Medical Directors Forum

Managing Cost of Niche Diseases Depends On Innovative Approaches


Mike Cantor, MD, JD
Chief Medical Officer, CareCentrix, Hartford

New treatments for rare or “niche” diseases have the potential to positively impact the lives of millions of patients. But they also bring new challenges for health plans struggling to manage rising diagnostic and treatment costs. The rapid increase in pharmacy costs is an especially difficult problem. Although there is no “silver bullet” to these challenges, two approaches can help assure better care for patients with niche diseases as well as manage costs.

First, providers should make sure that prescribed therapies are titrated according to accepted clinical guidelines.

Second, when clinically appropriate, as much care as possible should be provided in the patient’s home.

Consider as an example the management of chronic inflammatory demyelinating polyneuropathy (CIDP), a neurological disorder characterized by progressive weakness and impaired sensory function in the legs and arms. CIDP is closely related to Guillain-Barre syndrome, and some experts believe that it is the chronic form of the syndrome.

Mike Cantor, MD, JD

Mike Cantor, MD, JD
Chief Medical Officer, CareCentrix, Hartford

Treatment of CIDP often includes intravenous immunoglobulin (IVIG), which costs thousands of dollars per dose. Health plans should evaluate their infusion networks and specialty pharmacy vendors to ensure use of standardized approaches to IVIG treatment. The quality of IVIG treatment of patients with CIDP can be improved by using a protocol-driven approach that uses clinical measurements to determine appropriate dosing. In many cases, patients are started on IVIG and dose adjustments that would benefit the patient are made too late. What’s more, dose adjustments are sometimes made without careful consideration of objective data to guide dose escalation or reduction.

Successful new clinical programs use dosing protocols for IVIG based on just this kind of objective measurement. It is called the inflammatory neuropathy cause and treatment (INCAT) score.

A recent study published in the Annals of Neurology demonstrated that patients treated in a way that follows dosing protocols were more likely to have stable or improved INCAT scores compared with patients where physicians chose not to follow the protocol-based recommendation.

Moreover, this approach may reduce costs by more quickly identifying patients who are not responding to therapy, so the dose can be changed or the patient switched to medications that are less expensive than IVIG.

Another approach to reducing costs for treatment of CIDP is to move IVIG dosing from infusion centers to patient homes. Home infusions can be between 30% and 40% less expensive than treatment in an infusion center.

Patients are often not aware that home IVIG infusion is a safe—and less expensive—alternative. And in this era of rising copayments and coinsurance and high-deductible plans, patients as well as insurers benefit from the cost savings.

There’s no question that the advent of new treatments for niche illnesses is improving the quality of life and providing hope for patients with rare diseases. Previously, there were few, if any, treatments.

But health plan leaders can’t be caught flat-footed. They need to keep pace with the introduction of these new treatments and be ready with strategies that address patient needs and manage costs. Using objective clinical data to guide dosing and working to redirect care—when appropriate—to a patient’s home is a place to start.

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