Looming Crisis of Financial Toxicity in Neurologic Drug Pricing

We are experiencing a revolution in neurotherapeutics, with particularly robust growth in new drug approvals for orphan diseases (those affecting <200,000 Americans). Newly approved orphan drugs are expensive. In 2018, the average per patient cost for the top-selling 100 orphan drugs was more than $150,000/y (compared with $33,654 for nonorphan drugs). This trend is driving continued increases in drug costs.¹ The mean per capita prescription drug spending in the United States increased from $668 in 2004 to $1,126 in 2019. Total net medication spending is projected to reach $400 billion by 2025, a 10% increase from 2020.² Generic medications account for 90% of prescriptions but only 19.8% of costs. Rising costs are largely attributable to specialty medications, which accounted for 53% of spending in 2020, double that from 2010.³ Neuroscience orphan drug approvals are second only to those for oncology/hematology.⁴ Skyrocketing branded specialty drug costs are fueling an epidemic of financial toxicity.⁵ More than 20% of prescriptions are never filled, and even modest increases in monthly costs of neurologic drugs result in a significant increase in abandonment risk.⁶ Abandonment for high-priced medications is higher. Half of cancer patients prescribed a drug that costs >$2,000 abandon the prescription.⁷ No matter how effective a disease-modifying therapy may be, if a patient cannot afford the cost, it does not work.