No matter where you live, there is inevitably some type of natural disaster your community has to plan for. For instance in California, scientist warnings of the “big one” keep builders and policymakers up at night trying to prevent massive infrastructural destruction. Although less tangible, planning for disruptors in the health care system is no different.

PCSK9 inhibitors may test the viability and infrastructure of the American health system like never before. The inhibitors or monoclonal antibodies (mAbs), work by blocking proprotein convertase subtilisin-kexin 9 (“PCSK9”), a protein that reduces the liver’s ability to remove LDL cholesterol from the blood. By blocking PCSK9, the liver is able to remove LDL cholesterol thereby reducing plaque in the arteries and, hopefully, cardiovascular events.

With clinical trials underway and some showing hopeful outcomes, the lifelong maintenance therapy shows promise for a potentially huge patient population.

Like an earthquake, we don’t yet know what the magnitude or cost of the soon-to-market PCSK9 inhibitors will be. However, unlike an earthquake, we know when they’ll be here and that’s soon: a fact that has many payers losing sleep.

To gain some industry perspective on this topic, HIRC discussed the impending launch of PCSK9 inhibitors with its payer panel comprised of 15 pharmacy and medical directors from leading managed care plans in February 2015. Our panel pointed to three areas of concern that are keeping them up at night:

1. Price

PSCK9 inhibitors are both difficult and expensive to manufacture, thus the price of the new therapy is expected to be high. The estimated range is broad, from $4,000 - $12,000 annually per member. Combine that price tag with the knowledge that this is not just a one-time cost, but rather an ongoing maintenance therapy—as compared to the notoriously pricey Sovaldi, for instance—and the implications are huge. Understandably, a precise price estimate is a variable that our payer panel wishes it had to plug into their predictive models. As one panelist pointed out, this particular patient population may be accustomed to generic copays, so this class will likely require significant copay support from pharmaceutical manufacturers to ensure access.

2. Size of Population

Another variable is the size of the target population. Without the package insert, the exact PCSK9s indication is unknown. On the low end, if only approved for familial hypercholesterolemia, the patient population is estimated at about 600,000 Americans. If approved for those who are non-responsive to statins and/or statin intolerant, the initial eligible population could grow to about 3.5 million—and that isn’t all. Estimates can increase dramatically should the label be expanded or if the therapy is designated as primary prevention.

3. Outcomes

Robust long-term outcomes studies are not yet available for these products and won’t be until 2016-2017; so, while this new therapy has demonstrated that it is highly effective at improving more intermediate outcomes (lower LDL), payers also want to know whether it will reduce the likelihood of future cardiovascular events and, therefore, reduce medical costs.

Given the unknown variables above, it is difficult for payers to say exactly what their strategic approach will be to PCSK9s, but here are some ideas from our panel.

1. Strict Utilization Management

Two thirds of payers indicate that they’ll rely on relatively strict utilization management to ensure appropriate use, mostly for the statin intolerant population:

• Two of fifteen (13%) payers anticipate strict PA/notification criteria, requiring lab values and prescription by a specialist.

• Another 8 of 15 (53%) panelists indicate that they’ll also likely implement a step edit (perhaps a double step, some say) for both statin intolerant patients and non-responders, requiring try and fail of a generic statin.

2. Contracting

One third of payers interviewed also remain optimistic for contracting opportunities. Given the recent contracting activity in hepatitis C, played out in true game theory perfection, this could be another opportunity to drive down the price by choosing an exclusive preferred agent as multiple therapies are expected to launch around the same time.

Others simply aren’t sure yet with the unknown variables at hand, and state that the label will drive their strategy forward.

Concluding Thoughts

In preparation for the “big one,” Californians are used to bridges and freeways being closed on holiday weekends from time to time as state construction crews install the latest earthquake resistant technology. Unlike a devastating earthquake from which no one benefits, PSCK9s represent a potentially life-saving therapy for many patients with hard to treat cardiovascular diseases.

Strict utilization management, aggressive contracting, and perhaps a little regulatory action on specialty pharmaceutical pricing, may together be strong enough to ensure the inevitable shake-up doesn’t cause the whole system to crumble.