Will Aetna Stop Providing an Approved Orphan Drug to Treat Rare Disease Patient?

November 6, 2016

by James Radke, PhD, thanks to Rare Disease Report for this piece.

Orphan drugs are expensive. That is well established. However, the Orphan Drug Act was passed in order to allow companies an opportunity to develop those drugs for small populations. And because those populations are small, annual costs for many of these orphan drugs can run between $300,00 to $600,000.  While that may seem high, the cost to the medical community is relatively low given the small number of patients involved.
For the most part, medical insurance companies are very good at providing these orphan drugs to rare disease patients but like many of us have observed for both orphan and common drugs, there are always hiccups along the way. Right now, one family is experiencing one of those hiccups. Jack Frye, a young man with hunter syndrome may soon be denied the enzyme replacement therapy that has saved his life for the past decade. We talked to Jack’s mother, Kimberly Frye Bennet about her current situation with the insurance company in getting treatment for her son.

What is the current approved treatment for Hunter syndrome?

is the only approved treatment. Jack has been taking the medication for 10 years as of September 11th.

Has treatment improved your son’s quality of life?


Without treatment, what do you expect to happen to your son’s quality of life?

His will deteriorate cognitively and physically.

The last time he did without medication (as a result of Aetna dragging their feet) was when he missed 3 consecutive infusions and I observed a decline in his functioning.

During those missed infusions, his short term memory became more impaired. Things he was able to remember easily were no longer accessible to him. His breathing became more labored. His energy diminished. He could not move as quickly walk the stairs without increased effort or assistance. His range of motion decreased. His hearing was negatively impacted. His ability to toilet on his own changed (i.e.,increased number of accidents).

The loses are hard if not impossible to correct.

Why are you concerned that Aetna will stop providing treatment? 

Through the specialty pharmacy I learned Aetna is reconsidering his lifetime authorization with Accredo healthcare.  I have received nothing in writing from Aetna. It is Accredo healthcare who has reported to me that Aetna is not only refusing to honor their agreement to pay Accredo for the medication they are demanding Accredo reimburse them for medication costs they’ve already covered under this authorization.

 What options are available to you to get the treatment on your own or through another insurance company?

Honestly there are no options. Perhaps Medi Cal will cover it but the process to make that happen is unclear to me. The drug is cost prohibitive ($547,000 annually).

Would you care to comment on the fact that allowing insurance companies to not cover orphan drugs defeats the purpose of the Orphan Drug Act which is to provide treatment options for small patient populations.

Absolutely not. Why develop them just to limit them.


Images of Jack and his family provided by Levi Gershkowitz of Living in the Light. To see more of his photos, visit

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