Take-Up of FDA Fast Lanes

Oct 05, 2018

There’s always pressure to get the very best new drugs to patients faster, not only driven by commerce, but because it can make a life or death difference to patients. President Trump in particular has criticized the FDA for a slow and burdensome approval process. Leela Barham takes a look at the take-up of the existing options to speed up FDA approval.

The options for faster FDA approval

There are already options that have been introduced to speed up FDA approval, set out in Table 1.

Table 1: Expedited development and review options at the FDA

Option

Aim

Benefits

Process

Fast Track

To facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need

  • More frequent meetings with FDA to discuss drug development plan
  • More frequent written communication from the FDA
  • Eligibility for Accelerated Approval and Priority Review subject to meeting criteria
  • Rolling review so the company can have parts of its application reviewed in stages

Requested by company with a decision from the FDA within 60 days of the request

Breakthrough Therapy

To expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s)

  • All Fast Track features
  • Intensive guidance on efficient drug development program
  • Organizational commitment involving senior managers

Requested by company or suggested by the FDA to the company that the company considers applying

Decision from the FDA within 60 days of the request

Accelerated Approval

Allows drugs for serious conditions that filled an unmet medical need to be approved based on a surrogate endpoint

  • Faster approval than waiting for the final endpoints, but companies must still do phase 4 confirmatory trials

Company and FDA discuss option

Priority Review

Directs overall attention and resources to the evaluation of applications for drugs where there would be significant improvements in the safety or effectiveness of treatment, diagnosis or prevention of serious conditions when compared to standard applications

  • FDA aims to take action within 6 months versus the 10 months of a standard review

FDA decides on review designation

Source: Summarized from FDA Fast Track, FDA Breakthrough Therapy, FDA Accelerated Approval and FDA Priority Review

Fast Track was implemented in 1998, and even older are Priority Review and Accelerated Approval, with Breakthrough Therapy designation being the new kid on the block and introduced in 2012. That means that the issue of speed is not new, nor is the desire to really speed through those drugs that are most important to addressing serious conditions. Are these options being used?

More than half of novel drugs approved have used an expedited option

FDA is a very busy regulator, approving 46 novel drugs in 2017, the last year for which full data is available. FDA has also been approving drugs using the tools available to speed things up; in 2016 — when Trump become President — there was a peak of 73 per cent of novel drug approvals having used at least one of the expedited options in the last three years where full data is available (Figure 1).

Click here for Figure 1: Trend in overall use of FDA expedited development and review methods for novel drug approvals, 2015 to 2017

Of course, the detail that sits behind the headline of high take-up of expedited options shows various across the four options. Looking back further – from 2013 – allows a look at how the newest Breakthrough Therapy designation has been taking off too (Figure 2).

Click here for Figure 2: Trend in novel drug approvals and use of FDA expedited development and review methods, 2013 to 2017

Priority Review for two thirds of novel drug approvals

The FDA has ramped up the number of novel drugs approved under Priority Review from less than half to well over that, hitting a peak of 68 per cent approved using Priority Review in 2016 in the last five years. Priority Review is instigated by the FDA, not companies, so they must really think that these are drugs worth getting through the system that bit faster.

Fast Track falling

Companies are the ones who request Fast Track. The FDA doesn’t always grant companies their wish: FDA data shows that of 415 requests for Fast Track from 1 March 1998 to 30 June 2018, 278 were granted within the time limits set by the FDA (67%). Approvals using Fast Track have declined in the last five years, from the high of 44 per cent in 2014 to 39 per cent of novel drug approvals in 2017.

Accelerated Approval a rarer option

Accelerated Approvals have never hit the levels of either Priority Review or Fast Track, peaking at 27% in 2016 but more typically around 10% over the last five years.

Breakthrough Therapies on the rise

It’s those novel drugs approved with Breakthrough Therapy status that have really seen growth over the last five years. It’s risen as a proportion of novel drug approvals from 11 per cent in 2013 to 37 per cent in 2017.

Multiple options now common

Whilst any use of a speedier option has been common in the last three years– the question really is when it is not used – the number of novel drug approvals using only one option is now low and getting any two or three of the four options for expedited development and review is much more common (Figure 8). Getting all four though is rare too, as is getting those and orphan designation.

Click here for Figure 3: Trend in number of novel drug approvals by the number of FDA expedited options used, 2015 to 2017

Making use of available options

Based on the FDAs data – up to the end of 2017 – shows that FDA and companies make a great deal of use of options to speed things up. That may well mean that there is no need for any other options to add into the mix.

 

Leela Barham is a freelance health economist and policy expert. You can reach her on [email protected]. She is providing input as a subject matter expert into medicines pricing policy development with a UK government client and for the duration of her involvement in that project, she is restricted on what she can write about.

 

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