Breakthrough Therapy Opens Door to Streamlined Drug Development

Posted by Rebecca Levine on Wed,May 15, 2013 @ 07:51 AM

The FDA's New Breakthrough Therapy Designation

What does the FDA's new Breakthrough Therapy designation mean for pharma and biotech firms? Commentary by Pennside Partners, consultants to the pharmaceutical and biotechnology industries.The FDA recently added a new designation, ‘Breakthrough Therapy’, to its roster of pharmaceutical regulatory terminology in order to ensure that truly innovative compounds get to the patients who need them most as soon as possible.  This new designation was enacted as part of the 2012 FDA Safety and Innovation Act (FDASIA).  The Breakthrough Therapy designation will provide companies with the opportunity for a streamlined developmental process, which could greatly shorten the development timeline. 

How the Breakthrough Therapy Request Process Works

A company can request the designation from the FDA at any time after the original IND submission, but is under no obligation to publicly announce the request.  Once the FDA grants the compound the Breakthrough Therapy designation, the process for approval becomes abbreviated.  The FDA representatives effectively form an advisory panel with the company, in order to determine the most efficient trial designs.  The review time will also be shortened due to the rolling NDA / BLA submission. 

Oncology Likely to Benefit Most

While the language of the bill does not specifically refer to oncology as a primary source of Breakthrough therapies, it is likely that a majority of these designations will apply to oncology compounds.  Other diseases that have already received Breakthrough Therapy status include the rare genetic disease epidermolysis bullosa, hepatitis C and cystic fibrosis. 

Flexible Approval Standards

These new breakthrough therapies must show early clinical evidence of a substantial improvement over existing therapies for serious or life-threatening diseases.  The bill purposefully left the approval standards vague, so that a judgment call can be made by the reviewers at the FDA.  There is no efficacy bar set; however officials warn that if a company wonders if their drug is in a grey area, it is unlikely to be a true breakthrough. 

Rigorous Data Requirements Remain

The data requirements for Breakthrough Therapies will be as rigorous as other submissions, but other faster trial designs may be possible for these compounds.  The options could include a phase Ib expansion or single-arm pivotal trial for diseases states where there is no standard of care.  A phase IIb randomized controlled trial with a modest-sized population might also be an option for some of these compounds where substantial efficacy benefit is seen over the standard of care.  Both these options and others could lead to either full or accelerated approval.

50% Breakthrough Therapy Approval Rate So Far

Over a dozen companies have already sought the Breakthrough Therapy designation, with approvals being granted to approximately half.  One of the compounds recently granted Breakthrough Therapy status is Pfizer’s palbociclib, an oral inhibitor of cyclin-dependent kinases (CDK) 4 and 6.  The designation was granted based on preliminary data from their phase II trial in first-line post-menopausal patients with ER+, HER2- locally advanced or metastatic breast cancer.  Interim data presented at the 2012 San Antonio Breast Cancer Symposium showed that women treated with the combination of palbociclib plus letrozole achieved a statistically significant improvement in median progression free survival (PFS) compared to women who received letrozole alone (26.1 months and 7.5 months, respectively).

Official FDA Guidance Set for July 2013

The official master guidance will be released by the FDA in July of 2013; however it is clear that this new designation will provide a significant benefit to qualifying extraordinary therapies. 

Pharmaceutical Industry Hurdles

What remains unclear at this time is how the already underfunded FDA will manage to provide even more time and attention to these compounds and how much the designation will actually streamline the regulatory pathway. 


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They have offices in the United States, United Kingdom, and Switzerland.

Tags: FDA, Oncology, Cancer Drug Development, Drug Development, Breakthrough Therapy, FDASIA

Receive Blog Posts by Email

Follow Us

Learn More About Our Services

Learn more about Pennside's pharma war games, strategy workshops, and competitive simulations for the pharma and biotech industries


Learn more about Pennside's pharma launch analog services, including pharma business development, pharma benchmarking, pharma best practices, pharma business analytics, business intelligence, ci, competitive intelligence, pharma launch planning, and other strategic consulting services for the pharmaceutical industry. 

Learn more about Pennside's  pharmaceutical and biotechnology consulting services spanning the following areas: competitive intelligence and monitoring, pipeline analysis and disease landscape consulting, marketing/sales/reimbursement support benchmarking, launch support and best practices benchmarking, customer facing and new commercial models, licensing and support consulting, strategy workshops, and outsourced business intelligence and fixed term contractors consulting for the pharma and biotech industries. 

Learn more about Pennside's Robust Pharma Competitive Intelligence and CI Monitoring Services. We provide superior pharma strategies based on actionable corporate intelligence. 

Learn more about our Oncology Drug Development Support Services: oncology launch analog service, oncology congress coverage service, post-launch intelligence and market monitoring, oncology business development and licensing and M&A support, and tumor/MOA clinical pipeline analysis services. Oncology launch adoption and market strategy services include: launch planning support, primary field intelligence and market insights, oncology competitive pipeline analysis, inlicensing reviews, oncology biomarker analysis, and benchmarking of oncology organizations. Oncology Congress Coverage services include: preparing a targeted congress schedule, attending and capturing key oncology conference information, and presenting oncology conference findings.

Contact Us or Request Information

Experience You Can Trust

Trusted pharma and biotech consulting experts for over 25 years