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
Rank | Drug name | Number of prescriptions | Explore dataset |
---|---|---|---|
1 | Gabapentin | 169,924 | Explore |
2 | Baclofen | 114,866 | Explore |
3 | Atorvastatin calcium | 93,237 | Explore |
4 | Duloxetine | 79,674 | Explore |
5 | Levothyroxine sodium | 79,105 | Explore |
6 | Hydrocodone bitartrate and acetaminophen | 76,213 | Explore |
7 | Lisinopril | 73,343 | Explore |
8 | Omeprazole | 68,997 | Explore |
9 | Amlodipine besylate | 60,672 | Explore |
10 | Pantoprazole sodium | 54,373 | Explore |
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 | Explore dataset |
---|---|---|---|
1 | Xyrem | 78,329 | Explore |
2 | Xywav | 49,411 | Explore |
3 | Modafinil | 41,588 | Explore |
4 | Gabapentin | 38,172 | Explore |
5 | Dextroamphetamine saccharate, amphetamine aspartate, Dextroamphetamine sulfate and amphetamine sulfate | 30,255 | Explore |
6 | Atorvastatin calcium | 27,977 | Explore |
7 | Levothyroxine sodium | 26,856 | Explore |
8 | Omeprazole | 26,212 | Explore |
9 | Armodafinil | 26,060 | Explore |
10 | Methylphenidate hydrochloride | 25,374 | Explore |
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 | Explore dataset |
---|---|---|---|
1 | Pantoprazole sodium | 26,479 | Explore |
2 | Creon | 25,259 | Explore |
3 | Atorvastatin calcium | 24,594 | Explore |
4 | Gabapentin | 24,357 | Explore |
5 | Oxycodone hydrochloride | 22,908 | Explore |
6 | Amlodipine besylate | 21,902 | Explore |
7 | Omeprazole | 21,251 | Explore |
8 | Hydrocodone bitartrate and acetaminophen | 21,092 | Explore |
9 | Lisinopril | 18,042 | Explore |
10 | Metformin hydrochloride | 17,910 | Explore |
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 | Explore dataset |
---|---|---|---|
1 | Pyridostigmine bromide | 61,364 | Explore |
2 | PredniSONE | 37,115 | Explore |
3 | Atorvastatin calcium | 22,774 | Explore |
4 | Levothyroxine sodium | 21,557 | Explore |
5 | Gabapentin | 21,522 | Explore |
6 | Amlodipine besylate | 16,854 | Explore |
7 | Lisinopril | 16,409 | Explore |
8 | Mycophenolate mofetil | 15,376 | Explore |
9 | Omeprazole | 15,300 | Explore |
10 | Pantoprazole sodium | 14,257 | Explore |
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 | Explore dataset |
---|---|---|---|
1 | Atorvastatin calcium | 22,331 | Explore |
2 | Furosemide | 20,368 | Explore |
3 | Levothyroxine sodium | 19,636 | Explore |
4 | Gabapentin | 18,697 | Explore |
5 | Amlodipine besylate | 17,924 | Explore |
6 | Pantoprazole sodium | 17,460 | Explore |
7 | Omeprazole | 14,208 | Explore |
8 | Metoprolol succinate | 13,827 | Explore |
9 | Hydrocodone bitartrate and acetaminophen | 13,776 | Explore |
10 | Lisinopril | 13,760 | Explore |
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.