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How to evaluate breakthrough therapies

From our Expert Voices conversation on drug pricing.

The value of breakthrough therapies, such as Kymriah, the first approved Chimeric Antigen Receptor Therapy (CAR-T) should be evaluated with the following filters:

  1. Are these therapies providing incremental and meaningful benefit versus the available 'standard of care' and with a risk profile that is manageable for most of the patient population it will be used in? The evidence overwhelmingly indicates this is the case for Kymriah being used to treat children with leukemia.
  2. What is the current cost of alternative therapies for these patients, if they exist? For children with leukemia, their only other hope of a 'curative' treatment is a bone marrow transplant. The average cost of that and associated care is between $800,000 and $1 million, and reported success rates are lower than the new offering of CAR-T, which is only administered once to the patient in a 'transplant light' environment.
  3. What does it cost to make these therapies and deliver them to a patient? For most manufacturers of these therapies the initial 'cost of goods' will be high, much more than traditional medicines. As manufacturing science of these therapies improves, these costs will go down.

The bottom line: New therapies may cost comparatively less than existing ones — and deliver more.

Other voices in the conversation: