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At Sutter Children’s Center in Sacramento this week, the 7-year-old was given an injection of Spinraza, the first drug approved in the U.S. to treat spinal muscular atrophy, or SMA. ... With Spinraza, each thumb-sized 5-milliliter vial – about a teaspoon’s worth – costs $125,000. The recommended dosage is four “loading” injections within about two months, followed by maintenance shots every four months for life. For families such as the Goepperts, that price tag is staggering: roughly half a million dollars just for the initial series of Spinraza shots and $375,000 a year thereafter. (Buck, 2/20)

A new patient advocacy group launches Wednesday that distinguishes itself by focusing only on drug prices and eschewing money from the pharmaceutical industry at a time when drug makers are pouring millions into a campaign fighting efforts to regulate them. The formation of Patients for Affordable Drugs (PFAD) comes as local officials and members of Congress are decrying the latest huge drug price hikes, this time by drugmaker Kaléo for its version of the overdose antidote naloxone. (O'Donnell and Shesgreen, 2/22)

The organization Patients for Affordable Drugs launched a campaign to share the stories of Americans who struggle to cover the cost of their prescriptions and who are hoping to find a solution to lower drug prices. (2/22)

In a noteworthy deal, Gilead Sciences agreed to pay $125 million to Sarepta Therapeutics for a priority review voucher. But one Wall Street analyst expressed disappointment over the price tag and suggested the deal raises questions about how much these controversial vouchers can fetch going forward. (Silverman, 2/21)

When Marathon Pharmaceuticals’ $89,000 price for a year’s supply of its newly approved muscular dystrophy drug sparked outrage this month, the company’s CEO responded with another figure: $20. That’s how much he expects Duchenne muscular dystrophy patients to pay per prescription of the drug, he wrote in a letter posted on the company’s website. Insurers would cover the medication, and patients would pay only their typical co-pays. He touted the company’s patient assistance program to help patients defray out-of-pocket costs. (Schencker, 2/21)

Recent debates about drug pricing have tended to focus on manufacturers and patients, but Doug Collins is among the few legislators looking at the middlemen. Collins, a Republican congressman from Georgia, railed against the country’s three major pharmacy benefit managers in an interview with STAT, expressing his desire for a drug-pricing debate that centers on what he views as the true power centers instead of  “Pharma bro” and the push to allow drug-price negotiation under Medicare Part D. (Facher, 2/20)

Leveraging its buying power as one of the state’s largest health insurers, Harvard Pilgrim Health Care has struck two more deals to pay for expensive drugs based on how effectively they treat patients, an emerging strategy aimed at reining in medical spending. Harvard Pilgrim, which has 1.3 million members, said the agreements cover the rheumatoid arthritis medicine Enbrel, made by Amgen Inc., and Eli Lilly & Co.’s osteoporosis medicine Forteo. (McCluskey and Woodward, 2/22)

As big-game hunting goes, Sanofi chief executive Olivier Brandicourt is 0-for-2. Two months ago, Sanofi appeared to be on the verge of acquiring Actelion Pharmaceuticals, which would have been a notable accomplishment for a couple of reasons: Johnson & Johnson had just backed out of negotiations to acquire Actelion and Sanofi had recently lost out on bidding for Medivation, a stinging defeat in a growth strategy that relies on big deals. (Silverman, 2/16)

The CEO of the Pharmaceutical Research and Manufacturers of America on Tuesday came out strongly in favor of a rigorous Food and Drug Administration. His remarks come as the Trump administration weighs potential candidates to lead the agency who have suggested they would radically change how it vets new drugs. Asked by STAT about the notion that the FDA should no longer evaluate drugs for effectiveness, the industry trade group’s Stephen Ubl was unambiguous: “We believe that the FDA review process is the gold standard and should remain so.” (Robbins, 2/21)

Some state lawmakers are proposing to give the Insurance Department more teeth to tackle the problem of skyrocketing prescription drug prices. House Bill 161 would require pharmaceutical companies to disclose the cost of drugs — including materials, research, clinical trials, marketing and advertising.The bill is co-sponsored by Lancaster city Democrat Mike Sturla and Jim Cox, a Republican who represents part of northern Lancaster County. (Stauffer, 2/21)