How Vertex's fight with insurers is costing families

The out-of-pocket cost of treating cystic fibrosis is set to increase dramatically for many patients in the U.S. — one family faced a more than 200% increase — due to an escalating conflict between Vertex Pharmaceuticals and insurance companies, leaving patients caught in the middle of the country's increasingly complex health care system.

As STAT's Ed Silverman reports, the issue begins with high drug prices. Vertex's drugs have a list price of about $300,000 a year. To make them more affordable, the company offers copay assistance, covering much of the cost not picked up by a patient's insurance company. But insurers argue those assistance programs are little more than marketing tools used to direct consumers to higher-priced drugs and increase costs to the entire health care system, so they've instituted policies that shift more of the financial burden to patients. Vertex called those policies "predatory," and it has slashed its copay assistance in response.

"It's a battle about who makes the most money," said Meghan McGarry, a pediatric pulmonologist at the University of California, San Francisco. "And patients are being put at risk."

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Cell Therapy

A new medicine offers hope for a bleak diagnosis

An experimental treatment improved the outcomes of stem cell treatment for patients with advanced leukemia, a result that could usher in a change to how transplant medicine is practiced.

As STAT's Adam Feuerstein reports, the therapy, developed by Actinium Pharmaceuticals, led to a 22% durable response rate among patients with acute myeloid leukemia who received it to prepare their bone marrow for transplant. None of the patients on standard treatment had a durable remission.

Beyond its potential for patients with relapsed leukemia, Actinium's treatment could eventually provide an alternative to the intensive chemotherapy and external radiation commonly used to prepare, or "condition," patients for transplant, an arduous process used for cutting-edge cell, gene, and CRISPR therapies.

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Regulatory

The FDA had a busy Friday

With a long weekend looming, FDA officials made a pair of late-Friday decisions on two closely watched drug applications, approving two pathbreaking medicines that had sparked months of debate.

Apellis Pharmaceuticals won approval for Syfovre, the first treatment for geographic atrophy, a progressive eye disease and a leading cause of blindness in older people. The drug, injected into the eye, appeared to have an uncertain future when the FDA delayed its approval in November to consider new data.

Travere Therapeutics, a firm founded by Martin Shkreli, won FDA approval for the first treatment for a rare and deadly kidney disease that does not suppress the immune system. Travere's medicine, a pill called Filspari, will cary a black-box label warning of its potential liver side effects, which could limit its future use.

Financials

Moderna's chance to explain

Moderna's stock price fell as much as 7% last week after its seasonal flu vaccine — a potential star of the company's post-Covid-19 future — posted mixed results in a first look at Phase 3 data. Now the company, facing consistent questions about its plans for the future, will get a shot to defend its corporate vision.

On Thursday, Moderna will present its full-year earnings and field questions from analysts puzzling over how it plans to counteract the declining demand for its Covid-19 vaccine. The market for those vaccines will soon switch from government contracting to commercial sales, which will likely mean higher prices but lower volumes. Moderna's vaccine for RSV is expected to hit the market some time next year, but competition from Pfizer, GSK, and Johnson & Johnson will present a commercial challenge.

Then there's the flu. In last week's interim data, Moderna's vaccine was better at producing antibodies to influenza A than its active comparator, but it couldn't do the same in influenza B. More concerning were the side effects: 70% of volunteers who received Moderna's vaccine reported adverse reactions compared to 48% of those who got a competitor's vaccine.

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