Traditional specialty drugs for diseases such as rheumatoid arthritis and multiple sclerosis are best served under an open distribution model where multiple specialty pharmacies can take care of the patient population. However, rare disease specialty pharmacy patients require more support and resources to properly provide continuity of care. Thus, a limited distribution model is needed for each rare disease drug to provide a network of specialty pharmacies that can meet these special requirements. The limited distribution network landscape will continue to expand as more rare disease drugs are approved.
In the below chart, the FDA’s Center for Drug Evaluation and Research (CDER) has tracked the approval of orphan and non-orphan drugs over time.1 Over the past few years, the percentage of drug approvals for orphan drugs has been averaging over 50% and, based on the data, one could extrapolate that the orphan drug approval percentage will continue to increase over time.
With the high costs associated with rare disease drugs, it's important for payors to find ways to effectively balance affordability, timely access, and clinical expertise to meet the needs of a rare disease patient. “We must balance costs without sacrificing quality or clinical components to provide the highest value, whether it is under the medical or pharmacy benefit,” noted Chandra Wahrgren, MBA, SVP, Corporate Pharmacy Enterprise & Innovation, Moda Partners.
Working with payors to develop a partnership to meet the unique needs of the member patient and negotiate coverage, rare disease specialty pharmacies help meet the specific patient needs of the pharmaceutical manufacturer while creating value for employers. Rare disease specialty pharmacies provide rare disease clinical expertise, handle complex delivery needs, and provide the “high-touch” patient experience for continuity of care. There is a higher level of focus on a patient’s access to care when developing a rare disease limited distribution network. Also, there are multiple lines of business to consider when developing a limited distribution network.
Taking all of this into account, it is imperative to properly evaluate and choose a specialty pharmacy partner that can provide the optimal experience and support for your rare disease patients. Learn more about how Orsini Specialty Pharmacy partners with payors, patients and manufacturers.
Click here to download our whitepaper detailing what payors should consider when evaluating a specialty pharmacy partner.
1. FDA. CDER Continues to Make Rare Diseases a Priority with Drug Approvals and Programming to Speed Therapeutic Development. Accessed August 15, 2022. https://www.fda.gov/news-events/fda-voices/cder-continues-make-rare-diseases-priority-drug-approvals-and-programming-speedtherapeutic