This post was authored by Richard J. Kovacs, MD, FACC, member of ACC’s Board of Trustees.

It’s been a busy summer for U.S. Food and Drug Administration (FDA) news. Several key decisions made by the Agency and the courts in recent months are sure to impact medicine in the months and years to come.

Of course the big FDA headline in July was the Agency’s approval of the first proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitor – Regeneron Pharmaceuticals’ Praluent (alirocumab) – for the treatment of patients with familial hypercholesterolemia or clinical atherosclerotic cardiovascular disease in conjunction with maximally tolerated statin therapy and diet modification. The FDA is expected to make a decision about a second PCSK9 inhibitor – Amgen’s Repatha (evolocumab) – later this summer. This new class of lipid-lowering drugs has the potential to offer millions of patients with high LDL-C an alternative treatment option to statins, which have been associated with numerous side effects for decades.

The FDA also approved Novartis’ Entresto (sacubitril/valsartan) this summer, which means that a novel drug will hit the market for the treatment of chronic heart failure. This drug, which has the potential to impact millions of patients in the U.S., received expedited review under the FDA’s Priority Review Program, which aims to reduce the time it takes the FDA to act on an application from 10 months to six months. Priority review, part of the Prescription Drug User Fee Act (PDUFA) is reserved for drugs that “treat a serious disease or condition and may provide a significant improvement over available therapy.”

Without PDUFA, the FDA wouldn’t be able to collect user fees in order to fund the drug review process. This summer, the Agency kick started discussions about reauthorizing PDUFA and its device equivalent, the Medical Device User Fee Act (MDUFA), both of which are set to expire in 2017.

During two FDA-convened meetings in July, ACC stressed the importance of efforts focused on bringing safe and effective drugs and devices to patients, and encouraged the FDA to leverage medical specialty societies to explore new ways to accomplish the common goal.

During the FDA meeting on PDUFA, I shared how the cardiovascular therapy landscape has advanced significantly since the law was enacted in 1997. In this time period, there has been a more than 30 percent reduction in death from cardiovascular disease, an increase in patients over the age of 65, new options for the reduction of stroke risk in patients with atrial fibrillation, new treatments for the expanding population of patients with heart failure, and new cholesterol lowering drugs for the millions of Americans in need of risk reduction. In follow-up comments, the College stressed that the FDA needs to strike an appropriate balance between protecting the public health and encouraging creativity and scientific advancement. The Agency should advance regulatory science, modernize the drug safely system, and incorporate patients and their input into the total product lifecycle, the ACC advised.

On the topic of MDUFA, the ACC’s STS/ACC TVT Registry serves as an example of an innovative collaboration among stakeholders to address concerns regarding access to new technology. Stressing that MDUFA IV funds should be used to postmarket surveillance, the ACC recommends that the FDA implement 2015 Planning Board recommendations; develop and sustain device registries where appropriate; identify and increase use of novel techniques for postmarket surveillance; and draft and implement regulations and guidance to address issues pertaining to use of existing data sets for FDA activities.

Another recent FDA hot topic is the marketing of drugs for off-label use. After years of debate, a federal court in New York ruled that pharmaceutical companies can market drugs for off-label use as long as the claims are truthful. The battle came down to free speech. Amarin, which was challenging the FDA to obtain the right to market its fish oil drug, Vascepa (icosapent ethyl), for non-FDA approved indications, was granted the First Amendment right to distribute evidence-based information about off-label drug uses to physicians. This ruling signals that significant changes could be coming for drug marketing in the U.S.

User Fee Act reauthorization and off-label marketing are sure to stay in the headlines this fall and beyond. Stay tuned to ACC.org and the ACC Advocate newsletter for developments.

 

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