Our health is important to all of us and having a disease or condition requiring treatment is anxiety provoking, for both health and financial reasons. Current therapies for many diseases are not curative; they merely delay disease progression or ameliorate symptoms. Fortunately, the United States has a robust system of innovation for improving the prevention, diagnosis and treatment of disease. Unfortunately, these innovations tend to be priced at high levels, and thus add to the financial stress experienced by government programs like Medicare and Medicaid, private health plans, employee health benefit plans and individuals.
The current leading edge of innovation is in cell and gene therapies, which have the potential to address currently untreatable conditions or to improve outcomes for many diseases which have inadequate therapies today. Gene therapies are designed to replace malfunctioning genes which cause or contribute to disease. Many of the first gene therapies have addressed very rare conditions, many in children, which have no current treatments. Recently, gene therapies for more common conditions, such as sickle cell anemia or hemophilia, have been approved. There are potential safety issues with these therapies and the durability of effectiveness needs to be studied over a long period of time.
Gene therapies tend to be extremely expensive, priced in the millions of dollars. In some cases, however, such as hemophilia, they may offset current treatment costs which are also quite high. If a gene therapy works, it may actually cure the disease or condition, which is clearly a better outcome for the patient, and might cause a net spending reduction for the health system. But there is currently significant stress among payers about how to handle the cost of these therapies.
Cell therapies currently involve removing cells, usually immune system cells, from a patient, modifying those cells in some manner and replacing them in the patient. The initial cell therapies largely addressed cancer, and modified the immune cells to attack cancer cells. These therapies have shown good efficacy, but can have serious adverse effects. They tend not to be quite as expensive as gene therapies, but are still very costly, with medical and product prices often running from $500,000 to $1 million.
There are therapies which have the characteristics of both cell and gene therapy under development. An extensive pipeline of products, many addressing very common diseases, is in progress. Imagine, for example, being able to prevent or cure diabetes by a gene or cell therapy. Understanding the net benefit to the health system will be important — do they add cost over a patient’s lifetime or reduce it? An interesting introduction to the challenges of paying for gene therapies is found at this link, produced by an organization that is expert in this area. (ICER Paper) What is fairly clear is that for many patients, the health outcomes are a tremendous improvement over current treatments.
Kevin Roche runs The Healthy Skeptic, a website about the health care system, and has many years of experience working in the health care industry. If you have health care-related questions, you can contact Kevin at xuebpur@urnygul-fxrcgvp.pbz and he may answer the question in a column.
Read more from Kevin Roche at his website: healthy-skeptic.com
Kevin Roche
Kevin Roche runs The Healthy Skeptic, a website about the health care system, and has many years of experience working in the health care industry.
