Pipeline: What to Watch in 2015

Although the pipeline is filled with agents that improve current therapy, there are a handful of agents that have the potential to change the standard of treatment for their respective disease states, resulting in considerable increases in healthcare costs to payors. 2015 will be highlighted by the new treatments for dyslipidemia and heart failure.

Hyperlipidemia-PCSK9 Inhibitors

New agents in development, known as Proprotein Convertase Subtilisin/Kexin type 9 (PCSK9) inhibitors, have shown unparalleled efficacy but will carry a hefty price tag. PCSK9 inhibitors are potent low density lipoprotein cholesterol (LDL-C) lowering agents. Clinical trials have shown that, in patients currently treated with statins, PCSK9 inhibitors have demonstrated a ≥ 50% reduction in LDL-C. PCSK9 inhibitors are monoclonal antibodies delivered via subcutaneous injections that are administered every two weeks or every four weeks. Although available safety data with PCSK9 inhibitors have shown them to be well tolerated, the FDA has requested additional safety data regarding neurocognitive adverse events from the pharmaceutical companies.

The FDA is expected to make a decision regarding the approval for both evolocumab (from Amgen) and Praluent (alirocumab-from Sanofi/Regeneron) this summer. We predict pricing will be similar with a rumored price tag of $7,000- $12,000/year for these chronic therapies. Fortunately, given their nearly simultaneous launch this year, we expect these manufacturers will seek early marketplace advantages with robust rebate negotiations for preferred formulary placement.

The total financial impact on payors will not be determined solely by price, but also by the size of the population treated, which could vary dramatically depending upon the labeling that the FDA ultimately approves. Both manufacturers have studied the PCSK9 inhibitors in smaller populations (heterozygous familial hypercholesterolemia) as well as larger patient populations (e.g., statin-intolerant patients, high-risk patients with established cardiovascular disease). Thus, the potential number of candidates for these agents ranges from 600,000 up to an estimated 15 million patients in the U.S. Medimpact is currently exploring strategies to optimally manage these agents.

Heart Failure

In recent years, the cornerstone of heart failure therapy has been the use of generically available, angiotensin converting enzyme (ACE) inhibitors. The introduction of a new drug to treat heart failure offers a new treatment option for physicians and patients, potentially satisfying an unmet need, while at the same time replacing a significant portion of utilization in a generic class with an expensive branded agent. In 2013, it was estimated that heart failure affects 5.1 million people in the United States. The incidence of heart failure increases with age, and with the aging population in the U.S., that number is certain to continue to increase. Whether the increased drug spend of treating heart failure patients with this new agent will be offset through reduced medical spend remains an unknown at this time.

Sacubitril/valsartan (LCZ696) contains a combination of a first-in-class angiotensin receptor neprilysin inhibitor (ARNI), sacubitril, along with the angiotensin receptor blocker (ARB), valsartan. In the clinical trial PARADIGM-HF, LCZ696 demonstrated a 20% reduction in cardiovascular deaths, a reduced number of hospitalizations for heart failure, and a reduced risk for all-cause mortality compared to enalapril in patients classified with New York Heart Association class II- class IV heart failure. Assessment for cognitive function will be added to clinical trial measures by the manufacturer as neprilysin has been indirectly linked to Alzheimer's disease. No cognitive impairment has been observed in the current clinical trials. The FDA granted LCZ696 priority review and a decision is expected on its approval by August 2015. If approved, LCZ696 would potentially displace generic ACE-I/ARB use in heart failure patients. MedImpact will conduct a full review of LCZ696 to be presented during the 3rd quarter P&T Meeting.