Praluent's FDA review looms, but questions persist

Praluent and Repatha could cost between $7,000 and $12,000 per year of treatment.
Praluent and Repatha could cost between $7,000 and $12,000 per year of treatment.

The FDA later this week is expected to announce whether it has approved Sanofi's and Regeneron's Praluent (alirocumab)—one of two highly anticipated drugs that dramatically reduce cholesterol levels.

PCSK9 inhibitors are antibodies that deactivate a specific protein in the liver, which subsequently reduces levels of cholesterol in the bloodstream. Amgen, too, has a PCSK9 inhibitor in development, called Repatha (evolocumab). An FDA advisory panel ruled 13-3 in June in favor of recommending Praluent for approval and 11-4 in favor of Amgen's Repatha.

If the drug is approved, it will likely be the easy part of the drug's launch for Sanofi and Regeneron, which already are dealing with persistent concerns about the investigational medicine's potential lofty price tag and questions about who should receive the drug.

The FDA's stance for both Amgen's and Sanofi/Regeneron's PCSK9 inhibitors is that the drugs' ability to reduce cholesterol alone is sufficient for approval. However, both drugmakers are currently conducting outcomes trials for these drugs with which they might tout and promote additional claims to healthcare providers.

Intouch Solutions and MicroMass are two of the agencies tapped to develop the campaign for the PCSK9 inhibitor, according to MM&M's 2015 Agency Issue. Entree Health executives noted that the agency is working on the payer-access strategy for Amgen's Repatha.

Analysts have estimated that both Praluent and Repatha, which has a PDUFA date slated for August 27, will carry an estimated price tag between $7,000 and $12,000 per year of treatment. Payers say the population for these drugs could range between 200,000 to 2.3 million people depending on the drug's approved label.

Payers have said that this next generation of cardiovascular treatments could force insurers to incur substantial new costs. Pharmacy benefit manager Prime Therapeutics estimated that subsidizing the use of these drugs could cost as much as $23.3 billion annually.

The PBM says that if the drugs are made available to all statin-intolerant people regardless of their cardiovascular risk—which would be the drug's broadest approved label—this could add $6.71 to “commercially insured coverage costs” per member per month.

“If not managed appropriately, the costs of PCSK9s could have a dramatic impact on overall drug spend starting later this summer,” said Pete Clagett, SVP of integrated care and specialty at Prime in a release. “In a couple years, PCSK9s could cost America the equivalent of $73 per person.”

PhRMA, the drug industry's lobbying group, wrote in a recent blog post that despite the tens of millions of people in the US who struggle with uncontrolled cholesterol, “many of these people could potentially reach their cholesterol goal by taking existing medicines more consistently, combining treatments or changing doses and implementing lifestyle changes,” in lieu of taking Praluent or Repatha.

The Institute for Clinical and Economic Review announced Tuesday that it had received new funding to research cost-effectiveness of new drugs and would launch a new drug assessment program with its first target being the new cholesterol-lowering PCSK9 inhibitors.

The independent nonprofit plans to release public reports analyzing a drug's cost- and comparative effectiveness while also gauging its potential budget impact. In a drug approval landscape often characterized by “me-too approvals,” which are drugs that provide a minimal benefit over the standard of care, reports of this kind could put significant pressure on drugmakers to demonstrate a drug's effectiveness beyond laboratory biomarkers, like LDL-levels, and into real-world targets, like the incidence of heart attack.