Tackling the Growing Impact of Orphan Drug Designation and Limited Distribution Networks in Oncology

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).

Understanding the Orphan Designation

The designation of orphan drug has been commonly misunderstood. Image Credit: © Pniuntg - stock.adobe.com

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

Why the Rise in LDDs?

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:

1. Complexity of the Drug: LDDs often require special handling, storage, or administration, which necessitates a pharmacy staff with expertise and the infrastructure to manage these needs.
2. Patient Safety and Support: Manufacturers may restrict distribution to ensure that patients receive the necessary support, including education, monitoring, and adherence programs, from pharmacists with experience and specialized training in therapy management.
3. Regulatory Requirements: Certain drugs may have regulatory constraints that limit their distribution to a specific network, especially if the medication has reporting requirements or a risk evaluation and mitigation strategy.
4. Market Strategy: Manufacturers may also use limited distribution as a market strategy to maintain additional control over the supply chain inventory, minimizing the amount of high-cost drug that is unused or expired, gathering detailed data on the drug's use, or ensuring consistent pricing and reimbursement processes.
5. Patient Support Programs: Expanded manufacturer resources can aid patients through navigating complex insurance requirements, providing financial assistance, and offering access to patient assistance programs.

The Importance of Pharmacy Integration With Distribution Models

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.

Additional Resources

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:

• NORD: https://rarediseases.org/
• NORD patient assistance programs: https://rarediseases.org/patient-assistance-programs/
• FDA orphan drug designation/approvals search: https://www.accessdata.fda.gov/scripts/opdlisting/oopd/

REFERENCES

1. Cancer Facts & Figures 2024. American Cancer Society. 2024. Accessed November 7, 2024. https://www.cancer.org/research/cancer-facts-statistics/all-cancer-facts-figures/2024-cancer-facts-figures.html

2. IQVIA Institute. Orphan Drugs in the United States. National Organization for Rare Disorders. December 2020. Accessed November 7, 2024. https://rarediseases.org/wp-content/uploads/2021/03/orphan-drugs-in-the-united-states-NRD-2020.pdf