A new milestone in the fight against pediatric cancer

This week, Novartis received US FDA approval for our chimeric antigen receptor T cell (CAR-T) therapy that was developed with the University of Pennsylvania, for the treatment of  patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. This approval marks a historic moment in the fight against cancer. The process involves taking T cells from the patient’s own blood, reprogramming them in the lab to recognize and fight the cancer cells, and then reinfusing them back into the body.

This is also a historic moment for how our industry prices medicines. Novartis has announced a novel collaboration with the US Centers for Medicare and Medicaid Services (CMS), which is intended to include indication-based pricing for medicines and support payments for a medicine based on the clinical outcomes achieved.

Additionally, for this first indication, we will offer a unique outcomes-based approach that would allow for payment only when pediatric and young adult ALL patients respond to this therapy by the end of the first month. I am proud that Novartis is working with CMS on this first-of-its-kind collaboration, and we look forward to continuing to work with CMS to potentially expand this approach to other products and disease states.

These are important milestones for patients and their families, as well as for our team and partners who worked tirelessly to get here. This therapy treats very sick children who are often out of options and are about to die. They’re children who shouldn’t be fighting cancer, but should be out enjoying a normal childhood.

Take Emily Whitehead, for example. Diagnosed at the age of five, Emily was on the brink of death after her ALL relapsed and she wasn’t responding to existing treatments. Her parents were told by the local hospital that Emily’s only option was hospice care, but they refused to accept this. They called the Children’s Hospital of Philadelphia and learned that she could be the first child enrolled in the clinical trial for this therapy. Although she did suffer some serious side effects and a high fever, her father Tom got the call that there was no evidence of cancer in her bone marrow biopsy just 23 days after Emily received her reprogrammed T cells. He says it was the best call he ever received. Today, Emily is still in remission. She is living the life of a typical girl, and is at the top of her class academically.  

Stories like Emily’s inspire us at Novartis, pushing us to do everything we can to help patients. I’m often asked what I like most about leading a healthcare company – it is moments like this, when we can give people suffering with illness the potential to think about doing things they’ve only imagined before. It is my hope that this is just the beginning of a new era of cancer care, and that this new technology will become a platform to find other therapies for patients in need.