Most prescribed medications for patients with rare diseases

Rare diseases, while individually affecting a small number of patients, collectively pose a significant global health burden. Estimates suggest more than 400 million people worldwide live with a rare disease, with researchers identifying about 8,000 distinct conditions.

Orphan drugs, or drugs developed for rare diseases, can provide a critical lifeline for rare disease patients with limited treatment options. By analyzing prescription claims data, we can gain crucial insights into orphan drug utilization and the broader rare disease market. In this Healthcare Insight, we use the Atlas All-Payor Claims dataset to explore the most prescribed medications used to treat various rare conditions.

What is the market for rare disease drugs?

The market for orphan drugs is relatively steady, driven by scientific advancements, regulatory incentives like the Orphan Drug Act, and overall prescription drug market growth. While the development and commercialization of orphan drugs come with unique challenges, the potential for high returns and significant impact on patient lives make this an attractive sector. In 2023, more than half of the FDA’s Center for Drug Evaluation and Research (CDER) new drug approvals were orphan-designated.

How many medications have received FDA orphan drug designation?

The Orphan Drug Act, enacted in 1983 by the U.S. Congress, offers various incentives to encourage the development of treatments for rare diseases. From 1983 to 2022, 6,340 orphan drug designations were granted, representing drug development for 1,079 rare diseases. Top therapeutic areas for orphan drug development include oncology, neurology, and infectious disease.

What medications are prescribed to patients with rare diseases?

Medications prescribed for rare disease treatment often include a mix of specialized treatments, like orphan drugs, developed specifically for rare conditions and more commonly used drugs that address symptoms or associated health issues.

What are the most common drugs for patients with rare diseases?

For this analysis, we reviewed data across six rare disease diagnoses: cystic fibrosis, malignant neoplasm of the pancreas, multiple sclerosis, myasthenia gravis and other myoneural disorders, myelodysplastic syndrome, and narcolepsy. We then reviewed drugs prescribed to these patients using the Atlas All-Payor Claims dataset. The tables below show our list of rare diseases with the highest share of patient prescriptions and list the top medications prescribed to those patients in CY 2023.