PCSK9 Inhibitors: Smoothing the Path to Access and Value

The support of specialty pharmacy can facilitate patient access to this new class of drugs for the treatment of high cholesterol.

The sabres are rattling as the health care industry attempts to find an equitable tiered-access and pricing model for newly approved proprotein convertase subtilisin kexin 9 (PCSK9) inhibitors.

These new cholesterol-lowering maintenance drugs were initially priced at more than $1000 per month. As a result, a classic, bloody battle between biopharmaceutical manufacturers and payers has begun.

Manufacturers want top dollar for their therapies to ensure they recoup research and development spending while payers want to keep spending low and demand evidence-based value. Stuck in no man’s land are the prescribers and patients who need clarification on the requirements and pathway to approval for ultra-high-cost maintenance drugs like PCSK9 inhibitors.

In the midst of the shift away from fee-for-service to value-based pay, manufacturers can expect increasing scrutiny over the efficacy, appropriateness, and value derived from these drugs. They can further expect that prior authorization (PA) for PCSK9 inhibitors is going to be difficult to attain, to say the least.

They are going to need more robust support from their distribution network pharmacist partners. In response to the initial sticker shock to the PCSK9 inhibitor pricing announcement and the accompanying doomsday “$100 billion added cost to the health care system” rhetoric, the Institute for Clinical and Economic Review (ICER) recently published a draft report that contained a theoretical analysis on the cost-effectiveness of PCSK9 drugs for patients.

Using a “willingness to pay” threshold of $50,000 per quality-adjusted life-year gained, ICER arrived at cost-effective price of $2100 per year, a number nearly 7 times less than the manufacturer’s price. Although it is going to take a significant amount of yet-to-be-acquired real-world patient data to arrive at a closer value-based price, the pressure on manufacturers, prescribers, and patients to validate their need for PCSK9 inhibitors on PA forms is going to be heavy.

These parties should carefully consider the following in the interest of smoothing the path to payment approval, ensuring that the patients who can derive the highest value from PCSK9 inhibitors can access and adhere to them. Population health assessments related to any new therapy must wait until enough data points exist, and it starts one patient at a time.

Prescribers who provide solid documentation of lab results, assurance of adherence, and documentation of instances of acute care will be more likely to achieve simpler, swifter PA of PCSK9 inhibitors for their chronically ill patients for whom lower cost statins have not been effective.

(1) Established baseline lab data.

Tracked low-density lipoprotein and high-density lipoprotein levels at regular intervals are the most basic forms of empirical data payers will require before approving payment for PCSK9 inhibitors. If manufacturers, prescribers, and patients want to smooth the PA process, they will ensure these labs are done at the proper intervals.

These lab data are essential to proving that lower-cost statins have not been effective and will be critical to learning about the value of these drugs as they are prescribed, accessed, and administered. Supporting lab data that show the patient is not improving should be the first step on the path to securing PA.

(2) Assurance of adherence (with metrics).

As with any specialty medication therapy, excellent adherence is critical to derivation of value through improved outcomes and lower costs. In the case of PCSK9 inhibitors, what’s paramount for prescribers and patients is demonstration of outstanding adherence to previously prescribed, lower-cost therapies.

Before clinicians prescribe newer, higher-priced medications, however, they need to know if the patient is adherent to their current regimen. Before abandoning current treatments, prescribers should thoughtfully evaluate patient adherence plans and methodologies before abandoning lower-cost statins.

Combining lab data with assurance of adherence is the next most meaningful step forward to PCSK9 inhibitor payment authorization. Biopharmaceutical manufacturers who include specialty pharmacies with proven medication therapy management (MTM) programs within their distribution networks for PCSK9 inhibitors and other ultra-high-cost specialty therapies will find themselves ahead of the game when it comes to PAs because of the data available to prove adherence rates.

