Most prescribed medications for patients with rare diseases

Up to 30 million Americans may be living with a rare disease. Individual rare diseases affect fewer than 200,000 people each and there may be as many as 7,000 different conditions.

How many medications have received FDA orphan drug designation?

The Orphan Drug Act of 1983 started providing financial incentives to pharmaceutical companies to explore more treatments for rare diseases. Over 5,000 drugs and biologics received an orphan drug designation between 1983 and 2019 and over 700 of the designations have an associated approval. Top therapeutic areas for orphan drug development include oncology, neurology and infectious disease.

What medications are prescribed to patients with rare diseases?

While these orphan drugs help to cure and provide relief to symptoms related to rare conditions, patients with rare diseases may also have additional prescriptions they take to combat symptoms or treat co-morbidities.

For this analysis, we reviewed data across 142 rare disease diagnoses and 38 categories from the Definitive Healthcare ClaimsMx product. Some of the condition categories analyzed include amyotrophic lateral sclerosis (ALS), common variable immunodeficiency, cystic fibrosis, Duchenne muscular dystrophy, Fragile X syndrome, Morquio syndrome, multiple sclerosis (MS), pancreatic cancer, scleroderma and spinal muscular atrophy. We then reviewed the prescription medications used by these patients from our ClaimsRx product.

The tables below show the rare diseases from the 38 identified categories with the highest number of patient prescriptions and list the top medications prescribed to those patients.