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A New Line of Specialty Drugs: PCSK9 Inhibitors (Part 2)

This fall, a new line of specialty drugs will hit the market- PCSK9 inhibitors.  PCSK9 inhibitors are a new class of drugs used to lower LDL (bad) cholesterol levels through stifling a protein (PCSK9). The drug is injected once or twice a month and is targeted at a large population of Americans who have been unable to lower high cholesterol levels with statins or other medications.

The major group likely to benefit are people with Familial Hypercholesterolemia (FH)– an inherited disorder effecting around one in 500 Americans.  FH is genetic and causes LDL cholesterol levels to be very high leading to heart attacks and other cardiac events early in life. In clinical trials, PCSK9 inhibitors Repatha (by Amgen) and Praluent (by Sanofi/Regeneron) have been found to bring cholesterol levels to surprising low levels.

The price for these new cholesterol lowering medications is currently set at over $14,000 a year for life.

For individuals who suffer from chronic high cholesterol and other heart problems, these drugs have the potential to change their lives.  But how do we value these drugs taking into account their worth for people who need them and their overall value to society?

Value of Specialty Drugs Panel

This sets the stage for our first panel at our October 1st event titled Future of Specialty Drugs: A Policy Discussion on Treatments & Affordability.  The panelists are at the forefront of this current debate and include Katherine Wilemon, founder and president of the FH foundation; Steven Pearson, founder and president of the Institute for Clinical and Economic Review (ICER); and Josh Ofman, Senior Vice President, Global Value and Access, at Amgen.

  • Katherine Wilemon, founder of the FH Foundation, has been an advocate for the FDA’s approval of PCSK9 inhibitors. The FH Foundation is dedicated to raising awareness and research for Familial Hypercholesterolemia, which often goes undetected until an individual suffers a cardiac event and has, up until now been hard to treat effectively.  Wilemon has a personal interest in the approval as well- she has FH and had a heart attack at age 38.  In a Washington Post article about the federal advisory committee’s then upcoming recommendations she said, “’Without the new drug, my total cholesterol was about 200. The consistent message was that put me at severe risk’ for more cardiovascular problems because of the previous heart attack… ‘The bottom line is it’s very exciting for heart disease [treatment] in general, but absolutely essential for a population like ours.’”  For Wilemon and others like her, it might mean the difference between life and death:   “It represents a new era of hope for us,” said Wilemon according to a New York Times article.

It is too early to know whether the drugs’ ability to lower LDL cholesterol levels will correlate with a reduction in heart attacks and strokes. This is a key argument in how to assess its value (and in turn the price tag for consumers).

  • Steve Pearson doesn’t deny the innovation behind the new drug but does question its value and associated price tag.  Pearson is founder and director of ICER which released a new analysis recently arguing the PCSK9 inhibitors should be pricing significantly lower -$3,618 to $4,811 a year.  This is based on a calculating expected value through estimating an economic value for the drugs’ ability to prevent heart attacks and deaths and giving those gained years of life an associated price value (in economic terms a quality-adjusted life year). The “value-based price benchmark,” Pearson utilizes incorporates the benefits that treatment brings to patients along with cost implications. His argument rests on the lack of data that shows a reduction in number of heart attacks and cardiac events associated with taking the PCSK9 inhibitors.
  • Joshua Ofman disagrees with this methodological approach and resulting price point.  Ofman is a Senior Vice President at Amgen, one of the two companies that have the drug on review with the FDA.  He was recently quoted in an article in the Boston Globe as saying, “We disagree with ICER’s methodology, assumptions, and preliminary conclusions, none of which have undergone public comment or formal peer-review.”  Amgen has pointed to the review process used by ICER not assigning value to the benefit of meeting an unmet medical need and it is short term in terms of its valuation of the benefits.

In a panel discussion recently, Ofman said, ““As we focus on the costs of our interventions, we are often failing to recognize the costs of the diseases themselves.”  He raises the idea of innovating in public policy as a possible solution to this issue- finding unique ways to value and solve serious conditions while working together to improve the health of populations.

In the end, it seems, the panel can agree on the end goal: improving health of populations in need. How that is accomplished is open for debate.

Tip of medical needles

At the Tip of the Needle” by tschoppi is licensed under CC 2.0

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