Breakthrough Designated Drugs and Specialty Pharmacy


The most impactful of the FDA designations, this status may be applied to drugs used to treat serious or life-threatening conditions.

Getting a new drug to market is an expensive and time-consuming process. According to a study by the Tufts Center for the Study of Drug Development, it costs an average $2.558 billion for a new drug to be developed and go through all the necessary steps to gain FDA approval. For many drugs, this process can take more than a decade.1 Furthermore, most drugs in the earlier stages of research don’t make it that far and never reach the market.

To expedite the development process for selected promising drugs, allowing them to become available to patients sooner, the FDA has created special designations that can be earned when a drug meets certain criteria. Perhaps the most impactful of these designations in terms of the potential to expedite drug development and lower development cost, is breakthrough status. This status was created in 2012 and may be applied to drugs used to treat serious or life-threatening conditions where, “preliminary clinical evidence indicates that a drug may demonstrate substantial improvement over existing therapies on 1 or more clinically significant endpoints.”2 Many of these drugs fall into the realm of specialty pharmacy.

One interesting aspect of breakthrough status is that if it is granted early enough in the drug development process, a manufacturer may be able to submit a new drug application or biologic license application for FDA review based on the results of phase 1 or phase 2 trials.3 Because phase 3 clinical trials often take multiple years to complete and regularly involve hundreds or thousands of patients, removing this step in the drug development process is a huge savings in time, money, and other resources for a drug manufacturer. This also allows a drug to come to market sooner and make it available for patients in need of treatment.

Breakthrough status has the potential to provide benefits to both manufacturers and patients, but it also raises some concerns. Because drugs can be approved without running later-stage, more rigorous trials, there is some worry that the safety and efficacy of drugs approved under breakthrough status has not been sufficiency proven.

The FDA is constantly trying to maintain the correct balance of thoroughness and timeliness in drug development requirements and their review process. In the case of breakthrough drugs, the FDA has decided that the potential benefits outweigh the risks. By restricting breakthrough status to drugs that treat serious or life-threatening conditions where there is an unmet medical need, the FDA has decided that a bit more uncertainty regarding drug safety and efficacy is acceptable if it allows patients in need of treatment to gain access to new drugs more quickly.

After expedited approval, which may be earned with the breakthrough designation, drugs are often required to complete additional clinical trials to provide further confirmation of safety and efficacy. If these additional trials don’t provide favorable results, the FDA may retract approval, forcing the drug to be removed from the market.

Products with a breakthrough designation have become relatively common. As of August 25, 2017, the FDA has awarded 170 of the designations, and 61 therapies have been approved that have earned the status. Most of these products are for the treatment of cancer or rare inherited disorders.4

Recently approved or pipeline drugs that have earned breakthrough status are worth recognizing for those involved in specialty pharmacy. They may be, but are not required to be, a novel mechanism of action to treat a disease with limited other treatment options. However, it is worth noting that a product with a breakthrough designation is not inherently superior to currently available treatments. When FDA review is based on earlier stage trials with fewer patients, the breakthrough treatment may not have accumulated as much supporting evidence of safety and efficacy as drugs that have gone through clinical trials in the more traditional but time-consuming route.


1. Tufts CSDD assessment of cost to develop and win marketing approval for a new drug now published. Boston, MA: Tufts Center for the Study of Drug Development; March 10, 2016. Accessed October 10, 2017.

2. FDA. Fact sheet: breakthrough therapies. Accessed Updated December 10, 2014. Accessed October 10, 2017.

3. FDA. Guidance for industry expedited programs for serious conditions — drugs and biologics. Published May 2014. Accessed October 12, 2017.

4. Friends of Cancer Research. About breakthrough therapies. Accessed October 10, 2017.

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