CAR-T therapies are one of the most recently developed revolutionary cancer treatments and certainly one of the most hyped innovations in the Biotech space.
Clinical trials have shown rather unprecedented remission rates of up to 94% in some severe forms of cancer. Given that most of the trials have recruited patients that have not responded to all other treatments, the observed efficacy data were particularly impressive and boosted patients, HCPs and investor expectations. However, it has not been all smooth sailing and it is important to remember that the CAR-T cells have been linked to severe side effect such as neurotoxicity and cytokine release syndrome.
Nevertheless and rightly so, two of the tested CAR-T therapies have been granted an FDA approval. Back in August 2017, Novartis received first ever FDA approval for Kymriah to treat patients with acute lymphoblastic leukaemia. On October 18, the FDA approved a CAR-T therapy called Yescarta, developed by Kite Pharma, to treat adult patients with certain types of large B-Cell lymphoma who have not responded to or have relapsed after at least two other kinds of treatment.
These two groundbreaking therapies are not cheap. What is interesting, however, is the price difference between the two: while Novartis charges $475,000 for Kymriah, Kite/Gilead will sell Yescarta for $373,000 (in both cases, the price does not include any hospital expenses associated with patients T cell collection for genetic re-engineering and later on administration).
You could wonder how can you justify a difference of over $100,000 for therapies which share many similarities including a fairly complex manufacturing process. It is interesting to point out that the size of target audience for the two therapies differ. Kymriah's acute lymphoblastic leukemia is much smaller in terms of patient numbers compared to B-cell lymphomas for which Yescarta was approved.
I think it is difficult to argue that the CAR-T prices are not high but in the grand scheme of things, these innovative solutions come short of some other recent launches (e. g. Biogen Spinraza costs $750,000 for the first year of treatment of muscular atrophy).
Both Kymriah and Yescarta are intended to be one-time treatments to cure a patient's cancer. On top of that it is very likely these treatments will be prescribed on the 'pay-for-performance' basis meaning payments will not be collected if patients do not respond to the re-engineered T cells.
It remains to be seen how the pricing of the CAR-Ts will evolve over the next few years. I think we will not have to wait long before we see some change. The research will continue and the technology will evolve. An allogeneic CAR-T therapy i.e sourced from a healthy donor and ready to go when the patient needs it could potentially change the dynamics of treatment: quicker administration, lower cost.
Despite the many challenges, the CAR-T technology will revolutionize the way we treat cancer in my opinion. With time it will be refined to something truly game changing, potentially moving CAR-T to earlier lines. For now, both Kymriah and Yescarta will be used in patients who lack any other therapeutic solutions giving them hope to survive.
Revoir, who participated in a clinical trial, had failed two rounds of chemo, making her ineligible for a bone marrow transplant. Then, in May 2016, she received Kite’s CAR-T treatment at Moffitt Cancer Center in Florida. She was declared cancer-free at her six-month checkup and no longer requires any cancer treatment. “I feel like I have my life back—I’m able to do everything I did before,” said Revoir, a married mother of two grown daughters and retired graphics professional for the Greater Orlando Aviation Authority