to scrutinize attempts to prescribe PCSK9 inhibitors. But much of that scrutiny, in the near term, would be a moot point if the panelists’ attitude this week—that a wide swath of people with high cholesterol shouldn’t be eligible for PCSK9 use—becomes part of the FDA approval decision.
However, there is room for debate about what might happen if indeed FDA approves the two drugs for people with heterozygous FH, the form inherited from only one parent. “If they end up approving for that indication, it’ll mean doctors will have a fairly wide ability to prescribe the drugs,” said Ethan Weiss, a cardiologist at the University of California, San Francisco, who followed the proceedings this week.
Weiss, who does lab research and sees patients, shared reservations with some of the advisory panelists. He said that until the PCSK9 inhibitors are tested for “outcomes”—that is, to show a direct link to a reduction in heart disease, not just to a reduction in cholesterol levels—he would only prescribe them for the highest-risk patients, those with inherited FH mutations from both parents.
Outcomes data—different than the Phase 3 data used to make the case for FDA approval—are due from Sanofi and Amgen in late 2017 and early 2018, with more than 40,000 people enrolled. Pfizer’s PCSK9 inhibitor is a bit farther behind in Phase 3 tests but should report outcomes data when the other companies do.
“The committee leaves FDA with considerable discretion in how to define a label, and considerable uncertainty about the ability to rely on LDL as an endpoint going forward,” as noted by Prevision Policy, a regulatory consultancy in Washington D.C., in a research note after Tuesday’s vote.
Wilemon acknowledged that FH “exists on a spectrum,” which makes it harder for clinicians and insurers to pinpoint the disease and pay for its treatment. There might be times when FH patients, especially if diagnosed early, might do well simply with high-dose statins.
“PCSK9s aren’t the treatment of choice for every FH patient,” Wilemon said, but she is confident that insurers “understand the need for additional treatment options.”
For now, she can fly back to California knowing that FH patients and advocates helped push the panelists toward their decision. “You really made a difference by coming today,” one panelist said near the end of the meeting, according to Herper’s account.
Image courtesy of the National Library of Medicine.
