Rare Diseases and Orphan Drugs Growth Favorable to Specialty Pharmacies

Over 7,000 rare diseases affect more than 30 million people in the United States. Many rare conditions are life-threatening and most do not have treatments.

Drug, biologic, and device development in rare diseases is challenging for many reasons, including the complex biology and the lack of understanding of the natural history of many rare diseases. The inherently small population of patients with a rare disease can also make conducting clinical trials difficult.

Since the Orphan Drug Act was signed into law in 1983, the FDA has approved hundreds of drugs for rare diseases, but only about 5% of rare diseases have FDA-approved treatments.  

The Biopharmaceutical industry is making progress developing new drugs to treat rare diseases. More than 770 medicines have been approved by the U.S. Food and Drug Administration (FDA) since enactment of the Orphan Drug Act in 1983 and more than 560 medicines are in the current biopharmaceutical pipeline.

Biophama companies are increasingly interested in establishing relationships with  experienced specialty pharmacies to dispense rare disease drugs to patients.

The Accreditation Commission for Health Care launched in 2019 the Specialty Pharmacy industry’s first Distinction in Rare Diseases and Orphan Drugs. PANTHERx was the first pharmacy nationwide to be awarded the Distinction and Orsini Specialty Pharmacy was recently awarded the Distinction also.

The health care accreditation authority URAC created its own Rare Disease Pharmacy Center of Excellence designation in February as an add-on to the specialty pharmacy accreditation.


Takeaway: According to EvaluatePharma’s forecasts, orphan drug sales are set to grow 11 percent each year between now and 2024, significantly outpacing the overall pharma market