The recent FDA approval of CAR T-cell therapy is a major milestone in the longstanding effort to cure childhood cancer, once almost uniformly fatal.
The problem: Today childhood leukemia is treatable in most cases and 80% of children survive longer than five years. Unfortunately, early exposure to radiation and chemotherapy can have devastating long-term health impacts for childhood cancer survivors. Up to a third of long-term survivors will suffer from at least one life-threatening or debilitating medical problem caused by their "curative" treatment, which can include secondary cancers, severe cardiovascular disease, infertility, and endocrine disorders. Thus, many are trading one difficult disease for a lifetime battling numerous health problems.
Why it matters: Because they selectively target tumor cells and spare normal cells, new therapies like CAR-T presumably will result in less damage to normal tissues and cause fewer long-term health problems. They offer incredible hope to a new generation of childhood cancer patients that they can not only be cured, but can look forward to a productive life without additional health burdens.
The bottom line: While the upfront cost of these new therapies are high, any consideration of value should take into account the health care savings generated by an overall healthier and productive population of childhood cancer survivors.
Other voices in the conversation:
- Greg Aune, pediatric oncologist, Greehey Children's Cancer Research Institute: Value isn't just about surviving cancer
- David Mitchell, president and founder, Patients for Affordable Drugs: Drugs don't work if people can't afford them
- Usman Azam, president & CEO, Tmunity Therapeutics: How to evaluate breakthrough therapies
- Austin Frakt, health economist, Department of Veterans Affairs, Boston University and Harvard University: The public should have a say in what a drug is worth
- Paul Howard, senior fellow, Manhattan Institute: Price should be based on outcome