approvals occurred during the early days of the AIDS epidemic when drug approvals were long and drawn out and mechanisms to speed approval were lacking. Through the course of activism and effort, FDA reforms allowed for new means to speed the process of approvals though Fast Track and Accelerated Approval programs. Subsequent reforms such as breakthrough therapy designation and orphan drug status designation have intensified the agency’s ability to bring innovative treatments forward for approval.

One might think that AIDS organizations – such a prominent proponent of earlier progress and liberalization of FDA’s approval process – might continue in support for reform offered by the Cures Act. But that is not necessarily the case. Before the 2015 House vote, co-founders of the HIV advocacy organization Treatment Action Group (TAG) joined with former FDA Commissioner Dr. David Kessler to pen an op-ed in the New York Times called “Don’t Weaken the FDA’s Drug Approval Process“. One of the several concerns raised in the piece was that biomarkers could be used to approve a wider set of treatments beyond those that are life-threatening or serious conditions and could allow clinical experience to replace data from well-controlled clinical trials, long considered the gold standard. In addition, TAG has issued an Action Alert for its member related to the lame duck session vote.

It is worth noting that in the wake of liberalizing the FDA approach to approvals in the 1990s, afterwards there were some high profile drug withdrawals that called into question whether or not drugs were being approved too quickly, at a cost to safety. The pendulum, it appears, has now swung again.

For critics it boils down to the issue of safety versus speed and whether or not the Act is even needed. FDA already is the gold standard and approves drugs more quickly than ever. New mechanisms to enhance approval such as Breakthrough Therapy Designation have been put into place and companies are utilizing such means. There were a large number of drugs approved in the past year for rare diseases. And FDA looked to patient experience in approving one drug.

TAG is by no means the only consumer group in opposition, but certainly one of the more interesting given the history. The proposed legislation is certainly one of the most talked about for the lame duck session of Congress and lobbying is reportedly in full swing. The House Energy and Commerce Committee is issuing almost daily press releases on the bill. There is likely a vote this week in the House on the new version and the Senate is said to consider it in December. If passed and signed into law, it represents perhaps only a part of the change that may come in the wake of the elections. More on that in Part 2.