Emily Whitehead was a little girl on the brink of death. Diagnosed with acute lymphoblastic leukemia (ALL) at the age of five, Emily’s medical team tried traditional treatments, but her cancer still returned. Her parents were told that hospice care was her only option, but they refused to accept that. They called the Children’s Hospital of Philadelphia and received news that they could enroll Emily in a clinical trial for an experimental cellular therapy, using a new technology which had never been tried before in children.
The process involved taking T cells from Emily’s own body, reprogramming them in a lab to recognize and fight her cancer cells, and then reinfusing them back into her body. At first, Emily became very sick and her medical team induced a coma for 14 days. But the treatment worked, and just 23 days after receiving her reprogrammed T cells, there was no evidence of cancer in her bone marrow biopsy. Her parents took her home in June and that August, she went back to elementary school.
This week, the experimental therapy that saved Emily’s life was approved by the US Food and Drug Administration for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse. Novartis, in collaboration with the University of Pennsylvania, developed this novel therapy. Its approval marks a historic moment in the fight against cancer, validating an entirely new approach to cancer care that is wholly personalized.
Immunotherapies like this one are opening new ways to treat cancer, enabling us to target disease like never before and in cancers where progress has been elusive for years. Our hope is that this new technology will become a catalyst to find other therapies for patients in need. I liken this moment to the discovery of targeted cancer therapies, which helped establish a new paradigm for drug development and opened the door for many other advances in the treatment of cancer.
However, I would be remiss if I did not address the question of cost. We’re still in the early days for this new technology and its manufacturing process. The treatment is manufactured for each individual patient through a delicate, labor-intensive process that requires taking a patient’s blood, shipping it to a special facility where the T cells are expanded and genetically modified, and then shipping it back to the patient for infusion. Time is of the essence in this process and a significant amount of human oversight is required.
Over the last several years, we at Novartis have invested substantial amounts of money into this technology, running clinical trials with partners and honing our manufacturing process. We acutely understand that the patients who need this therapy are often out of options and are about to die. They are children, young people who should just be kids, with school, sports, music, and friends to occupy their time. Kids who should not be fighting cancer.
When setting the price for this technology we considered many factors including its clinical benefits, the outcomes it provides to patients, and the value it offers to health systems. We sought input from several external health economic experts. Additionally, we considered health technology appraisals, including an assessment led by the University of York and produced by NICE of the United Kingdom, which determined a cost-effective price would be $600,000 to $750,000. We also looked at the current standards of care, such as the cost of allogenic stem cell transplants, which is between $540,000 and $800,000 for the first year in the US. Recognizing our responsibility to bring this innovative treatment to patients, we have set the price below that level at $475,000 for this one-time treatment.
To create more value for health systems, 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 ALL patients respond to this therapy by the end of the first month. I am proud that Novartis is working with CMS on this novel collaboration, and we look forward to continuing to work with CMS to potentially expand this approach to other products and disease states. Ultimately, value-based pricing approaches like this one could help the US adopt new technologies in the fight against cancer in a way that lowers total costs for the US healthcare system.
To ensure that patients receive the treatment in a safe and timely manner, Novartis is also providing traditional support to patients by helping them navigate insurance coverage and by providing financial assistance for those who are uninsured or underinsured.
Three years ago, Forbes reported on our efforts in this area, and on Emily’s story. Today, she is still in remission, is at the top of her class academically, and is enjoying the life of a typical girl.
It is our hope that we can continue to find new therapies like this one that can change the practice of medicine and help more patients like her.
