資料來源： https://www.bloomberg.com/news/articles/2016-08-16/amgen-sanofi-cholesterol-drugs-not-cost-effective-study-says

Amgen Inc., Sanofi and Regeneron Pharmaceuticals Inc. would need to cut prices on their new cholesterol-lowering drugs by more than two-thirds to make them cost-effective to use in the U.S., a study found.

The high list prices of the injectable medicines Praluent and Repatha -- which on average cost more than $14,000 per patient each year in the U.S. -- is "uniquely challenging" because they are meant to be lifelong treatments for a growing number of people, according to the study, published Tuesday in the Journal of the American Medical Association.

Use of the newer drugs instead of older medicine ezetimibe would reduce cardiovascular care expenses by $29 billion over five years as fewer people suffered heart attacks and strokes requiring hospital treatment, the researchers estimated. Giving Praluent and Repatha to all eligible patients would also contribute to a $120 billion increase in annual U.S. health-care spending, a gain of about 4 percent, they said.

"This hopefully should prompt some discussion about what are reasonable or tolerable drug prices in the U.S.," Kirsten Bibbins-Domingo, an author of the report and a professor at the University of California, San Francisco, said in a phone interview. "We modeled many, many, many scenarios, and this drug is not cost-effective at the ticket price."

Statin Alternatives

Praluent, developed by Sanofi and Regeneron, and Repatha, an Amgen treatment, were approved by the U.S. Food and Drug Administration last year. The drugs, used to inhibit the protein known as PCSK9, are aimed at patients who have genetic conditions or can’t tolerate cheaper cholesterol treatments such as the widely used statins.

Insurers and pharmacy-benefit managers have warned PCSK9 inhibitors could become one of the costliest drug classes ever because of their prices and the large number of people in the U.S. with high levels of bad cholesterol. Those costs have led to tight restrictions for their use in the U.S., the largest market for prescription drugs.

"The potential increase in health-care expenditures at current or even moderately discounted prices could be staggering," despite savings from averted cardiovascular disease, the study found.

Exaggerated Use

Regeneron and Sanofi said the analysis "exaggerates real-world use of PCSK9 inhibitors," leading to an inflated estimate of the cost burden.

Pharmacy benefit managers and insurers frequently negotiate for large, secret rebates off the list prices for top-selling drugs, thereby reducing the costs for their clients. The study looks at list or wholesale prices, which are significantly higher than what is actually paid, the companies said in an e-mailed statement.

Three-quarters of patients prescribed Praluent have been denied access to the drug by their health plans, decisions fueled by misleading cost studies, they said.

In a separate statement, Amgen said it "strongly believes in the clinical and economic value of Repatha" and that the analysis presents "just one view" on the medicine. Other research has shown a "value-based price" for Repatha of as much as $15,000, more than the current list price and the net price purchasers pay in the U.S., Amgen said.

Potentially Persuasive

Amgen expects to report results from a study of Repatha’s effects on cardiovascular events such as heart attacks and strokes in the first quarter of 2017, the company said last month. If positive, it may persuade insurers to ease restrictions. Regeneron has said that interim outcomes data for Praluent could be released as early as this year.

Amgen reported Repatha sales for the first time in April, and they missed expectations by a large margin. Analysts estimate that Repatha’s sales will climb from about $154 million this year to more than $2.45 billion by 2020. Praluent, meanwhile, is projected to generate $179 million in 2016, and grow to almost $2 billion by the end of the decade.

Cholesterol drugs aren’t alone in attracting scrutiny. The pharmaceutical industry in the U.S. has faced criticism for high prices, with new hepatitis C treatments causing an uproar, even as they’ve been heralded as medical breakthroughs because they can cure the disease in 12 weeks.

"To me, this is the next extension of that conversation because this is an even larger population than the hep C population, and they are drugs that are meant to be used for a lifetime,’’ Bibbins-Domingo said.