Cancer-killing drug approved, but will you be able to afford it?

Novartis' CAR-T gene therapy, the first approved by FDA, to be priced based on cancer patients' outcomes

The United States Food and Drug Administration (FDA) on Wednesday announced that it has approved a cell-based gene therapy in the United States.

US regulators approved a revolutionary new treatment from Novartis that modifies the body's own immune-system cells and reinjects them to treat, and in many cases cure, patients with deadly blood cancers who have run out of other options. The approval of the immunocellular therapy follows the advice of the FDA's Oncologic Drugs Advisory Committee, which voted 10-0 in July to recommend approval of tisagenlecleucel for pediatric ALL.

But over the past several years, immunotherapy-therapies that enlist and strengthen the power of a patient's immune system to attack tumors- has emerged as what many in the cancer community now call the "fifth pillar" of cancer treatment. Novartis has agreed a risk evaluation and mitigation strategy (REMS) with the FDA that amongst other things will see the therapy delivered only at certified treatment centres. They call the one-time infusion CAR-T cells and they will not charge the patient if he/she does not show signs of improvement within a month.

A spokeswoman for Switzerland-based Novartis declined to say how much the drug will cost, though analysts have estimated a price of $500,000 or more. It's expected to be a 22-day turnaround for Novartis' Kymriah treatment.

Kymriah has a $475,000 price tag; however, patients who do not respond within a month of treatment will not be charged, according to Novartis. Vehicle T-cell therapies can damage healthy immune cells, including the cells that produce the antibodies that fight disease. "Emily's cancer remains in remission, and in larger trials, we're seeing overall remission rates over 80 percent, which is a remarkable improvement upon previous treatment success rates", said lead investigator of the CHOP and global trials of the therapy, Stephan Grupp, MD, PhD, the Yetta Deitch Novotny Professor of Pediatrics at Penn and director of the Cancer Immunotherapy Frontier Program and chief of the section of Cell Therapy and Transplant at CHOP.

The therapy is called Kymriah, and it is being made by Norvartis. This gene treatment boosts T cells - the immune system's soldiers - by reprogramming them to carry a "chimeric antigen receptor" or auto.

Despite Kymriah's success in the clinical trial, Walid Gellad, a doctor and professor at the University of Pittsburgh, suggested to Axios that treatments with undetermined effectiveness-even if they have the potential to save lives-shouldn't cost more than proven measures such as bone marrow or kidney transplants, adding: "This is an wonderful therapy, but there has to be a limit at which point companies can no longer charge desperate patients, or taxpayers, enormous sums". "The real question is going to come when this therapy is poised to be expanded into other patients who are older, and providing the chance for 60 or 70 years of [additional] life is not what you're talking about, and then we're really going to have some tough value questions that we should be prepared to ask". However, ultimately, the cell therapy could go a long way in saving hundreds of young people's lives each year. This allows the cells to replicate quickly and zero in on cancer cells, fighting the disease for years.

For one, the treatments will have to treat more types of cancer than the one approved on Wednesday. "The lessons learned from the clinical trials of auto T-cell operations will assist us in being able to provide to the larger population".