The first gene therapy in the US has been approved by the FDA. This therapy called Kymriah has been created by Novartis and modifies the cells of the patient who undergoes that treatment. The cells are genetically altered in order to treat deadly leukemia or to make cancer go in remission for some patients.
While the treatment is very expensive, going up to $475,000 and has received a lot of criticism for it, the therapy might be a one-time injection to cure a certain disease.
The Kymriah therapy might or might not be covered by insurance companies, and this issue will sure rise in the future as more gene-based therapies are going to be developed. There are 504 gene therapies that would treat cancer, genetic disorders, infectious diseases and they’re in clinical trials. Out of these 34 therapies are in the testing stage.
Gene Therapies Treat Several Diseases
There have been studies on different treatments that tackle diseases such as Sickle cell disease, hemophilia, Immunodeficiency disorders and some cancers and how much they cost over a year for one patient. The executive director of the Alliance for Regenerative Medicine, Michael Werner has said that the gene therapy should be an effective treatment that would improve people’s quality of life, keep them out of hospitals and away from other future cost for medicine.
If it’s effective, this would mean that even though gene therapy costs a lot, on the long run it will prove cost-effective as the patient won’t need any other treatments in the future.
Ed Schoonveld, an expert in drug pricing and market access mentioned that they don’t know from the beginning the full picture of its effect – if it’s long-term or not and if gene therapy would be life-changing or even save lives, adding a price tag is a lot more difficult.