As the role of pharmacists continues to grow in overall health care delivery, it is becoming expected that they offer expanded services and programs that improve adherence rates, provide patient education, and remove barriers to access. Consistent, direct patient outreach, a clinical focus, customized adherence packaging—including nascent technologies that will tell the care team precisely when a patient has taken their medications—and other pharma-analytics are a few resources available that manufacturers, prescribers, and payers should demand from their specialty pharmacy networks.

Claims data plus a medication management entity that provides adherence data are powerful tools. Tapping all available adherence data resources, including the resources available from pharmacists, builds a stronger case based on empirical evidence that is going to be needed to assert it is time for the more expensive drug to prove its value.

Prescribers can assume that payers are going to want to see proof of adherence and labs at the recommended intervals on an ongoing basis if patients are going to stay on PCSK9 inhibitors, unless the patient is going to pay cash.

(3) Acute instances of care.

One of the data points prescribers should anticipate and incorporate into their patient access plans for PCSK9 inhibitors is instances of acute care. Much of the value derived from high-cost specialty therapies comes in the avoidance of acute instances of care.

This is the entire goal of effective MTM programs for specialty therapies. If a patient has a history of familial hypercholesterolemia and has been hospitalized on multiple occasions for heart bypass surgery, this person will likely achieve access to PCSK9 inhibitors more swiftly.

Patients who have not yet been hospitalized, however, are less likely to have their request for payment authorization for high-cost therapies approved in a rapid fashion. Once the payment or coverage has been approved, it is highly likely that patients who have experienced multiple acute care instances are going to require a significant amount of hand-holding to not only remain adherent to their PCSK9 inhibitor regimens, but also to reconcile multiple medications, multiple physicians, and rigorous follow-up visits (including the establishment of baseline and ongoing labs mentioned above).

This process necessitates assignment of a quarterback (clinical pharmacists are best suited for this role) to coordinate and execute the calls made by the head coach (the physician) on the sideline. There is a large gap between the medication management chronically ill patients receive in clinical settings and the support they get after returning home.

That gap lowers adherence rates. One manufacturer representative told me recently that adherence rates to oral oncological therapies are in the 60% range.

That means 40% of patients undergoing treatment for cancer are not adherent to their life-saving, ultra-high-cost therapies. Specialty pharmacies that provide controlled trial—like conditions in the home through best-in-class MTM protocols should be best positioned to deliver value for manufacturers, payers, and patients.

A peer-reviewed study conducted by my colleague Patrick Dunham, president and CEO of Curant Health, found that patients on highly active antiretroviral therapies receiving enhanced MTM services increased adherence rates by a margin of 69% after 6 months. The overall annual health care spend for those same patients decreased by $3000 per patient per year.

Focusing on patient outcomes and additional services by specialty pharmacies mirrors the move Medicare is making toward value-based care for reimbursements. As these forces grow in influencing the health care system, the beneficiaries will be patients whose lives improve, the manufacturers who can more effectively account for the value their therapies provide, and the payers whose highly adherent patients experience fewer instances of more costly acute care.

For specialty pharmacies, elevating the outcomes and experiences of patients over all else is a winning strategy for developing the best partnerships with all stakeholders, including biopharmaceutical manufacturers. What is needed to smooth the pathway to value-based pricing, access, and payment for these high-cost specialty therapies are clearly defined, simplified waypoints and metrics.

Until those become more clear, biopharmaceutical manufacturers would do well to assist prescribers by providing guidance and methods for prospective patients’ paths to PCSK9 inhibitors and by directing them to specialty pharmacies that provide patient education, MTM proven to improve adherence, and the pharma-analytics that will be crucial to securing payment authorization, access, and value derivation from any high-cost therapy for chronically ill patients. SPT

About the Author

Marc O’Connor is chief operating officer for Curant Health. Curant Health treats patients nationwide through its medication management protocols, including medication reconciliation and establishment of personalized medication regimens, and supports its provider partners and care coordination with its award-winning electronic health record, MedPlan. Curant’s health care professionals provide individualized care proven to improve the lives and reduce the overall health care costs of chronically ill patients.