New Drug CHF w/ Reduced EF

• by HeartOfBrick
• 2015-07-14 11:07:43
• General Posting
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FDA approved new drug for those of us with CHF and reduced EF. Consider talking to your cardiologist about being put on this new treatment. Begins shipping this week.

It used to be that the major hurdle in getting a new drug approved was successfully completing phase 3 trials – the long-term studies needed to prove to regulators that your drug was both effective and safe. These trials can take years to run and cost hundreds of millions of dollars. Yet, despite doing successful studies for many years in preparation for phase 3, these late stage studies are not guaranteed to be successful. Many a phase 3 program has gone down in flames despite promising earlier trials. Thus, the successful completion of phase 3 and ultimately, approval of the drug by the FDA, is the cause of jubilation at the sponsoring company and especially by the hundreds of people involved in completing the successful program.

This is likely what happened yesterday at Novartis when it received FDA approval for its new drug to treat heart failure, Entresto (previously known by its code name LCZ696). This drug is the first of a new class called angiotensin receptor neprilysin inhibitors. In its development of Entresto, Novartis carried out an extensive clinical plan, the cornerstone of which being PARADIGM, an 8,442 patient trial of people with heart disease in which half of the patients received the Novartis drug and the other half received enalapril, a generic drug that is the current standard of care. The results of the trial proved remarkable as Entresto showed a 20% reduction in the risk of death from cardiovascular causes or hospitalization for worsening heart failure.

Novartis’ clinical plan was a risky one in that PARADIGM was designed to compare its drug against standard of care. Oftentimes, pharmaceutical companies are criticized for running clinical trials designed to make their drug look as good as possible by picking either a placebo or a weak drug as the comparator. That was not the case here. Furthermore, this was an expensive roll of the dice for Novartis as this trial likely cost hundreds of millions of dollars to run with no guarantee of success. But, by setting such a high bar for their drug with PARADIGM, they carried out the type of study that patients and physicians need by utilizing the drug in a real world setting and comparing it to the best treatment. The reward can be found in the response of cardiologists such as Clyde Yancy, the chief of cardiology at Chicago’s Northwestern Memorial Hospital who told the Wall Street Journal that Entresto is “one of the few times that we have identified a medication that is better than the standard. It’s clearly superior to what we have.” This is how clinical trials should be run and MUST be run in order to prove the value of a new drug to the world.

Normally, that would be the end of the story. But another hurdle now looms in the form of payers. Novartis has set a price of $12.50/day for Entresto (about $4,500/year). This is a far cry from the costs of breakthrough cancer or hepatitis C medications, which can often exceed $100,000/year. Novartis estimates that over 2 million Americans should be eligible for this drug and one quickly comes to the realization that this is going to be a multibillion dollar revenue generator for Novartis. Payers, however, are also painfully aware of this and might be reluctant to add another new drug to their cost basis. So now a new battle faces Novartis: convincing payers to allow access to this drug for patients in their healthcare systems. Despite the fact that Entresto is a drug that will allow heart failure patients to live longer and avoid hospitalizations, Novartis is now in discussions to justify the price of the drug with respect to the overall benefits to patients and the saving of downstream healthcare costs. In other words, it will be awhile before many heart failure patients will get access to this important medicine.

Getting FDA approval for a new important drug is always a wonderful event. Just as important in this new world of healthcare is getting “approval” of the payers – an approval that is proving challenging.