Healthcare Insights
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.
Which drugs 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.
In this Healthcare Insight, we use the Atlas All-Payor Claims dataset to explore the most prescribed medications used to treat various rare conditions.
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 2025. Note that a patient could have more than one prescription.
| Rank | Drug name | % of prescriptions | Explore dataset |
|---|---|---|---|
| 1 | TRIKAFTA | 3.04% | Explore |
| 2 | CREON | 2.33% | Explore |
| 3 | ALBUTEROL SULFATE HFA | 1.96% | Explore |
| 4 | PULMOZYME | 1.88% | Explore |
| 5 | AZITHROMYCIN | 1.82% | Explore |
| 6 | ALBUTEROL SULFATE | 1.73% | Explore |
| 7 | SODIUM CHLORIDE | 1.53% | Explore |
| 8 | OMEPRAZOLE | 1.29% | Explore |
| 9 | PREDNISONE | 1.28% | Explore |
| 10 | FLUTICASONE PROPIONATE | 1.22% | Explore |
Fig. 1 Data from the Atlas All-Payor Claims dataset. Commercial claims data is sourced from multiple medical claims clearinghouses in the United States and updated monthly. Data accessed March 2026.
Cystic fibrosis (CF) is a progressive, genetic disease that primarily affects the lungs and digestive system. Common medications for patients with CF include Trikafta (targets the defective CFTR protein caused by the genetic mutation), Pulmozyme (helps thin mucus buildup in the lungs), and Creon (provides digestive enzymes).
| Rank | Drug name | % of prescriptions | Explore dataset |
|---|---|---|---|
| 1 | ATORVASTATIN CALCIUM | 2.31% | Explore |
| 2 | PANTOPRAZOLE SODIUM | 2.11% | Explore |
| 3 | GABAPENTIN | 1.94% | Explore |
| 4 | AMLODIPINE BESYLATE | 1.72% | Explore |
| 5 | CREON | 1.69% | Explore |
| 6 | LEVOTHYROXINE SODIUM | 1.61% | Explore |
| 7 | ELIQUIS | 1.55% | Explore |
| 8 | OXYCODONE HCL | 1.54% | Explore |
| 9 | OMEPRAZOLE | 1.39% | Explore |
| 10 | LOSARTAN POTASSIUM | 1.27% | Explore |
Fig. 2 Data from the Atlas All-Payor Claims dataset. Commercial claims data is sourced from multiple medical claims clearinghouses in the United States and updated monthly. Data accessed March 2026.
Common medications for patients with pancreatic cancer include atorvastatin calcium, which studies indicate has potential to inhibit tumor cell proliferation and invasion. Other drugs like pantoprazole sodium (used to treat too much acid in the stomach), Creon® (used to improve food digestion), and Oxycodone HCL (used to treat moderate to severe pain) may also be prescribed to pancreatic cancer patients.
| Rank | Drug name | % of prescriptions | Explore dataset |
|---|---|---|---|
| 1 | GABAPENTIN | 2.96% | Explore |
| 2 | ATORVASTATIN CALCIUM | 2.06% | Explore |
| 3 | BACLOFEN | 1.82% | Explore |
| 4 | LEVOTHYROXINE SODIUM | 1.72% | Explore |
| 5 | DULOXETINE HCL | 1.46% | Explore |
| 6 | AMLODIPINE BESYLATE | 1.28% | Explore |
| 7 | HYDROCODONE-ACETAMINOPHEN | 1.23% | Explore |
| 8 | OMEPRAZOLE | 1.23% | Explore |
| 9 | PANTOPRAZOLE SODIUM | 1.21% | Explore |
| 10 | TRAZODONE HCL | 1.17% | Explore |
Fig. 3 Data from the Atlas All-Payor Claims dataset. Commercial claims data is sourced from multiple medical claims clearinghouses in the United States and updated monthly. Data accessed March 2026.
