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. Since the Orphan Drug Act was signed into law in 1983, the FDA has approved hundreds of drugs for rare diseases, but most rare diseases do not have FDA-approved treatments.
After nearly four decades of relatively consistent growth, orphan drugs are forecast to exceed $200 billion in sales in 2024. That is over 18% of overall prescription sales in 2024. In 2019, just under half (44%) of new FDA approvals went to orphan drugs.
The increase for orphan and rare diseases in the marketplace is resulting in a segment of specialty pharmacy that are focusing on rare diseases. The Accreditation Commission for Healthcare (ACHC), expanded its offerings in specialty pharmacy by launching in 2019 the industry’s first Distinction in Rare Diseases and Orphan Drugs.
The ACHC Distinction in Rare Diseases and Orphan Drugs encompasses processes and procedures necessary to bring medications for rare diseases into market from FDA approval to patient management, employee education, and medication handling and dispensing.
AMI, utilizing its recently launched Specialty Pharmacy Database, has identified the specialty pharmacies at the forefront of this trend and provided profiles of each pharmacy that includes: address, phone, website, accreditation(s), company background, executives, financial/investor information, products and services including for rare diseases, and recent activities.
Rare Disease Focused Specialty Pharmacies’ report Table of Contents:
- Rare Disease Background – ps. 5-6
- Accredo Health – pgs. 7-9
- AllianceRx Walgreens – pgs. 10-11
- Amber Specialty Pharmacy – pgs. 12-13
- AnovoRx Specialty Pharmacy – pgs. 14-16
- AppianRx – pgs. 17-19
- ARJ Infusion Services – pgs. 20-21
- Biologics by McKesson – pgs. 22-23
- BioMatrix Specialty Pharmacy – pgs. 24-26
- BioPlus Specialty Pharmacy – pgs. 27-28
- California Specialty Pharmacy – pgs. 29-30
- Elixir/Rite Aid – pgs. 31-33
- Eversana – pgs. 34-36
- Evolutionary RX – pg. 37
- Heritage Biologics – pgs. 38-40
- KnippeRx – pgs. 41-43
- Optime Rx – pgs. 44-45
- OptumRx/Diplomat – pgs. 46-47
- Orsini Specialty Pharmacy – pgs. 48-50
- PANTHERx Rare – pgs. 51-54
- Soleo Health – pgs. 55-56
- US Bioservices – pgs. 57-59