Move over Sovaldi: Could PCSK9 inhibitors be a bigger cost challenge?

The introduction of high-cost, high-use PCSK9 inhibitors (proprotein convertase subtilisin/kexin 9) enzyme inhibitors to treat high cholesterol may take the industry a step closer to a future where “healthcare costs break the bank,” according to industry insiders.

Estimates of annual pricing for PCSK9 inhibitors are in the cost range of $7,000 to $12,000. Even if PCSK9 inhibitors are indicated for a very narrow patient population, cost estimates show that this new class of drugs will eclipse initial costs of Sovaldi seen at its launch. In addition, PCSK9 inhibitors are biologics, so there will not be a simple pathway to less-costly generics for at least 10 years.

Several pharmaceutical manufacturers are currently developing PCSK9 inhibitors, which are projected to gain FDA approval by mid-2015.

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“The cost of these drugs will add nearly $50 per month to the premium costs for every insured person in the United States,” said Don Hall, a former health plan CEO of Delta Sigma LLC, in Littleton, Colo. “If this was the last of the high-cost, high-use pharmaceuticals, the system could adjust and move forward. Unfortunately it's only the beginning as new drugs for a range of neuromuscular diseases and cancer are poised to hit the market in the next few years. We are quickly getting to the point where healthcare costs crush our economy.”

Currently, patients with high cholesterol levels are often prescribed a statin, a once-a-day oral medicine. Statins are effective and affordable for the majority of people with high cholesterol.

CVS Health weighed in on the next chapter in the debate about the impact of expensive drugs on the healthcare system in a commentary in a Health Affairs blog.

"With the launch of Sovaldi to treat hepatitis C in 2013, we saw a first glimpse of the impact of high-priced specialty drugs that serve patient populations in the millions, but that was just the tip of the iceberg," Troyen A. Brennan, MD, chief medical officer, CVS Health, said in a press release. "Like the hepatitis C treatments, PCSK9 inhibitors represent a significant advance in treating intractable diseases convenient and highly effective with few side effects but they also pose a much more complex financial dilemma since, unlike the hepatitis C treatments which offer a cure in as little as 12 weeks, PCSK9 inhibitors will be prescribed as ongoing maintenance therapy for the duration of patients' lives."

While the annual cost of treatment with a PCSK9 inhibitor will likely be a lot lower than a course of treatment with one of the HCV medicines, costs will be compounded over the long-term, according to CVS. HCV medicines generally offer a cure in about 12 weeks, but PCSK9 inhibitors will be prescribed as a maintenance medication for the duration of patients’ lives, which could span decades.

Injected once or twice a month, evidence from clinical trials suggests PCSK9 inhibitors are well-tolerated and highly effective in reducing low-density lipoprotein (LDL) or bad cholesterol. Many believe these medicines will first be indicated for familial hypercholesterolemia, a genetic form of high cholesterol affecting approximately 620,000 Americans. Yet, there is a growing interest in additional patient subgroups where PCSK9 inhibitors may be appropriate, including for patients who are intolerant to statins, those who have more severe cases where statins are not effective, and those with a history of coronary artery disease. As a result, as many as 15 million Americans could eventually be considered candidates for this new class of drugs once approved.

Once approved, formulary managers will consider several factors when making decisions about formulary placement, including the efficacy of the drug(s), their indication and the size of the eligible patient population, according to CVS.

Related:Speciality drugs: A good value, despite high costs

Experts agree that careful managed care oversight and compliance with clinical guidelines will be vital to ensuring quality of care for patients while helping control healthcare costs when PCSK9 inhibitors are approved.