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.

Top 10 prescriptions for patients with multiple sclerosis

Rank Drug name Number of prescriptions
1 Gabapentin 169,924
2 Baclofen 114,866
3 Atorvastatin calcium 93,237
4 Duloxetine 79,674
5 Levothyroxine sodium 79,105
6 Hydrocodone bitartrate and acetaminophen 76,213
7 Lisinopril 73,343
8 Omeprazole 68,997
9 Amlodipine besylate 60,672
10 Pantoprazole sodium 54,373

Fig. 1  Data from the Definitive Healthcare ClaimsMx and ClaimsRx product. Commercial claims data is sourced from multiple medical claims clearinghouses in the United States and updated monthly. Data accurate as of February 2022.

Some of the most common prescriptions for patients with multiple sclerosis include gabapentin, an anticonvulsant used to relieve pain for conditions of the nervous system, and baclofen that helps relax muscles and relieve spasms, cramping and tightness caused by multiple sclerosis.

Top 10 prescriptions for patients with narcolepsy

Rank Drug name Number of prescriptions
1 Xyrem 78,329
2 Xywav 49,411
3 Modafinil 41,588
4 Gabapentin 38,172
5 Dextroamphetamine saccharate, amphetamine aspartate, Dextroamphetamine sulfate and amphetamine sulfate 30,255
6 Atorvastatin calcium 27,977
7 Levothyroxine sodium 26,856
8 Omeprazole 26,212
9 Armodafinil 26,060
10 Methylphenidate hydrochloride 25,374

Fig. 2  Data from the Definitive Healthcare ClaimsMx and ClaimsRx product. Commercial claims data is sourced from multiple medical claims clearinghouses in the United States and updated monthly. Data accurate as of February 2022.

The top drug used by patients with narcolepsy is Xyrem® (sodium oxybate), an orphan drug developed to treat symptoms associated with narcolepsy.

Top 10 prescriptions for patients with pancreatic cancer

Rank Drug name Number of prescriptions
1 Pantoprazole sodium 26,479
2 Creon 25,259
3 Atorvastatin calcium 24,594
4 Gabapentin 24,357
5 Oxycodone hydrochloride 22,908
6 Amlodipine besylate 21,902
7 Omeprazole 21,251
8 Hydrocodone bitartrate and acetaminophen 21,092
9 Lisinopril 18,042
10 Metformin hydrochloride 17,910

Fig. 3  Data from the Definitive Healthcare ClaimsMx and ClaimsRx product. Commercial claims data is sourced from multiple medical claims clearinghouses in the United States and updated monthly. Data accurate as of February 2022.

Common medications for patients with pancreatic cancer include pantoprazole sodium (used to treat too much acid in the stomach) and Creon® (pancrelipase) (used to improve food digestion).

Top 10 prescriptions for patients with myasthenia gravis and other myoneural disorders

Rank Drug name Number of prescriptions
1 Pyridostigmine bromide 61,364
2 PredniSONE 37,115
3 Atorvastatin calcium 22,774
4 Levothyroxine sodium 21,557
5 Gabapentin 21,522
6 Amlodipine besylate 16,854
7 Lisinopril 16,409
8 Mycophenolate mofetil 15,376
9 Omeprazole 15,300
10 Pantoprazole sodium 14,257

Fig. 4  Data from the Definitive Healthcare ClaimsMx and ClaimsRx product. Commercial claims data is sourced from multiple medical claims clearinghouses in the United States and updated monthly. Data accurate as of February 2022.

Pyridostigmine bromide, used to improve muscle strength, has the most prescriptions among patients with myasthenia gravis.

Top 10 prescriptions for patients with myelodysplastic syndrome

Rank Drug name Number of prescriptions
1 Atorvastatin calcium 22,331
2 Furosemide 20,368
3 Levothyroxine sodium 19,636
4 Gabapentin 18,697
5 Amlodipine besylate 17,924
6 Pantoprazole sodium 17,460
7 Omeprazole 14,208
8 Metoprolol succinate 13,827
9 Hydrocodone bitartrate and acetaminophen 13,776
10 Lisinopril 13,760

Fig. 5  Data from the Definitive Healthcare ClaimsMx and ClaimsRx product. Commercial claims data is sourced from multiple medical claims clearinghouses in the United States and updated monthly. Data accurate as of February 2022.

Patients with myelodysplastic syndrome most commonly take atorvastatin calcium (for high cholesterol) and furosemide (used to treat fluid retention, swelling and high blood pressure).

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Healthcare Insights are developed with healthcare commercial intelligence from the Definitive Healthcare platform. Want even more insights? Check out our Rare Disease Study Infographic or start a free trial now.

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