Using the body's own cells to fight disease – long a medical dream – is now finally a reality.
Now comes the bill.
Last month Stanford the primary hospital within the country to make use of a brand new cell therapy value $515,000 to treat a patient with advanced melanoma. A related approachThe project will cost $420,000 to $475,000 and can provide hope for patients with deadly blood cancer.
Meanwhile, cells fixed through gene therapy can slow the progression of intractable diseases akin to: B. decelerate and even stop Sickle cell anemia or beta thalassemia – on the exceptional price of $2.1 million to $4.25 million each.
Cell therapy technologist Thomas Orozco defrosts the treated immune cells of a patient with advanced melanoma. The cells are taken from the patient's tumor and enriched in a laboratory to higher fight the cancer.[/caption]
This is the longer term of medication, experts agree. But the associated fee of this recent class of medical treatment raises concerns about availability and affordability, whilst its potential grows.
“Cell and gene therapies can change the lives of thousands of people, but only if we ensure sustainable access to them for all patients,” said Sarah Emond, president of the influential organization Institute for Clinical and Economic Reviewa Boston-based nonprofit organization assesses the worth of medicines.
The prices aren’t yet prohibitive because so few persons are currently being treated. Patients must travel to specific treatment centers and aren’t referred by general practitioners. But referrals and demand should increase as more treatments are introduced to serve a broader population.
Most health insurers within the United States aren’t designed to support one-time personal therapies that provide long-term advantages at unprecedented prices.
“These are precision medicines,” he said David Miklos, director of the Stanford Bone Marrow and Cell Therapy Program, where lots of of cancer patients have been treated with CAR T-cell therapies. “It’s different than buying a pill at the CVS pharmacy that can work for everyone.”
The promise of cell and gene therapies has fascinated scientists for many years, but progress has been slow and fraught with setbacks. Now the long research journey has finally paid off as FDA-approved products are entering the clinic.
“The technology to bring it to life has finally caught up with the ideas behind it,” said the Stanford assistant professor of medication Dr. Allison Betaf Warnerwho conducts Stanford's melanoma study.
Cell-based strategies are delivering essentially the most famous cancer treatments in many years.
In an approach called chimeric antigen receptor (CAR) T-cell therapy, immune cells are collected from patients and genetically modified to higher fight lymphoma, leukemia and, more recently, multiple myeloma.
Another uses a special approach and uses tumor-infiltrating lymphocytes (TILs). These immune cells are taken from the tumor, amplified within the laboratory after which given back to the patient. In clinical studies, about 30% of patients experienced shrinkage or disappearance of the incurable melanoma tumors.
“I am very happy that it is here now. … I walked the tightrope and didn't fall off,” said a Stanford patient who asked not to be named.
Gene therapy also uses manipulated cells in which genes are replaced, deleted or inserted. Kendric Cromer, a 12-year-old boy from a suburb of Washington, DC, was born on Wednesday the world's first person with sickle cell anemia to begin FDA-approved gene therapy. Both Stanford and UCSF will offer this treatment.
Scientists are now working to expand the therapeutic repertoire to combat solid tumors, autoimmune diseases, aging, HIV and more.
“It’s just the beginning of a new era,” said the biochemist Wendell LimDirector of the UC San Francisco Cell Design Institute.
“It shows that we can take a living cell and change how it works so that it makes new decisions and performs complex actions. It processes information like a small computer,” he said. “That’s very different from a static thing, like a chemical.”
It's still early and few patients are taking advantage of these new breakthrough therapies, doctors said.
Why? It is not yet known that sick people are not referred by their community doctors, said Miklos. The treatment is risky. Or patients may live far from the country's estimated 30 “centers of excellence” such as Stanford and UCSF and are put off by travel and accommodation costs.
Payment is not guaranteed; it will be decided on a case by case basis. Medicare and MediCal cover the cost of care if it is deemed medically necessary. Emperor too.
The vast majority of private insurers cover treatments, although back-and-forth negotiations are sometimes required, said Gary Goldstein, who oversees operations for Stanford's Blood & Marrow Transplant Program.
Sticker price covers parts only – no labor, no warranty. Drug prices are not regulated like utilities are, and there is no cap on what a company can charge.
Estimates place the total cost of gene therapies over the next decade at a staggering $35 billion to $40 billion. The cost of future cell therapies that could help a wider range of patients will likely be higher.
Drugmakers argue that the prices reflect the major clinical benefits as well as the risks and uncertainties of development. They add that a one-time treatment for a chronic illness can actually save money by eliminating lifelong care.
“We have never cured patients with a single treatment,” he said Dr. Greg Allen from UCSF, which is developing immune cell therapies for notoriously difficult-to-treat tumors such as those of the pancreas and lung. “So it’s very exciting.”
For some patients, it could be their last best chance.
“I don’t believe that life has a price,” said Betof Warner of Stanford. “These are patients who have no other choice.”
If millions of people are helped, as hoped, it will put budget pressure on the federal government and larger payers, while smaller employers, state Medicaid plans and regional health insurers may find providing access financially impossible, economists warn. Health care costs are already outpacing inflation, rising 7% this year.
“It’s going to put a lot of strain on the system,” he said Edwin Park at Georgetown University's McCourt School of Public Policy. “But the issue is critical because you don’t want the high price of these therapies to result in low-income people not having access to them.”
Governments and commercial insurers are already testing the effectiveness of treatments. Some impose strict restrictions on eligibility. They demand discounts and rebates from manufacturers.
If manufacturing were done locally — making the cells at Stanford or UCSF, for example, rather than at distant pharmaceutical companies — that would reduce costs, Miklos said.
As competition increases, prices will fall, he predicted.
Meanwhile, the health system needs to focus on finding innovative payment solutions, Emond said.
One suggestion is to amortize the amounts paid by insurers over time, similar to a home mortgage. Another option is for pharmaceutical companies to provide a pro-rata reimbursement if a patient's condition does not improve – a “pay-for-performance” model. Another option can be a subscription-based approach like Netflix, where insurers pay a monthly fee to get access to the quantity of therapy needed.
“As we look to the future,” Emond said, “this is a moment when we can discuss how we can do things differently.”
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