Keeping the fast lane open

Will FDA’s ‘breakthrough therapy’ designation really speed approval times?

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NEW YORK—After receiving bipartisan support in Congress andbeing signed into law in 2012, the U.S. Food and Drug Administration's (FDA)"breakthrough therapy" designation promises to hasten revolutionary newtherapies through the often onerous approval process. Despite broad supportamong pharmaceutical industry leaders, some concerns remain that this latestattempt at creating a regulatory "fast lane" will be doomed to the same logjamthat 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 ofdrugs for serious or life-threatening conditions. Organizationally, the FDA hasa separate senior management team that guides the review of these therapies andconsults with the applicants to expedite the review process.
As of July 2013, the FDA had received 77 requests forbreakthrough therapy designation, of which 25 have been accepted and 27 deniedso far. Among the approved breakthroughs announced thus far are GlaxoSmithKlinePLC's Drisapersen (GSK2402968/PRO051) for treating Duchenne Muscular Dystrophy,Merck's Lambrolizumab for treating melanoma, Novartis' Serelaxin (RLX030) for treatingacute heart failure and Vertex Pharmaceuticals' combination of Kalydeco andVX-809 for treating cystic fibrosis.
Qualification for breakthrough therapy designation requirespreliminary clinical evidence that demonstrates the drug may have substantialimprovement on at least one clinically significant endpoint over availabletherapies.
In an interview with Reuters on July 24, 2013, VertexPharmaceuticals CEO Dr. Jeffrey Leiden said that working with the FDA underbreakthrough therapy designation was a completely different experience than thetypical drug approval process. The discussions focused on streamlining allaspects of the review process in order to move the evaluation along quickly.Communicating directly with FDA reviewers allowed researchers at Vertex toresolve issues in minutes what would ordinarily have required weeks or monthsof back-and-forth communications through ordinary FDA channels.
At a Washington, D.C., briefing, Johnson & Johnson'shead of global regulatory affairs, Dr. Jay Siegel, expressed his expectationthat the FDA's review of his company's experimental cancer drug ibrutinib willbe shortened by two years thanks to its breakthrough therapy designation.
Although the pharmaceutical industry's initial reaction tothe FDA breakthrough therapy designation has been largely positive, and theearly adopters have enjoyed speedy review in the year since the program wassigned into law, recent analysis from pharmaceutical industry data experts atContext Matters in New York shows there may be important questions about theFDA's regulatory pipeline left unanswered.
Processing times for FDA approvals in general in the UnitedStates have lengthened significantly, and priority review processing times inparticular have slowed markedly in just the past few years. In preliminaryanalysis by Context Matters, they calculate that in 2008 the average cycle timewas 10.1 months for a priority review and 21.2 months for a standard review. By2011, 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 asslow or slower than the standard review process. 
These lengthy review times—even for priority review—no doubtnecessitated the creation of a new "fast lane" for breakthrough therapies inthe first place. It behooves researchers to raise questions about how thebreakthrough therapy regulatory track can avoid the same pitfalls that havebefallen previous attempt as fast-tracking reviews.
"There are a lot of questions that seem obvious to anyonelooking at this issue would ask, but the answers aren't sitting there," saysKermit Daniel, chief analytic officer at Context Matters. "We're trying to makesense of the absence of answers. The data is spread around everywhere."
Researchers may consider beginning to look toward the FDA'scounterparts in the European Union (EU) for solutions and best practices.Whereas the FDA states its goal is to complete reviews in six months forpriority and 10 months for standard, EU regulators list review within 210 daysas a requirement. These quicker reviews—measured in days rather thanmonths—mean fewer logjams for new products entering the regulatory pipeline.
In addition, regulators in the U.S. and the EU returndifferent data to the applicants. Whereas the FDA publishes only approvedreviews, the EU regulatory body publishes relevant information about theirreview even when the product is rejected. This service provides applicants withvaluable insights that can help them to address the regulatory concerns andavoid the guesswork in subsequent applications.
Context Matters is a data analytics company that offers itsclients awareness of the variety of data related to drug development. Thecompany was founded on the belief that a data-driven approach is key toreshaping the way decisions are made in the industry. It uses curatedinformation tied together from multiple sources to extract meaningful data thatis updated and quality checked, and offers a user-friendly interface thatprovides intuitive access to the data and reporting.

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