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Delivering global medication solutions
How Aetna International brings effective treatment to members with rare diseases
The impact of a diagnosis
Being diagnosed with a medical condition usually involves learning about treatment options and possibly starting new medications. But what happens when you’re diagnosed with a rare condition that has limited therapy and treatment options that are only available in certain locations?
That’s what happened to Amit1, an Aetna International member whose home was in India. While in the United States, he was diagnosed with Graves’ orbitopathy, also known as Thyroid Eye Disease (TED). TED is estimated to affect 16 in 100,000 women and 2.9 in 100,000 men2, making it considered a rare disease.3
Challenges to accessing a promising new treatment
In January 2020, the U.S. Food and Drug Administration (FDA) approved TEPEZZA® as the first drug treatment available for adults with TED.4 Amit’s physician recommended that he begin the treatment, which involved receiving eight intravenous infusions administered by a medical professional once every three weeks for a total of five months of treatment.5
The problem? Amit was scheduled to return to India before that five-month course of treatment would be complete — but the drug wasn’t available anywhere in the world other than the U.S. And distribution of the medication was so limited that it wasn’t even available to all U.S. pharmacies.
Prescription concierge to the rescue
Thankfully, we already had a system in place to help members get the medications they need through coordinated efforts between our clinical care management team and our international prescription concierge service. In Amit’s case, additional considerations unique to Tepezza and India’s clearance requirements made the delivery more complex.
Our prescription concierge service first had to coordinate within its network of international pharmacy partners to have the medication shipped directly to Amit in India. Because Tepezza had to remain refrigerated at a specific temperature range while in transit,6 a courier service specializing in global biopharma logistics was used so that the proper temperature could be monitored and maintained throughout the journey.
Amit also received needed support and guidance for obtaining the required import permit, confirming pre-clearance of the shipment, and ensuring that the medication would stay at the correct temperature while waiting to be cleared by customs. The happy ending? Amit received the medication and was able to resume his infusions just two days after arriving in India.
A glimmer of hope for those diagnosed with rare diseases
TED is just one of about 7,000 rare diseases affecting an estimated 25 to 30 million Americans, according to the National Organization for Rare Disorders — and more than 90 percent of those lack FDA-approved treatment.7 While this outlook may seem bleak, there is hope, according to Wiley Chambers, M.D., deputy director of the Division of Transplant and Ophthalmology Products in the FDA’s Center for Drug Evaluation and Research.
“[Tepezza] has the potential to alter the course of [thyroid eye] disease, potentially sparing patients from needing multiple invasive surgeries by providing an alternative, non-surgical treatment option,” said Chambers upon the drug’s approval in 2020. “For a variety of reasons, treatments for rare diseases are often unavailable. This approval represents important progress in the approval of effective treatments for rare diseases.”8
1. Name has been changed for privacy.
2.“Thyroid Eye Disease.” Rare Diseases. 2020, rarediseases.org/rare-diseases/thyroid-eye-disease/#:~:text=Thyroid%20eye%20disease%20is%20a,swelling%2C%20and%20tissue%20changes%20occur.
3. “DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration 21 CFR Part 316 [Docket No. FDA-2011-N-0583] RIN 0910-AG72 Orphan Drug Regulations.” Federal Register Vol. 78, No. 133. 12 June 2013, www.govinfo.gov/content/pkg/FR-2013-06-12/pdf/2013-13930.pdf.
4. “FDA approves first treatment for thyroid eye disease.” FDA News Release. 21 January 2020, www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-thyroid-eye-disease.
5. Horizon Therapeutics plc. “TEPEZZA FAQS.” TEPEZZA. September 2022, www.tepezza.com/about-tepezza/frequently-asked-questions.
6. Horizon Therapeutics plc. “TEPEZZA Preparation and Administration Overview.” TEPEZZA. September 2022, www.tepezzahcp.com/starting-patients/tepezza-administration.
7. “Rare Disease Facts.” Rare Diseases. February 2019, rarediseases.org/wp-content/uploads/2019/02/nord-rareinsights-rd-facts-2019.pdf.
8. “FDA approves first treatment for thyroid eye disease.” FDA News Release. 21 January 2020, www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-thyroid-eye-disease.
