Payers Need Enhanced Utilization Management to Optimize High-Cost Therapies, Panel Says

Financial toxicity of emerging high-cost therapies for rare and orphan diseases is a growing challenge for payers who are tasked with reviewing the appropriateness of new approvals entering the market each year. Over 70 novel drugs have been approved by the FDA since last year, of which more than half are indicated for the treatment of rare or orphan diseases, and by 2024, orphan drugs are expected to comprise 20% of the total drug spend.

Stakeholders of a Thursday panel discussion at the Academy of Managed Care Pharmacy (AMCP) Nexus 2022 meeting discussed the availability of enhanced utilization management (UM) programs for payers that can reduce waste among large patient populations they serve and result in significant savings annually, while providing members with the support needed for these rare, complex conditions.

Amy Ware, PharmD, director, Specialty Clinical Solutions, Magellan Rx Management, noted that the incentives, particularly market exclusivity, provided by the Orphan Drug Act of 1983 has led to substantial innovation within these diseases. However, there are now rare diseases that are crowded with products, with more coming down the pipeline, she said. And while these great strides in medical technology have benefitted patients, they pose great challenges for management of these products among payers in being able to cope with the financial stress.

“As we look ahead, there's a continued trend toward the approval of specialty medications and drugs for rare diseases, with 68% of medications submitted to the FDA considered specialty and 38% for rare diseases. The result of all this is an increase in spend that outpaces the increase in utilization,” said Ware.

Currently, an estimated 93 approved medical benefit drugs in the market exceed $300,000 in annual costs, with high-cost categories of chimeric antigen receptor (CAR) T-cell therapy, spinal muscular atrophy (SMA), and gout impacting managed care.

“Continued growth like this among high cost and orphan drugs is why worldwide drug sales are forecast to grow at a compound annual rate of 12.3% from 2019 to 2024, which is approximately double the rate forecast for the nonorphan drug market,” she added.

The lack of competition due to market exclusivity is a major barrier to access for some patients who may lack alternative treatments to manage their diseases, said Ware. And beyond the expensive list prices of these therapies, direct medical costs from inpatient/outpatient care, physician visits, medications and their administration, and durable medical equipment, as well as indirect costs (eg, productivity loss, forced retirement) compound cost further for patients and payers. In 2019, the economic burden of rare disease reached nearly $1 trillion in the United States.

Given these challenges for payers and patients alike, there are several management tools available to address waste, noted Brian MacDonald, PharmD, director, Clinical Strategy, Magellan Rx Management, which include comprehensive prior authorization (PA) criteria, enhanced UM and dose optimization strategies, and case management programs for the patient.

Focusing on enhanced UM, sample components of these strategies for high‐cost orphan conditions on medical and pharmacy benefit were addressed:

• Inclusion of mandated manual chart review in PA review
• Internal rare disease expert to “own” such cases and develop policies
• Specialty-matched physician review and support
• Approvals that trigger enrollment in complex case management

Goals such as timely access to renowned rare disease experts to provide impartial second opinion and ensuring appropriate diagnostic and treatment decision-making were touted by MacDonald through these enhanced UM programs, as well as optimizing current and long-term care plans and cost avoidance through reduction of potentially inappropriate treatment.

“What that personalized care team might look like for any given payer—it probably starts first and foremost with nurse outreach and having a nurse case manager reach out to the member and kick off that coordination of care; tapping into the social workers to provide support for the needs of the member and/or their caregivers; pharmacists can help with that medication reconciliation,” he said.

“And then in terms of the support, behavioral health care and access to a key opinion leader (KOL) for a second opinion for your plan recommendations is key, and throughout that process, having that provider engagement—contacting the prescribers and their teams to make sure everyone's aware and on board for with the needs of each particular matter.”

Regarding real-world application of enhanced UM strategies, Aravind Chodavapu, PharmD, clinical program manager, Blue Cross and Blue Shield of Alabama, discussed his organization’s experience partnering with Magellan Rx to launch a program focused on ensuring appropriate use while supporting members post-determination.

A total of 24 medical benefit drugs were selected for inclusion in the enhanced UM process based on several factors such as annual cost of therapy, clinical criteria for coverage, and KOL feedback.

From January 1, 2021 through March 30, 2022, 342 requests were received, said Chodavapu, of which 311 were reviewed. Teprotumumab was the most requested drug for review (n = 94), followed by pegloticase (n = 75) and eculizumab (n = 71). Results showed that from the 311 requests, 66 case management referrals were provided since the program's inception.

“Some of the more commonly found reasons for referral included high pill burden, high pill burden with behavioral health medications, and acute inpatient hospitalization or emergency department visits,” he noted. “In order to stratify members we need to prioritize case management, certain triggers were built into the workflow that included overall member medication list, history of present illness, and other clinical criteria that can be found in that clinical documentation.”

Since the program launch, $19,197,920 in total cost avoidance was achieved based on medical claims data only. Compared with the previous 5 quarters prior to program launch, total cost avoidance increased by 21.7% vs traditional UM. Moreover, cost avoidance from drugs in scope for the enhanced UM program represented 39.2% of total medical cost savings, explained Chodavapu.

Limitations were cited for the findings, which were the absence of pharmacy benefit claims as it limits cost avoidance and savings estimates to medical drug claims only and lack of long-term data to quantify the impact of case management on overall quality of life.

“We found that this program has been beneficial to our customers by ensuring that we're doing all we can to manage their funds appropriately, all while ensuring that members are getting the appropriate access to these high cost therapies,” he concluded. “This collaboration with Magellan allows for us to have a more patient-centric approach with our interventions as well.”