Kaiser report says America hitting the wall as new gene-therapies rise above $1 million; “personalized” medicine looks great, but we won’t all get access to it.

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The News:

Posted by Kaiser Health News–on October 17, 2017

Out­rage over the high cost of can­cer care has fo­cused on sky­rock­et­ing drug prices, in­clud­ing the $475,000 price tag for the coun­try’s first gene ther­apy, No­var­tis’s Kym­riah, a leukemia treat­ment ap­proved in Au­gust.

But the total costs of Kym­riah and the 21 sim­i­lar drugs in de­vel­op­ment–known as CAR T-cell ther­a­pies–will be far higher than many imagine, reach­ing $1 mil­lion or more per pa­tient, ac­cord­ing to lead­ing can­cer ex­perts. The next CAR T-cell drug could be ap­proved as soon as No­vem­ber.

Al­though Kym­riah’s price tag has “shat­tered on­col­ogy drug pric­ing norms,” said Leonard Saltz, chief of gas­troin­testi­nal on­col­ogy at Memo­r­ial Sloan Ket­ter­ing Can­cer Cen­ter in New York, “the sticker price is just the start­ing point.”

According to Saltz, the side effects of these therapies add to the cascade of costs with which consumers are confronted because they require sophisticated management. For this class of drugs, Saltz ad­vised con­sumers to “think of the $475,000 as parts, not labor.”

Dr. Hagop Kan­tar­jian, a leukemia spe­cial­ist and pro­fes­sor at the Uni­ver­sity of Texas MD An­der­son Can­cer Cen­ter, es­ti­mates Kym­riah’s total cost could reach $1.5 mil­lion.

CAR T-cell ther­apy is ex­pen­sive be­cause it requires doctors to har­vest pa­tients’ im­mune cells, ge­net­i­cally alter them to amp up their abil­ity to fight can­cer, then re­in­fuse them into pa­tients.

Kan­tar­jian added that tak­ing the brakes off the im­mune sys­tem can lead to life-threat­en­ing com­pli­ca­tions that re­quire lengthy hos­pi­tal­iza­tions and ex­pen­sive med­ica­tions, which are pre­scribed in ad­di­tion to con­ven­tional can­cer ther­apy, rather than in place of it.

Dr. Keith Eaton, like nearly half of pa­tients who re­ceive CAR T-cell ther­apy, de­vel­oped a life-threat­en­ing com­pli­ca­tion in which his im­mune sys­tem over­re­acted. He says he feels for­tu­nate to be healthy today.

Dr. Eaton, a Seat­tle on­col­o­gist, said he ran up med­ical bills of $500,000 when he par­tic­i­pated in a clin­i­cal trial of CAR T cells in 2013, even though all pa­tients in the study re­ceived the med­ica­tion for free. Eaton, who suf­fered from leukemia, spent nearly two months in the hos­pi­tal.

Like Eaton, nearly half of pa­tients who re­ceive CAR T cells de­velop a se­vere or life-threat­en­ing com­pli­ca­tion called “cy­tokine storm,” in which the im­mune sys­tem over­re­acts, caus­ing dan­ger­ously high fevers and sud­den drops in blood pres­sure. These pa­tients are typ­i­cally treated in the in­ten­sive care unit, and they are often faced with se­ri­ous side ef­fects in­cluding stroke-like symp­toms and coma.

Eaton, now 51, said the cy­tokine storm felt like “the worst flu of your life.” His fever spiked so high that a hos­pi­tal nurse as­sumed the ther­mome­ter was bro­ken, to which he replied, “It’s not bro­ken. My tem­per­a­ture is too high to reg­is­ter on the ther­mome­ter.”

Following his recovery, Eaton was recommended for a bone-marrow transplant–an­other har­row­ing pro­ce­dure—which cost hun­dreds of thou­sands of dol­lars. Tests now show no ev­i­dence of leukemia. His in­surer paid for al­most every­thing.

To subdue un­rest about price, No­var­tis will charge for the drug only if pa­tients go into re­mis­sion within one month of treat­ment. In a key clin­i­cal trial, 83% of the chil­dren and young adults treated with Kym­riah went into re­mis­sion within three months. No­var­tis calls the plan “out­comes-based pric­ing.”

Com­pany spokes­woman Julie Masow said, “costs will vary from pa­tient to pa­tient and treat­ment cen­ter to treat­ment cen­ter, based on the level of care each pa­tient re­quires.” She added that, “Kym­riah is a one-time treat­ment that has shown re­mark­able early, deep and durable re­sponses in these chil­dren who are very sick and often out of op­tions.”

