Exploring Eye-Opening Statistics on Rare Diseases and Healthcare Spending Trends

1. Combined, rare diseases affect nearly 1 in 10 Americans. Even though by definition, a rare disease is a condition that affects fewer than 200,000 people across a broad range of possible disorders, combined, there are at least 7,000+ identified rare diseases. Of these, only 5% have an approved treatment.

(Source: Global Genes, 2023)

2. The average annual cost per patient with a rare disease is approximately $266,000, significantly higher than for common conditions.

(Source: IQVIA Institute, 2023)

3. Rare disease patients represent less than 10% of the insured population but account for over 25% of healthcare spending in some commercial plans.

(Source: Milliman Research, 2022)

4. Orphan drugs (for rare diseases) accounted for 55% of new drug approvals in the U.S. in 2023, a growing share compared to the past decade.

(Source: FDA CDER, 2023)

5. Orphan drugs can cost $100,000 to over $1 million per patient annually, posing major challenges for private and public insurers.

(Source: ICER, 2023)

6. In the U.S., orphan drugs now make up nearly 20% of all prescription drug spending, despite serving fewer than 10% of patients.

(Source: IQVIA, 2023)

7. Over 80% of rare disease patients report delays in diagnosis or misdiagnosis, which can lead to increased utilization and cost for insurers.

(Source: National Organization for Rare Disorders, NORD, 2022)

8. 46% of rare disease patients face insurance denials or delays when seeking treatment, often due to lack of evidence or high cost.

(Source: EveryLife Foundation, 2023)

9. Only 1 in 3 rare disease therapies is covered comprehensively across major U.S. insurance formularies.

(Source: Avalere Health, 2022)

10. The average out-of-pocket cost for rare disease patients is 3–5 times higher than for patients with common chronic illnesses.

(Source: NORC at the University of Chicago, 2022)