Only 35% of patient requests for Amgen Inc.'s Repatha and Regeneron Pharmaceuticals Inc. and Sanofi's Praluent were approved by commercially insured and Medicare health plans in 2016, according to a new Amgen-funded study.
That figure is lower than a previous estimate by the American Heart Association that 53% of patients were rejected and 47% accepted.
Another Amgen-funded analysis found that commercial payer's current criteria fail to prioritize the highest-risk patients for PCSK9 therapies, a newer class of cholesterol drugs that includes Repatha and Praluent, which both sell for more than $14,000 without discounts.
Both the Amgen studies focusing on access to PCSK9 inhibitors will be shared at the American College of Cardiology, or ACC, annual meeting in Orlando, Fla., a year after the company presented Repatha data at the same meeting that left some analysts and payers disappointed with its effect on mortality risk and other markers such as strokes and heart attacks.
Since then, other reports, including a cost-effectiveness analysis from price watchdog the Institute for Clinical and Economic Review, have recommended significant price slashes for both PCSK9 medicines.
Regeneron and Sanofi will present much-anticipated long-term data from its trial of 18,000 patients for Praluent on March 10.
In Amgen's first study, the rate of cardiovascular events — including heart attacks, strokes and hospitalization for blocked arteries — was higher among the 65% of denied patients, leading the company to conclude that an estimated 110,000 cardiovascular events may have occurred among rejected patients.
The difference between the two groups was less than 1 percentage point per patient years, a measure of risk that incorporates both the number of patients and the number of years covered by the analysis.
"Based on the rejection and event rates observed in the 2016 data, we estimate that among appropriate patients prescribed PCSK9 inhibitors, over 110,000 acute cardiovascular events could have occurred in patients rejected access to a PCSK9 inhibitor," Seth Baum, president of the American Society for Preventive Cardiology and lead study investigator, said in a statement.
For the second study, researchers evaluated a year of data for 5,276 commercially insured patients with atherosclerotic cardiovascular disease, a condition caused by the buildup of cholesterol plaque on artery walls. They concluded that the stringent criteria from payers could delay or deny treatment and may not be identifying the ideal patients for this therapy.
"We are in active discussions with payers to align on clinically grounded, utilization management policies with the goal of best serving the needs of appropriate patients and ensuring access to Repatha," Sean Harper, executive vice president of research and development at Amgen, said in a statement.
Both Repatha and Praluent are included on national formularies, or covered medicines lists, for main pharmacy-benefit managers such as Express Scripts Holding Co. and CVS Health Corp.'s Caremark unit. However, many payers limit coverage only to patients with the most severely high cholesterol, such as those with hereditary orders, arguing that much less-expensive medicines such as statins are effective enough for most patients.
In August 2017, Amgen released its own analysis that concluded a 33% price cut would be cost-effective for many patients. That is roughly the discount the company already gives to pharmacy-benefit managers, it said at the time.
Regeneron CEO Len Schleifer has also spoken out about payer barriers to PCSK9 therapies, saying in the company's latest earnings call that "payers are making it very tough for doctors to fill out the forms."