The financial toxicity of emerging high-cost therapies for rare and orphan diseases is a growing challenge for payers who are responsible for reviewing the appropriateness of new approvals entering the market each year. Over 70 new drugs have been approved by the FDA since last year, more than half of which are indicated for the treatment of rare or orphan diseases, and by 2024, orphan drugs are expected to account for 20% of total drug spending. medications.
Stakeholders in a Thursday roundtable 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 the waste among the large patient populations they serve and result in significant savings each year, while providing members with the support needed for these rare and complex conditions.
Amy Ware, PharmD, director, Specialty Clinical Solutions, Magellan Rx Management, noted that the incentives, particularly market exclusivity, provided by the Orphan Drugs Act of 1983 have led to substantial innovation in these diseases. However, there are now rare diseases that are rife with products, and more are on the way, she said. And while these great advances in medical technology have benefited patients, they pose great challenges for the management of these products among payers by allowing them to cope with financial stress.
“As we look to the future, there is a continued trend toward approval of specialty drugs and drugs for rare diseases, with 68% of drugs submitted to the FDA considered specialties and 38% for diseases rare. The result of all of this is an increase in spending that outpaces the increase in usage,” Ware said.
Currently, 93 approved medical benefit drugs on the market are estimated to 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 expensive and orphan drugs is why global drug sales are expected to grow at a compound annual rate of 12.3% from 2019 to 2024, roughly double the rate forecast for the non-orphan 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 conditions, Ware said. And beyond the high list prices of these therapies, the direct medical costs of inpatient/outpatient care, doctor visits, medications and administration, and durable medical equipment, as well as indirect costs ( eg, loss of productivity, forced retirement) further exacerbate costs for patients and payers. In 2019, the economic burden of rare diseases reached nearly $1 trillion in the United States.
Given these challenges for payers and patients, there are several management tools available to combat waste, noted Brian MacDonald, PharmD, Director, Clinical Strategy, Magellan Rx Management, which include pre-authorization criteria ( AP), enhanced MU and dose optimization strategies. , and patient case management programs.
Focusing on enhanced MU, examples of components of these strategies for high cost orphan conditions on medical and pharmaceutical benefits were discussed:
- Inclusion of mandatory manual file review in PA review
- In-house rare disease expert to “own” these cases and develop policies
- Examination and support of physicians corresponding to the specialty
- Approvals that trigger enrollment in Complex Case Management
Goals such as timely access to renowned rare disease experts to provide an unbiased second opinion and ensure 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 avoiding costs through the reduction of potentially inappropriate treatment.
“What might that personalized care team look like for a given payer – it probably starts first and foremost with nurse education and having a nurse case manager contact the member and initiate that coordination of care; engage social workers to meet the needs of the member and/or their caregivers; pharmacists can help with this medication reconciliation,” he said.
“And then in terms of support, behavioral healthcare and access to a key opinion leader (KOL) for a second opinion for your plan recommendations is essential, and throughout that process having this supplier commitment – reach out to specifiers and their teams to ensure everyone is aware and responsive to the needs of each particular topic. »
Regarding the 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 in partnering with Magellan Rx to launch a program focused on ensuring proper use while supporting members after determination.
A total of 24 medical benefit drugs were selected for inclusion in the enhanced UM process based on several factors such as annual treatment cost, clinical coverage criteria, and KOL feedback.
From January 1, 2021 to March 30, 2022, 342 applications were received, Chodavapu said, of which 311 were reviewed. Teprotumumab was the most requested drug for review (n=94), followed by pegloticase (n=75) and eculizumab (n=71). The results showed that of the 311 requests, 66 case management referrals have been provided since the program’s inception.
“Some of the most frequently found referral reasons included high pill burden, high pill burden with behavioral health medications, and acute hospitalization or emergency room visits,” he noted. “In order to stratify members, we need to prioritize case management, certain triggers have been built into the workflow, including members’ overall medication list, current illness history, and other clinical criteria that can be found in this clinical documentation.
Since the program’s inception, $19,197,920 in total avoided costs have been realized based on medical claims data alone. Compared to the previous 5 quarters before the launch of the program, total cost avoidance increased by 21.7% compared to traditional Unified Messaging. Additionally, drug cost avoidance under the enhanced UM program accounted for 39.2 percent of total medical cost savings, Chodavapu explained.
Limitations were cited for the results, namely the lack of drug benefit claims, as this limits cost avoidance and savings estimates to only drug claims and the lack of long-term data for quantify the impact of case management on overall quality of life.
“We have found that this program has benefited our clients by ensuring that we are doing everything we can to manage their funds appropriately, while also ensuring that members get appropriate access to these expensive therapies,” a- he concluded. “This collaboration with Magellan also allows us to have a more patient-centered approach with our interventions.”