Some doc­tors said Kym­riah, which could be used by about 600 pa­tients a year, of­fers an in­cal­cu­la­ble ben­e­fit for des­per­ately ill young peo­ple. Kym­riah is ap­proved for chil­dren and young adults with a type of acute lym­phoblas­tic leukemia and al­ready have been treated with at least two other can­cer ther­a­pies.

“A kid’s life is price­less,” said Dr. Michelle Her­mis­ton, di­rec­tor of pe­di­atric im­munother­apy at UCSF Be­nioff Chil­dren’s Hos­pi­tal San Fran­cisco. “Any given kid has the po­ten­tial to make fi­nan­cial im­pacts over a life­time that far out­weigh the cost of their cure. From this per­spec­tive, every child in my mind de­serves the best cu­ra­tive ther­apy we can offer.”

Other can­cer doc­tors say the No­var­tis plan is no bar­gain.

About 36 per­cent of pa­tients who go into re­mis­sion with Kym­riah re­lapse within one year, said Dr. Vinay Prasad, an as­sis­tant pro­fes­sor of med­i­cine at Ore­gon Health & Sci­ence Uni­ver­sity. Many of these pa­tients will need ad­di­tional treat­ment, said Prasad, who wrote an ed­i­to­r­ial about Kym­riah’s price Oct. 4 in Na­ture.

“If you’ve paid half a mil­lion dol­lars for drugs and half a mil­lion dol­lars for care, and a year later your can­cer is back, is that a good deal?” asked Saltz, who co-wrote a re­cent ed­i­to­r­ial on Kym­riah’s price in JAMA.

(By Liz Szabo. Orig­i­nally posted at Kaiser Health News, a na­tional health pol­icy news ser­vice that is part of the non­par­ti­san Henry J. Kaiser Fam­ily Foun­da­tion.)

Steve’s Take:

I’ve been extremely fortunate to run marathons ranging from the breath-taking beauty of the “Big Sur” in Carmel, CA, to the New York City event, which meanders through all five boroughs. They’re both 26.219 miles long.

And everyone I’ve known who’s ever run a marathon eventually “hits the wall” toward the end. The body is tapped out of the fuel needed to continue running, it starts to feel like jello, and your whole world shrinks to the size of a ping-pong ball.

Boiling it down, the Kaiser report essentially says America is now, or very soon will be, hitting the same type of wall with the cost of immuno-oncology (IO) therapy.

And although the report doesn’t say it explicitly, this nation will not be able to afford to make this wondrous treatment available to everyone. Do the math per Kaiser’s whole cost-of-care calculation–not just the cost of the “medicine,”–and it becomes quite clear.

In simple terms, the dilemma, already faced in other “rich” nations like the UK and Canada, is we all want personalized medicine. But at a total cost-of-care price of around $1.5 million per patient, who is going to pay for it?

As I’ve written before, most insurance companies will only pay for the standard treatments, and they will only consider cellular therapy when all other options fail. Ideally, we use our own immune system to fight cancer, and the therapy would be a one-event cure as opposed to traditional treatments including checkpoint inhibitors, where the patient takes the med for a specific period of time and hopes the cancer is eradicated.

Adding insult to injury, a new study shows that many of these pricey cancer drugs don’t even work.

Bottom Line:

The payer determines who gets access to these extremely costly “wonder drugs.” That’s because insurance companies will not pay for a new therapy unless it is proven that it works and is in fact a “cure” (equals no relapse after initial remission) by their standards not just the FDA’s. It also must be proven to be safe and save the insurance company money.

Steve's Take: We need a national solution to paying for expensive new #genetherapy treatments Click To Tweet

But in order to ascertain this, economic data must be collected for a predetermined period of time not just to demonstrate these factors, but also that the med saves the company money compared to “standard of care” treatment. This can be generic versions of the drug or biosimilar of a biologic drug.

If the insurance company will not cover the ultra-expensive, single-dose therapy, then the patient or family will. Most patients and families cannot afford these potential cures and they instead will be forced to rely upon the standard treatment(s) and cross their fingers.

One intractable dilemma, however, is that as people live longer, they require more health care because of additional diseases or disorders concurrent with a primary disease or disorder like cancer. These comorbidities, as they are known, automatically jack up the cost of care.

To prevent healthcare costs from mushrooming out of control, especially now with CAR-T and other budding programs, the entire US healthcare system has to shift in a fundamental manner not seriously addressed in either Obamacare or the recent GOP proposals to repeal and replace it.

As in the UK and Canada, among other nations, we need to alter our national mindset so everyone unconsciously practices preventive car and takes responsibility for their health, embraces the notion of treating patients with the right drug–with the freedom of advanced technology being covered by insurance.

We’re now “hitting the wall” on the cost issue. Whether it’s a universal payer system, a government-run and -funded system or a hybrid like it is currently, we’re much closer with these new gene therapies to making a national decision which way we’re going.