In the past 100 years, many of the diseases that were once commonplace yet deadly are now easily treatable and manageable because of the development of new drugs, treatments, and an approval process that keeps the American public safe. The drug approval process, however, is usually long and can keep lifesaving drugs from patients for years, before the proper clinical trials are completed and the Food and Drug Administration (FDA) reviews all of the testing data.

The process aims to approve new drugs that show an improvement over previously approved drugs, either by being more effective or holding a different, more convenient form (such as a pill compared to injection).

Breakthrough Therapy Status

The FDA drug approval process has been notoriously slow and measured, but this is why the FDA came up with the "breakthrough therapy" designation for drugs that show immense promise.

This title gives a drug company a way to speed up the development of a promising drug by showing evidence from mid-stage and smaller clinical trials, rather than waiting years to complete late-stage and far larger clinical trials. Bestowing the breakthrough status on a drug also allows the FDA to review the drug far more quickly and speed up the approval process.

Closer Review Of Drugs

This is not just a handshake between the FDA and a drug maker. The process requires extensive meetings with the FDA and having the drug developer give advice and be interactive during the approval process to address issues the review board may have. This includes making sure that the clinical trials are designed as efficiently as possible, targeting a specific group of people, as many new drugs now target a subset of a patient population.

Additionally, the drug must be tested in patients against the current standard of care, not only a placebo, to show it is better than what is currently given to patents and offers a significant enough enhancement in treatment. Additionally, the designation is reserved for treatments that work on the underlying cause of a disease, and not the symptoms. For instance, breakthrough status would not be given to a drug that would improve memory in Alzheimer's patients, but would be given to a drug that would reduce amyloid-beta plaques in the brain, which many believe causes the disease.

The breakthrough therapy designation was enacted in July 2012 in a new law called the Food and Drug Administration Safety and Innovation Act (FDASIA). Within the year it has been enacted, applications to be considered a breakthrough therapy have resulted in over 62 requests for drugs that show early clinical results bearing a significant positive outcome in patients. Around 24 drugs have been granted this coveted status as a result of extensive review of early-stage clinical trials.

The FDA previously had three other programs for over 20 years: Fast Track, Accelerated Approval, and Priority Review. But breakthrough designation required early clinical data in people showing an "unprecedented effect." The close relationship allows the company to "design collaborative, multidisciplinary development plans that hasten timelines to approval and minimize the number of patients exposed to less efficacious treatment or placebos."

The Result

Yesterday, there was a congressional briefing on the status and efficacy of breakthrough status designations. Jay Siegel, Johnson & Johnson's head of global regulatory affairs, spoke about the accelerated drug development process, and how it has shaved two years off of the close to 10 years that a typical drug takes to go to market. "This has had an enormous impact," Siegel said. "There is a very proactive role on the part of the FDA. They'll pick up the phone and call us and say 'Have you thought of this way to do this faster? Have you thought about this problem?'"

If a drug can gain approval in the U.S. up to two years before the European Union or Japan, will the other regulatory agencies change their policies to follow the FDA or will the drug need to go through a standard approval process? Additionally, many worry that the breakthrough designation might become overused and tax the system with too many drugs to approve in a short period of time. Regardless, bringing drugs that have been proven safe and effective to patients more quickly in a streamlined process will help people to live better lives.