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Breakthrough therapies are saving lives. Can we afford them?

Lisa M. Krieger, Bay Area News Group on

Published in Health & Fitness

Harnessing the body’s own cells to fight disease, long a medical dream, is finally a reality.

Now comes the bill.

Last month, Stanford became the first hospital in the nation to use a new $515,000 cell therapy to treat a patient with advanced melanoma. A related approach, costing $420,000 to $475,000, is offering hope to patients with lethal blood cancers.

Meanwhile, cells fixed by gene therapy can slow, even stop, the progression of intractable diseases like sickle cell or beta thalassemia — for the extraordinary price of $2.1 million to $4.25 million each.

This is the future of medicine, experts agree. But the cost of this new class of medical treatment is raising alarm about availability and affordability, even as its potential grows. It’s time to reimagine our payment models, they said.

“Cell and gene therapies have the possibility to transform thousands of lives but only if we ensure sustainable access to them for all patients,” said Sarah Emond, president of the influential Institute for Clinical and Economic Review, a Boston-based nonprofit that assesses the value of medicines.

 

The prices aren’t yet unmanageable, because so few people are currently treated. Patients must travel to designated treatment centers, and too few are referred by community physicians. But demand should increase as more treatments are introduced that serve a wider population.

Most health insurers in the United States aren’t set up to support one-time personal therapies that deliver long-term benefits, at unprecedented prices.

“These are precision medicines,” said Dr. David Miklos, chief of the Stanford Bone Marrow and Cell Therapy Program, where hundreds of cancer patients have been treated with the CAR-T cell therapies. “It’s different than buying a pill at the CVS pharmacy that can work for anybody.”

It is a triumph decades in the making. The promise of cell and gene therapies has intrigued scientists for decades, but progress was slow, with many setbacks. Now the long research journey has finally paid off, with FDA-approved products entering the clinic.

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