Healthcare Insights

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

RankDrug nameNumber of prescriptionsExplore dataset
1Gabapentin169,924Explore
2Baclofen114,866Explore
3Atorvastatin calcium93,237Explore
4Duloxetine79,674Explore
5Levothyroxine sodium79,105Explore
6Hydrocodone bitartrate and acetaminophen76,213Explore
7Lisinopril73,343Explore
8Omeprazole68,997Explore
9Amlodipine besylate60,672Explore
10Pantoprazole sodium54,373Explore

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

RankDrug nameNumber of prescriptionsExplore dataset
1Xyrem78,329Explore
2Xywav49,411Explore
3Modafinil41,588Explore
4Gabapentin38,172Explore
5Dextroamphetamine saccharate, amphetamine aspartate, Dextroamphetamine sulfate and amphetamine sulfate30,255Explore
6Atorvastatin calcium27,977Explore
7Levothyroxine sodium26,856Explore
8Omeprazole26,212Explore
9Armodafinil26,060Explore
10Methylphenidate hydrochloride25,374Explore

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

RankDrug nameNumber of prescriptionsExplore dataset
1Pantoprazole sodium26,479Explore
2Creon25,259Explore
3Atorvastatin calcium24,594Explore
4Gabapentin24,357Explore
5Oxycodone hydrochloride22,908Explore
6Amlodipine besylate21,902Explore
7Omeprazole21,251Explore
8Hydrocodone bitartrate and acetaminophen21,092Explore
9Lisinopril18,042Explore
10Metformin hydrochloride17,910Explore

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

RankDrug nameNumber of prescriptionsExplore dataset
1Pyridostigmine bromide61,364Explore
2PredniSONE37,115Explore
3Atorvastatin calcium22,774Explore
4Levothyroxine sodium21,557Explore
5Gabapentin21,522Explore
6Amlodipine besylate16,854Explore
7Lisinopril16,409Explore
8Mycophenolate mofetil15,376Explore
9Omeprazole15,300Explore
10Pantoprazole sodium14,257Explore

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

RankDrug nameNumber of prescriptionsExplore dataset
1Atorvastatin calcium22,331Explore
2Furosemide20,368Explore
3Levothyroxine sodium19,636Explore
4Gabapentin18,697Explore
5Amlodipine besylate17,924Explore
6Pantoprazole sodium17,460Explore
7Omeprazole14,208Explore
8Metoprolol succinate13,827Explore
9Hydrocodone bitartrate and acetaminophen13,776Explore
10Lisinopril13,760Explore

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.