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    Controversy surrounds Exondys 51 approval: What to know


    On formulary or not, insurers decide

    Health plans have weighed the evidence and among the largest insurers, all but Anthem have put the new drug on formulary, albeit with conditions.

    In addition, Express Scripts, a pharmacy benefits manager based in St. Louis, decided not to include the drug on its national formularies based on the currently available research related to the safety and efficacy of the medication.

    Randy Vogenberg, principal at the Institute for Integrated Healthcare, says that insurers made their decisions about coverage based on a few considerations.

    “Some insurers are trying to slow down rare disease approvals and create better standards around acceptable data for coverage consideration; some have not addressed what to do in a controversial situation like the one surrounding this product at a time when tremendous economic pressure surrounds biologic and specialty drugs,” he says.

    Kaiser’s approach

    Kaiser Permanent put Exondys 51 on its national Part D formulary but not on its California drug list.

    “We cover Exondys 51 when the drug is determined to be medically necessary by a member’s physician, as we do with all FDA-approved pharmaceuticals,” Awsare says. “We continually monitor the latest evidence to ensure that the medications we use are safe and effective. We would not deny any medication that improves life or affects a disease positively.”

    Awsare says the drug is under discussion for use on the California formulary by a team of specialists, including a pediatric neurologist, physical and rehabilitation clinicians, and a geneticist, who will make a recommendation to physicians based on risks and benefits.

    Physicians will receive these guidelines on prescribing Exondys 51, but Awsare says they make the ultimate decision.

    Money matters

    The sticker shock of the new drug—an estimated $300,000 per year—has been diluted somewhat by the arrival of many other expensive specialty drugs reaching the market in the last five years.

    Awsare says that while the new drugs for hepatitis C—Sovaldi (sofosbuvir) and Harvoni (ledipasvir and sofosbuvir) sell for close to $100,000, they had good evidence to back them; however, he admits that some other specialty, high-cost drugs have been approved conditionally by the FDA through its accelerated approval process, Avastin (bevacizumab) for the treatment of malignant brain tumors among them.

    “In addition, as an integrated healthcare system, members have a single premium to use on both medical and pharmacy services so an expensive drug could prevent other patient care,” he says.

    Awsare points out that benefits of Exondys 51 are not as obvious as a drug that lowers cholesterol and prevents hearts attacks or strokes—a “no brainer,” he says.

    Next: What does Anthem think?


    Mari Edlin
    Mari Edlin is a frequent contributor to Managed Healthcare Executive. She is based in Sonoma, California.


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