CAR-T Therapy: Personalized medicine with the promise of a cure; payers will decide who gets it

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The News:

Swiss drug giant Novartis AG has drawn widespread praise and a rising dose of controversy pertaining to its breakthrough CAR-T therapy CTL019. The medicine is under review to treat pediatric and young adult relapsed and refractory B-cell acute lymphoblastic leukemia (ALL).

@Novartis has drawn praise and controversy for its breakthrough CAR-T therapy CTL019 Click To Tweet

But the Food and Drug Administration isn’t the only obstacle for Novartis’s CAR-T, which could be the world’s first. There’s the reality of who will be able to afford the one-time treatment, because it will be mega expensive, FiercePharma points out.

So while FDA approval looms, Novartis is using stem cell transplants as a benchmark for pricing and considering pay-for-performance deals to make the medications accessible for payers and patients alike, R&D chief Vas Narasimhan (pdf) told Bloomberg.

CAR-T therapies are individualized treatments manufactured from a patient’s own T cells, with the promise of a cure. As one-time treatments, just as gene therapies are, they’re expected to be among the world’s most expensive medicines once approved. At the moment, Novartis is vying with Kite Pharma and Juno Therapeutics, among others, which have their own CAR-T products lining up in the FDA queue.

Earlier this year, England’s cost gatekeeper National Institute for Health and Care Excellence (NICE) did the math and found that CAR-T medications could be worth up to $649,000, assuming that patients gain 10 quality-adjusted life years over the current standard of care. At the time, a Novartis spokesperson said “it is too early for us to comment on pricing; however, we will at the time of approval.”

An April ranking of the world’s costliest drugs determined that uniQure Inc.’s gene therapy Glybera surpassed all others at $1.2 million. Weak demand, however, forced the company to drop Glybera since the prices were collected.

Other expensive medicines include Biogen Inc.’s spinal muscular atrophy med Spinraza, with a price tag of $750,000 and BioMarin Inc.’s Brineura to treat the ultra rare CLN2 disease–an inherited disorder that primarily affects the nervous system–at $702,000.

Kite’s axicabtagene ciloleucel is due for FDA action by Nov. 29 in refractory non-Hodgkin’s lymphoma, according to BioPharmCatalyst. EvaluatePharma analysts have assessed the Kite drug, KTE-C19 for non-Hodgkin lymphoma, as one of the top 10 drug launches of 2017.

Steve’s Take:

Most of us want to live longer with a good quality of life. To achieve this, scientists at Novartis and other leading drug developers have sailed into uncharted waters in understanding the cause or mechanism of action of diseases, which is not a simple achievement to say the least.

In order to accomplish this, drug development takes increasingly longer and, therefore, is becoming more and more costly. On average, according to a 2014 study published by Tuft’s Center for the Study of Drug Development, it takes 11 years (range 10–15) to develop a drug from research to approval with a cost of $2.6 billion.

In the past, drug development was mostly focused on small molecules where chemical pathways were well known and where a chemical reaction will occur in the same manner whether the first time or another time, notes Regina Au at BioMarketing Insight.

Then the industry moved into biologics or large molecule where the biology is more complicated, and one cannot predict how a cell is going to react each time. Large molecule drug discovery, therefore, is inherently more expensive and scientists also had to figure out a way to get these large molecules to the right target.

Today, there is immuno-oncology (IO) therapy, which is extremely complex compared to small or large molecule. I-O uses drugs known as immunotherapies that target the body’s immune system to help fight cancer. Scientists understand how the immune system works, but not thoroughly enough to know how it will react when one starts to manipulate the human immune system.

In calculating the cost to produce a CAR-T therapy, because the complex process has to be conducted separately for each individual, it becomes extremely costly. Manufacturing costs usually come down when there is economy of scale, but with CAR-T therapy, there is no economy of scale since it is personalized to each individual. The cost of this therapy can only be determined by the biotech company that is actually developing the CAR-T therapy.

This is the dilemma. Society wants personalized medicine yet who is going to pay for the cost of it? Insurance providers will not pay for CAR-T therapy because it presently is unproven by regulatory standards as well as their own company standards; the side effect profile is risky even though it can be managed, and it is very expensive. Today, most insurance companies will only pay for the standard treatments and it’s only when all other therapies fail will they consider cellular therapy.

It’s ideal if we can 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.

Bottom Line:

The actual answer to who gets access to these extremely costly “wonder drugs” relies on the payer, Ms. Au posits. That’s because an insurance company will not pay for a new therapy unless it is proven that CAR-T therapy, for example, works and is a cure, by their standards not just the FDA’s, it is safe, and it saves the insurance company money.

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 have to pay for it. 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.

Steve's Take: CAR-T therapies should be covered by #healthinsurance Click To Tweet

These new therapies should be covered by insurance. When donor CAR-T cells from another person are demonstrated to work as well as the patient’s own CAR-T cells, the cost of therapy will almost certainly decrease appreciably.

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

In order 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.

We need to alter the national mindset such that everyone unconsciously practices preventive care, takes responsibility for their own health, embraces the notion of treating the right patient with the right drug, with the freedom of advance technology being covered by insurance, and encourages the principle that not every disease needs to be treated with a nuclear missile when a small caliber pistol will achieve the same outcome.

If we don’t, the healthcare cost dilemma in the US will persist as a knotty, national scourge for which there will continue to be plenty of debate, but no cure.