The White House on FDA Approval Burden – Is There a There There?

In his remarks to a joint session of Congress delivered this week, President Trump expressed the point of view that the “slow and burdensome approval process at the Food and Drug Administration keeps too many advances… from reaching people in need.” He went on to say that if “we slash the restraints” then we can ostensibly speed the arrival of new medicines.

But is it accurate to characterize the FDA’s process as slow and burdensome?  Are new treatments in fact being kept from reaching people in need?  Are people with rare diseases having their hopes for treatment quashed by an overbearing bureaucracy?

These questions are actually not new. In fact, they have been around for quite some time and most certainly came to a head when in the early years the HIV/AIDS epidemic was exacting a heavy toll and there were no treatments. How to speed approvals has long been a question that has always interested policy makers. And while the objective of doing so is everyone’s goal, experience has demonstrated that achieving the goal is more likely the result of a process than an event.

According to testimony submitted by the General Accounting Office before the Senate Committee on Labor and Human Resources in 1996 on FDA Review and Approval Times, in 1987 it took an average of 33 months for New Drug Applications (NDAs) submitted to the agency to be approved. This was during the early and very savage years of the AIDS epidemic when thousands upon thousands of people were dying without any meaninful treatment for their HIV infection. According to the same testimony, by 1992 – just 5 years later – the number of months to approve a new drug had dropped to 19 months.

More improvement was forthcoming. With the advent of the Prescription Drug User Fee Act (PDUFA) in 1992, the agency acquired new revenue that allowed for an expanded capacity to review NDAs with the consequence that approval times were reduced even further. To make a long story short, today PDUFA brought the consideration time for a new drug just 10 months in most cases, and even faster in special cases.

That is because in the beginning in 1992, new review mechanisms began to be introduced that would speed the consideration of important new drugs that would make a difference to people who had serious medical conditions. These new mechanisms provide enhanced pathways for new product consideration and include Accelerated Approval, Fast Track, Priority Review and Breakthrough Designation. Each contributes in its own way to moving an investigational molecule down the approval pathway by either speeding up the process or bringing in additional FDA resources. In addition, to encourage the development of drugs for rare diseases such as mentioned in the speech this week, there is Orphan Drug Status which qualifies a drug sponsor with incentives, including tax credits for qualified clinical testing,among others.

But do these mechanisms get used to help bring drugs to patients more quickly?

By my count in 2016 there were 32 FDA announcements involving drug approvals (I keep a database of FDA press releases).

  • A majority (18) of these were announcements of the approvals of drugs that were either not life-threatening/serious conditions or
  • where there were already other treatments approved (12),
  • approval of a biosimilar (3),
  • approval of a generic (1)
  • or approval of an OTC switch (1)
  • giving ok to an expanded label (1).
  • 14 were announcing approvals of treatments for serious and/or rare conditions or offered new ways to treat. Of those 14 announcements, all of them had either one, multiple or in some cases all of the enchanced pathways described above and 9 were orphan drugs. The approvals were in different areas of oncology, hepatic diseases and central nervous system disorders.

In other words, FDA is approving miracle drugs for rare conditions in which people were waiting for treatment and doing so in increasingly short periods of time. Great strides have been made since the days of the 1980s, and the progress has continued. FDA deserves credit, as do patient advocates.

It may be obvious to most that what the Administration is talking about is not the actual time FDA takes to approve a compound, but the burden of proof that drug sponsors must meet in order to gain approval. Or perhaps it is not so obvious. In any case, that is quite a different subject from a process being characterized as “slow” and perhaps one with quite a different outcome.

If the “slow” process referred to is one that requires speeding up drug marketing by lowering the burden associated with the development of data to show efficacy and safety, then it is really talking about the quality of new medicines. That again is a topic that the first generation of HIV advocates know something about – having mounted a movement to do just that.  We would do well to listen. As former AIDS activists and a former FDA Commissioner pointed out in an opinion piece in the New York Times last June, significant progress can be – and has been made in working with FDA to effect faster approvals.  That has been achieve not by working against FDA but rather with FDA. There has been significant progress in speeding up approvals while ensuring quality for both safety and efficacy in the medicines that are needed but faster approval should not come at the expense of quality and safety. That much should be clear to any new FDA Commissioner.

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