FDA forestalls unpopular payer decisions on PCSK9s

Praluent’s transatlantic regulatory milestones show the differences in the influence of regulators and payers in the respective US and European markets.

The European Medicines Agency’s recommendation to include the broad population of patients unable to reach cholesterol goals on statins shows that national health programmes might be able to negotiate a lower price to allow wide usage, while the US FDA’s decision to limit access to a smaller group shows that Sanofi and Regeneron will need to demonstrate a survival benefit before expanding utilisation. Competitor Amgen, which won the race to the EU market, now knows what to expect when the US makes a call on Repatha next month.

Olive branch

Praluent on Friday became the first PCSK9 inhibitor to achieve approval in the world’s biggest drug market only hours after it became the second to earn the backing of European regulators. The big difference is in the label: the FDA supported Praluent’s use in patients with familial hypercholesterolaemia and with high-risk atherosclerotic cardiovascular disease, while the EMA’s recommendation additionally included patients not achieving goals with statins or those who cannot take statins.

The European recommendation was similar to that of Repatha, which earned the agency’s backing in May and full approval earlier last week.

With US approval in the bag, Sanofi and Regeneron gave the sector a glimpse of the price for these complex biological drugs: $40 a day, or $14,600 a year. This came in well above the conservative $4,250 after-discount price expected by Leerink analyst Seamus Fernandez.

After last year's launch of Gilead’s hepatitis C drugs Sovaldi and Harvoni, with their $1,000-plus per day costs, payers have been ringing alarm bells about pricing. The FDA’s label, which is not likely to change at least until cardiovascular outcomes data emerge in 2017, ought to ease some insurer worries as eligible patients number just 8-10 million.

“I do think payers will be more comfortable with a label that really focuses on the very high-risk patients,” Robert Terifay, Regeneron’s commercial senior vice-president, said on a conference call.

In an act of payer diplomacy Mr Terifay said the Regeneron promotional campaign would focus on making sure that patients are on the maximum tolerated dose of statins before seeking further intervention, something that has not been a physician focus since generic statins became available and the branded companies stopped advertising.

Mr Nice Guy

Payer relations will be an essential part of this story, especially as the Regeneron-Sanofi portrait of the US launch market is fairly similar to a worst-case scenario drawn up by executives at the pharmacy-benefit manager CVS/Caremark ($200bn a year for new cholesterol drugs? Try again, payers, February 20, 2015).

How well Regeneron and Sanofi have done in this department will become more clear if, as expected, Repatha gets the all-clear by August 27. Formulary exclusions could come into play at that point, as happened with the hep C drugs. With promotion starting today and product shipping on Wednesday, Praluent will have an important but not unassailable advantage.

With an explicit FDA label restricting use to sicker patients, US payers might not have to use some of the utilisation management techniques that often make them unpopular; patients and physicians see prior authorisation and step therapy as burdensome, and often it is the insurance industry, rather than pharma, that feels the criticism.

In Europe, however, the wider population eligible for PCSK9 use is likely to trigger stronger utilisation management. This is counterbalanced by the lower price that Sanofi has acknowledged it will need to agree to access national formularies.

How these moving parts affect utilisation is hard to gauge, but it is likely that Europe will give a preview of what the US would look like should the massive outcomes studies show a survival benefit and the US labels be expanded. Should this come to pass it would not be a surprise to see price cuts to satisfy payers and take market share.

Project Study Trial ID
Praluent Odyssey Outcomes NCT01663402
Repatha Fourier NCT01764633

To contact the writer of this story email Jonathan Gardner in London at jonathang@epvantage.com or follow @ByJonGardner on Twitter

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