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Keeping the fast lane open
September 2013
by Jim Cirigliano  |  Email the author


NEW YORK—After receiving bipartisan support in Congress and being signed into law in 2012, the U.S. Food and Drug Administration's (FDA) "breakthrough therapy" designation promises to hasten revolutionary new therapies through the often onerous approval process. Despite broad support among pharmaceutical industry leaders, some concerns remain that this latest attempt at creating a regulatory "fast lane" will be doomed to the same logjam that have plagued previous alternative regulatory pathways.  
The stated goal of breakthrough therapy designation, according to the FDA's website, is to expedite the development and review of drugs for serious or life-threatening conditions. Organizationally, the FDA has a separate senior management team that guides the review of these therapies and consults with the applicants to expedite the review process.  
As of July 2013, the FDA had received 77 requests for breakthrough therapy designation, of which 25 have been accepted and 27 denied so far. Among the approved breakthroughs announced thus far are GlaxoSmithKline PLC's Drisapersen (GSK2402968/PRO051) for treating Duchenne Muscular Dystrophy, Merck's Lambrolizumab for treating melanoma, Novartis' Serelaxin (RLX030) for treating acute heart failure and Vertex Pharmaceuticals' combination of Kalydeco and VX-809 for treating cystic fibrosis.  
Qualification for breakthrough therapy designation requires preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapies.
In an interview with Reuters on July 24, 2013, Vertex Pharmaceuticals CEO Dr. Jeffrey Leiden said that working with the FDA under breakthrough therapy designation was a completely different experience than the typical drug approval process. The discussions focused on streamlining all aspects of the review process in order to move the evaluation along quickly. Communicating directly with FDA reviewers allowed researchers at Vertex to resolve issues in minutes what would ordinarily have required weeks or months of back-and-forth communications through ordinary FDA channels.  
At a Washington, D.C., briefing, Johnson & Johnson's head of global regulatory affairs, Dr. Jay Siegel, expressed his expectation that the FDA's review of his company's experimental cancer drug ibrutinib will be shortened by two years thanks to its breakthrough therapy designation.  
Although the pharmaceutical industry's initial reaction to the FDA breakthrough therapy designation has been largely positive, and the early adopters have enjoyed speedy review in the year since the program was signed into law, recent analysis from pharmaceutical industry data experts at Context Matters in New York shows there may be important questions about the FDA's regulatory pipeline left unanswered.
Processing times for FDA approvals in general in the United States have lengthened significantly, and priority review processing times in particular have slowed markedly in just the past few years. In preliminary analysis by Context Matters, they calculate that in 2008 the average cycle time was 10.1 months for a priority review and 21.2 months for a standard review. By 2011, the average cycle time for priority reviews had burgeoned to 19.5 months, versus 17.5 months for standard review. Their conclusion is that, over time, the "fast lane" that priority review was intended to be has, in fact, become as slow or slower than the standard review process. 
These lengthy review times—even for priority review—no doubt necessitated the creation of a new "fast lane" for breakthrough therapies in the first place. It behooves researchers to raise questions about how the breakthrough therapy regulatory track can avoid the same pitfalls that have befallen previous attempt as fast-tracking reviews.
"There are a lot of questions that seem obvious to anyone looking at this issue would ask, but the answers aren't sitting there," says Kermit Daniel, chief analytic officer at Context Matters. "We're trying to make sense of the absence of answers. The data is spread around everywhere."  
Researchers may consider beginning to look toward the FDA's counterparts in the European Union (EU) for solutions and best practices. Whereas the FDA states its goal is to complete reviews in six months for priority and 10 months for standard, EU regulators list review within 210 days as a requirement. These quicker reviews—measured in days rather than months—mean fewer logjams for new products entering the regulatory pipeline.  
In addition, regulators in the U.S. and the EU return different data to the applicants. Whereas the FDA publishes only approved reviews, the EU regulatory body publishes relevant information about their review even when the product is rejected. This service provides applicants with valuable insights that can help them to address the regulatory concerns and avoid the guesswork in subsequent applications.  
Context Matters is a data analytics company that offers its clients awareness of the variety of data related to drug development. The company was founded on the belief that a data-driven approach is key to reshaping the way decisions are made in the industry. It uses curated information tied together from multiple sources to extract meaningful data that is updated and quality checked, and offers a user-friendly interface that provides intuitive access to the data and reporting.
Code: E091326



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