About the Authors
Stacey McCullough, PharmD, is the chief pharmacy officer at NCODA.
Kara Sammons, MSPharmReg, CPhT, is a senior manager - engagement and advisory at NCODA.
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Pharmacy teams are learning to adapt to the challenges of limited distribution networks and orphan drug designation in oncology, with integrated pharmacy support playing a crucial role in enhancing patient access and adherence.
The increasing prevalence of medications that require the pharmacy team to navigate prior authorization and limited distribution networks is a challenge that is only expected to increase with the robust oncology pipeline in development. As part of a series on these emerging changes, I want to share a look at the impact within the oncology landscape of orphan drug designation and limited distribution drugs (LDD).
Cancer is a dominant health challenge for the estimated 1.9 million Americans diagnosed annually, according to the American Cancer Society.1 A significant percentage of those cancer diagnoses impact just a few thousand people per condition, and because of that, many cancer therapies fall within the threshold for orphan drug designation, which means the disease affects 200,000 Americans or fewer.
However, the designation of orphan drug has been commonly misunderstood. Both consumers and professionals may think of orphan drugs as extremely rare or last-resort treatments, but it’s important to understand that many of these approved medications are widely utilized, first-line therapies. Additionally, over 40% of orphan drug act approvals since 2010 have been for cancer treatments, according to data from the National Organization for Rare Disorders (NORD).2
A limited distribution drug is a medication that will be available only from a restricted number of pharmacies. Manufacturers may make the decision to use this model for orphan drugs or other special education and monitoring, precautions, or safety factors, including:
Stacey McCullough, PharmD, is the chief pharmacy officer at NCODA.
Kara Sammons, MSPharmReg, CPhT, is a senior manager - engagement and advisory at NCODA.
Due to designations such as an orphan drug or LDD, the fulfillment process for oral anti-cancer medications can be complex. The approach requires a highly coordinated pharmacy team to navigate benefits investigation, insurance coverage, prior authorizations, financial assistance, and patient education, in addition to drug procurement, proper handling, storage, dispensing, and delivery. By addressing logistical challenges and barriers, I believe medically integrated pharmacy teams can work together to enhance the patient experience by improving access to care, supporting sustainable affordability, providing tailored education to clarify therapy goals, and empowering patients in pursuing optimal medication adherence.
Stay tuned for part 2 of this series, where we’ll take a closer look at specialty medications and distribution systems, examining how these models impact patient access and care.
Has your practice experienced challenges in accessing orphan drugs or essential medications due to insurance plan design? If so, please let NCODA know at contact@ncoda.org. We are actively collaborating with our partners and members to provide resources for these critical issues affecting oncology patients nationwide.
For more information on orphan drugs and rare disease treatments, visit:
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