A closer look at the downside of the Vertex – Cystic Fibrosis Foundation deal

And some comments from the Knight Science Journalism Tracker: 

A new cystic fibrosis drug–the first to be developed based on understanding of the genetics of the illness–is an orphan drug that can help only about 4 percent of patients with cystic fibrosis. Its financial toxicity is such that it can destroy the finances of those whose lives it saves: It costs $307,000 per year. And it sits in the center of a swarm of conflicting interests involving a pharmaceutical company, a non-profit patients’ organization, and researchers who have taken their money

This comes from John Fauber of the Milwaukee Journal-Sentinel, whodescribes a disturbing case 


Click here for the MJS story:

What happens when a disease-fighting charity dives into venture capitalism?

In the first case of its kind, the results include one of the planet’s most expensive pills, huge sales projections for a drug company and windfalls for executives who sold stock in the glow of enthusiastic news releases abo

ut the drug.

downloadKalydeco is a breakthrough drug designed from knowledge of the genetic roots of cystic fibrosis, a lung disease that kills most victims before they reach middle age. Developed by Vertex Pharmaceuticals with a $75 million investment from the Cystic Fibrosis Foundation, it is an early example of “venture philanthropy,” where a nonprofit helps finance development of a treatment in return for a cut of sales.

Yet it costs each patient $307,000 a year to take two Kalydeco pills a day – a price borne by taxpayers through Medicaid and other government programs and by the workers and companies who finance employee health insurance plans.
Much of the initial science behind Kalydeco involved nonprofit research universities and hospitals, with funding from the Cystic Fibrosis Foundation and by taxpayers through the National Institutes of Health.

In 2012, with less than a full-year on the market, Vertex sold $172 million worth of Kalydeco and the foundation cashed in by selling future royalties from the drug to an undisclosed firm for $150 million. The foundation is investing that money with Vertex and other companies to develop more new drugs.

For more on the links between patient advocacy groups and drug companies, see TR’s  2006 Washington Post story:

Some groups’ Web sites feature the names of their corporate donors, but often these names appear only in an annual report. Exact donation amounts are rarely listed, though some groups are willing to provide numbers on request.

People who look to these groups for independent information may never learn about their financial ties to drug makers. That needs to change, say some consumer advocates, now that nonprofits are playing a larger role in drug safety debates. When disease advocacy groups weigh in on a drug up for review by the FDA’s Drug Safety and Risk Management Advisory Committee, they say, the public may be unaware of potential conflicts of interest.



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