Replies to post #213347 on Biotech Values

[DewDiligence]

Kymriah/CAR-T outcomes-based pricing (from NVS PR):

https://globenewswire.com/news-release/2017/08/30/1104337/0/en/Novartis-receives-first-ever-FDA-approval-for-a-CAR-T-cell-therapy-Kymriah-TM-CTL019-for-children-and-young-adults-with-B-cell-ALL-that-is-refractory-or-has-relapsed-at-least-twice.html

Novartis…announced a novel collaboration with the United States Centers for Medicare and Medicaid Services (CMS) focused on improving efficiencies in current regulatory requirements in order to deliver value-based care and ensure access for this specific patient population.

This approach is intended to include indication-based pricing for medicines and supports payments for a medicine, such as Kymriah for its initial indication, based on the clinical outcomes achieved, which would eliminate inefficiencies from the healthcare system. Other value-based approaches related to future indications for Kymriah and CAR-T cell therapies are under discussion.

Furthermore, Novartis is collaborating with CMS to make an outcomes-based approach available to allow for payment only when pediatric and young adult ALL patients respond to Kymriah by the end of the first month.

Future potential indications would be reviewed for the most relevant outcomes-based approach. [In other words, the pricing is likely to vary based on the indication.]

The actual price (for pediatric ALL) has not yet been disclosed, as far as I know.

[DewDiligence]

FDA approves Roche’s Actemra for CAR-T-induced CRS:

http://www.roche.com/media/store/releases/med-cor-2017-08-30.htm

[DewDiligence]

Kymriah FDA label:

https://t.co/8bhOn1PlzI

[Biowatch]

"I don't consider CAR-T a form of gene therapy"

It absolutely is gene therapy! You genetically modified the T cells to make them behave in a new way, and attack a new target.

If you did not introduce a new gene into the T-cells, there would be no therapeutic benefit.

Consequently, it is gene therapy.

The patient can't pass the genetic change to anybody else, let alone their offspring, and the genetically modified cells won't last forever, even though they can replicate, but that doesn't mean it isn't gene therapy.

From your link:

Kymriah is a genetically-modified autologous T-cell immunotherapy. Each dose of Kymriah is a customized treatment created using an individual patient’s own T-cells, a type of white blood cell known as a lymphocyte. The patient’s T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.

[DewDiligence]

FDA approves NVS’ Kymriah—(CAR-T)—in adult DLBCL—its second indication:

https://globenewswire.com/news-release/2018/05/01/1494437/0/en/Kymriah-tisagenlecleucel-first-in-class-CAR-T-therapy-from-Novartis-receives-second-FDA-approval-to-treat-appropriate-r-r-patients-with-large-B-cell-lymphoma.html