Rev­o­lu­tion­ary gene ther­apy ap­proved for leukemia — at $475,000 fee

New ap­proach har­vests can­cer pa­tients’ im­mune cells, but price tag is called ex­ces­sive

USA TODAY US Edition - - NEWS - Liz Sz­abo

The na­tion’s first sanc­tioned gene ther­apy — ap­proved Wed­nes­day to fight leukemia that re­sists stan­dard ther­a­pies — will cost $475,000 for a one-time treat­ment, its man­u­fac­turer an­nounced.

The Food and Drug Ad­min­is­tra­tion ap­proved the ther­apy, called Kym­riah, in chil­dren and young adults with acute lym­phoblas­tic leukemia whose dis­ease has come back in spite of pre­vi­ous treat­ments. These pa­tients typ­i­cally have a poor prog­no­sis, surviving three to nine months, ac­cord­ing to No­var­tis, which makes the in­no­va­tive ther­apy.

The Switzer­land-based com­pany said the drug will cost noth­ing if pa­tients fail to ben­e­fit in the first month.

Kym­riah treats can­cer in an en­tirely new way. The in­di­vid­u­al­ized ap­proach in­volves har­vest­ing can­cer pa­tients’ im­mune cells, ge­net­i­cally engi­neer­ing them, then re­turn­ing them to pa­tients’ bod­ies. The ge­netic engi­neer­ing process aims to rev up pa­tients’ im­mune sys­tems to bet­ter fight can­cer.

“We’re en­ter­ing a new fron­tier in med­i­cal in­no­va­tion with the abil­ity to re­pro­gram a pa­tient’s own cells to at­tack a deadly can­cer,” said Scott Got­tlieb, the FDA com­mis­sioner. “New tech­nolo­gies such as gene and cell ther­a­pies hold out the po­ten­tial to trans­form medicine and cre­ate an in­flec­tion point in our abil­ity to treat and even cure many in­tractable ill­nesses.”

No­var­tis is work­ing with 20 hos­pi­tals to pro­vide Kym­riah within a month. Even­tu­ally, the ther­apy will be of­fered at 32 sites, the com­pany said. The first pa­tients could be treated within days. The com­pany is train­ing hos­pi­tals and staff to pro­vide the treat­ment, which can cause a lifethreat­en­ing im­mune re­ac­tion, as well as long-term com­pli­ca­tions.

No­var­tis said it priced its drug based on sev­eral con­sid­er­a­tions. Bri­tish health au­thor­i­ties have said a price of $649,000 for a one­time treat­ment would be jus­ti­fied given Kym­riah’s sig­nif­i­cant ben­e­fits.

No­var­tis also con­sid­ered the cost of bone-mar­row trans­plants, which are given to many leukemia pa­tients whose can­cer re­lapses.

Those trans­plants can cost up to $800,000, No­var­tis said.

An ad­vo­cacy group called Pa­tients for Af­ford­able Drugs re­cently met with of­fi­cials at No­var­tis to ask it to set a “fair” price for the drug, whose early devel­op­ment was sup­ported by $200 mil­lion in fed­eral re­search grants.

David Mitchell, a mul­ti­ple myeloma pa­tient and pres­i­dent of the ad­vo­cacy group, de­scribed the drug ’s price tag as “ex­ces­sive.”

“No­var­tis should not get credit for bring­ing a $475,000 drug to market and claim­ing they could have charged peo­ple a lot more,” he said. “The drug pric­ing sys­tem in Amer­ica is com­pletely bro­ken. Un­til pol­icy in this coun­try changes, the vi­cious cy­cle of pa- tients strug­gling un­der high drug prices will con­tinue.”

The FDA also is con­sid­er­ing a CAR T-cell ther­apy from Cal­i­for­nia-based Kite Pharma. Gilead Sci­ences — which has been crit­i­cized for the $84,000 price tag of its hepati­tis C drug — an­nounced Mon­day that it will buy Kite for $11.9 bil­lion.

No­var­tis of­fi­cials said they will of­fer a pa­tient as­sis­tance pro­gram to help peo­ple with out-of-pocket costs.

Ex­perts have noted that hid­den costs could fur­ther add to pa­tients’ fi­nan­cial bur­dens.

Be­yond the cost of the pro­ce­dure, pa­tients would need to pay for tra­di­tional chemo­ther­apy, which is given be­fore CAR T-cell ther­apy to im­prove its odds of suc­cess. They would also have to foot the bill for travel and lodg­ing to one of the hos­pi­tals equipped to pro­vide the high-tech treat­ment.

Be­cause pa­tients can de­velop life-threat­en­ing side ef­fects weeks af­ter the pro­ce­dure, doc­tors will ask pa­tients to stay within two hours of the hospi­tal for up to a month.

In New York, even bud­get ho­tels cost more than $200 a night — an ex­pense not typ­i­cally cov­ered by in­sur­ance.

Pa­tients who de­velop a dan­ger­ous com­pli­ca­tion, in which the im­mune sys­tem over­re­acts and at­tacks vi­tal or­gans, might need cov­er­age for emer­gency room care.

“The drug pric­ing sys­tem in Amer­ica is com­pletely bro­ken. Un­til pol­icy in this coun­try changes, the vi­cious cy­cle of pa­tients strug­gling un­der high drug prices will con­tinue.” David Mitchell, pres­i­dent of Pa­tients for Af­ford­able Drugs

Kaiser Health News, a non-profit health news­room whose sto­ries ap­pear in news out­lets na­tion­wide, is an ed­i­to­ri­ally in­de­pen­dent part of the Kaiser Fam­ily Foun­da­tion.


Hu­man T cells be­long­ing to can­cer pa­tients ar­rive at No­var­tis Phar­ma­ceu­ti­cals’ fa­cil­ity in Mor­ris Plains, N.J.

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