Multiple sclerosis (MS) is a chronic, inflammatory disease that affects the central nervous system (CNS), which includes the brain, spinal cord, and optic nerves. Some of the frequently prescribed medications for patients with multiple sclerosis include gabapentin, an anticonvulsant used to relieve pain for conditions of the nervous system, and baclofen, which helps relax muscles and relieve spasms, cramping, and tightness caused by multiple sclerosis.
| Rank | Drug name | % of prescriptions | Explore dataset |
|---|---|---|---|
| 1 | GABAPENTIN | 2.38% | Explore |
| 2 | ATORVASTATIN CALCIUM | 2.20% | Explore |
| 3 | LEVOTHYROXINE SODIUM | 1.84% | Explore |
| 4 | PREDNISONE | 1.73% | Explore |
| 5 | PYRIDOSTIGMINE BROMIDE | 1.66% | Explore |
| 6 | AMLODIPINE BESYLATE | 1.44% | Explore |
| 7 | PANTOPRAZOLE SODIUM | 1.43% | Explore |
| 8 | OMEPRAZOLE | 1.27% | Explore |
| 9 | LOSARTAN POTASSIUM | 1.19% | Explore |
| 10 | HYDROCODONE-ACETAMINOPHEN | 1.18% | Explore |
Fig. 4 Data from the Atlas All-Payor Claims dataset. Commercial claims data is sourced from multiple medical claims clearinghouses in the United States and updated monthly. Data accessed March 2026.
Myasthenia gravis (MG) is a chronic autoimmune disease that weakens voluntary muscles. Gabapentin represents the highest share of prescriptions among patients with myasthenia gravis.
| Rank | Drug name | % of prescriptions | Explore dataset |
|---|---|---|---|
| 1 | ATORVASTATIN CALCIUM | 2.49% | Explore |
| 2 | LEVOTHYROXINE SODIUM | 2.14% | Explore |
| 3 | GABAPENTIN | 1.89% | Explore |
| 4 | PANTOPRAZOLE SODIUM | 1.79% | Explore |
| 5 | AMLODIPINE BESYLATE | 1.78% | Explore |
| 6 | FUROSEMIDE | 1.56% | Explore |
| 7 | METOPROLOL SUCCINATE | 1.51% | Explore |
| 8 | ELIQUIS | 1.41% | Explore |
| 9 | LOSARTAN POTASSIUM | 1.27% | Explore |
| 10 | OMEPRAZOLE | 1.24% | Explore |
Fig. 5 Data from the Atlas All-Payor Claims dataset. Commercial claims data is sourced from multiple medical claims clearinghouses in the United States and updated monthly. Data accessed March 2026.
Myelodysplastic syndrome (MDS) is a group of bone marrow disorders that affect blood cell production. Patients with myelodysplastic syndrome might be prescribed drugs like atorvastatin calcium (for high cholesterol) and furosemide (used to treat fluid retention, swelling, and high blood pressure).
| Rank | Drug name | % of prescriptions | Explore dataset |
|---|---|---|---|
| 1 | DEXTROAMPHETAMINE-AMPHETAMINE | 2.22% | Explore |
| 2 | GABAPENTIN | 1.95% | Explore |
| 3 | MODAFINIL | 1.70% | Explore |
| 4 | ATORVASTATIN CALCIUM | 1.66% | Explore |
| 5 | LEVOTHYROXINE SODIUM | 1.60% | Explore |
| 6 | OMEPRAZOLE | 1.25% | Explore |
| 7 | DULOXETINE HCL | 1.24% | Explore |
| 8 | BUPROPION XL | 1.18% | Explore |
| 9 | PANTOPRAZOLE SODIUM | 1.18% | Explore |
| 10 | DEXTROAMPHETAMINE-AMPHET ER | 1.15% | Explore |
Fig. 6 Data from the Atlas All-Payor Claims dataset. Commercial claims data is sourced from multiple medical claims clearinghouses in the United States and updated monthly. Data accessed March 2026.
Narcolepsy is a neurological sleep disorder that affects the brain's ability to regulate sleep-wake cycles. Dextroamphetamine-amphetamine ranks first among medications prescribed to patients with narcolepsy. It helps to keep these patients awake by increasing activity in certain brain chemicals.
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 2025, approximately half of newly approved drugs received orphan designation, according to the FDA’s Center for Drug Evaluation and Research (CDER).
Learn more
For a deeper dive into rare disease trends, read our blog on the latest advancements in rare disease research.
Healthcare Insights are developed with data from the Definitive Healthcare product portfolio and federal sources. Want even more insights? Start a free trial now and get access to the latest intelligence on hospitals, physicians, and other healthcare providers.
