“Since the discovery of the JAK2 mutation in 2005, there has been tremendous excitement about the development of selective JAK2 inhibitors as a new treatment for MPNs. After six years of waiting, several drugs are in advanced clinical trials and one may soon be available for patients. Given these new drugs and other lines of clinical investigation, there are many reasons to be optimistic about the prospects for a cure.” - John Crispino, PhD, Northwestern Memorial Hospital, Scientific Advisor for MPN Research Foundation

Drug Reimbursement

Off-label drugs and the compendia

Many MPN patients are struggling to get insurance coverage for their medicine. This is because most treatments prescribed are not United States Food and Drug Administration (FDA) approved for MPNs and are considered ‘off-label’ -- meaning they are for indications other than those referenced in the FDA approved label. Hydrea, Pegasys and IntronA are FDA approved drugs, but are not FDA approved for MPNs.

Approximately half of all anticancer drugs are prescribed off-label. Some managed care organizations and private health insurance plans have declined to reimburse the cost of drugs used ‘off-label’ to treat cancer on the ground that these uses are “experimental” or “investigational.”

The Fee-for-Service Medicare program recognizes certain published compendia as authoritative references to identify medically accepted, unlabeled uses of drugs in anticancer treatment regimens. Some insurers refer to compendia when making policy decisions, thus creating a strong financial incentive for manufacturers to obtain a favorable compendium recommendation.

Of the four compendia, only two mention Hydrea for off-label use in Polycythemia Vera and none mention Pegasys which is growing in use among PV patients. Hydrea is typically universally reimbursed for all MPNs due to its compendia listing and relative low cost. This is not the case for Pegasys. Many MPN patients are experiencing insurance denials for Pegasys. Pegasys is a pegylated form of Interferon alpha made by Roche. It is FDA approved for Hepatitis C and as a result of its efficacy and tolerance in MPN phase II studies it is being prescribed more often in treatment for MPNs. While some patients are fortunate to have Pegasys approved upon initial prescription by their physician, many are being denied coverage immediately or after one year of treatment because it’s not FDA approved for MPNs.

Additional information on the history of off-label drug use can be found here.

What does all this mean for MPNs? Some of the more common treatments for MPNs include IntronA, Hydrea and Pegasys. None of these are FDA approved for MPN and thus are being prescribed off-label. The MPN Research Foundation is currently looking into encouraging drug companies to apply for compendia listings

Documentation for Pegasys Appeals Process

How one patient persuaded her insurance company to pay for Pegasys


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