Navigating the financial and ethical challenges of high-cost biologicals in neurology

Several high-cost biologicals have been approved for use in treating neurologic disorders, reflecting the complexity and expense of developing and producing these therapies. Below are a few examples, along with the expected annual cost for patients and a discussion of the implications for the healthcare system.

1. Ocrevus® (ocrelizumab)

• Indication. Ocrevus is approved for the treatment of multiple sclerosis, including both relapsing forms of multiple sclerosis and primary progressive multiple sclerosis.
• Cost. The annual cost for Ocrevus is approximately $65,000 to $70,000 per year, depending on the dosage and administration specifics.
• Healthcare implications. Ocrevus is a significant advancement in the treatment of multiple sclerosis, particularly for primary progressive multiple sclerosis, for which there were previously few effective treatments. However, its high cost poses a challenge for healthcare systems, particularly in terms of insurance coverage and accessibility for patients. The expense of long-term treatment can strain both public and private healthcare budgets, leading to difficult decisions about resource allocation.

2. Spinraza® (nusinersen)

• Indication. Spinraza is used to treat spinal muscular atrophy, a genetic disorder characterized by progressive muscle wasting and weakness.
• Cost. The first year of treatment with Spinraza can cost around $750,000, with subsequent annual maintenance treatments costing about $375,000.
• Healthcare implications. Spinraza’s introduction marked a breakthrough in the treatment of spinal muscular atrophy, offering hope to patients with a previously untreatable condition. However, its extraordinarily high cost makes it one of the most expensive drugs on the market, raising concerns about long-term sustainability within healthcare systems. The cost can limit access, with insurance companies and government programs facing pressure to cover such expensive treatments, potentially at the expense of other healthcare services.

3. Zolgensma® (onasemnogene abeparvovec)

• Indication. Zolgensma is a gene therapy approved for the treatment of spinal muscular atrophy in children under 2 years old.
• Cost. Zolgensma is a one-time treatment that costs approximately $2.1 million.
• Healthcare implications. As a one-time treatment, Zolgensma offers a different cost structure compared to chronic therapies, but its price tag is staggering. Although it potentially eliminates the need for ongoing treatment, the upfront cost creates significant financial challenges for healthcare systems. This has sparked debates about pricing models for gene therapies and the need for innovative payment structures, such as annuity-based payments or outcome-based pricing.

4. Soliris® (eculizumab)

• Indication. Soliris is approved for the treatment of neuromyelitis optica spectrum disorder, a rare autoimmune disease that affects the optic nerves and spinal cord.
• Cost. The annual cost of Soliris is approximately $500,000 to $700,000 per year.
• Healthcare implications. Soliris, originally developed for other rare conditions, has a high cost that reflects its status as an orphan drug. The extension of its use to NMOSD further strains healthcare budgets, particularly because neuromyelitis optica spectrum disorder is a chronic condition requiring ongoing treatment. The high cost of Soliris raises questions about the pricing of orphan drugs and the balance between incentivizing innovation and ensuring affordability.

Healthcare system implications

The introduction of these high-cost biologicals presents significant challenges to healthcare systems worldwide. The financial burden of these drugs can lead to increased insurance premiums, higher out-of-pocket costs for patients, and tough decisions about which treatments to cover. For public healthcare systems, the cost of these drugs can lead to budget constraints, forcing trade-offs between funding for high-cost biologicals and other essential healthcare services.

In addition to financial challenges, the high cost of these treatments can also limit access, particularly in lower-income countries or for patients without comprehensive insurance coverage. This raises ethical concerns about equity in healthcare and the need for policies that ensure that all patients, regardless of their financial situation, can access life-saving treatments.

As more high-cost biologicals are developed, there is an increasing need for innovative payment models, such as outcome-based pricing, to ensure that these drugs are affordable and accessible while still incentivizing pharmaceutical companies to invest in developing new therapies.

Related MedLink Neurology Podcast content:

• The Neurotransmitters: Thoughts on the Cost of Neurology Medications

MedLink acknowledges the use of ChatGPT-4, an Artificial Intelligence chatbot, in drafting this blog entry.

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