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  • Writer's pictureD. Maurice Kreis

The CFF Cashes Another Big Check

To know me is to know that my favorite charity is the Cystic Fibrosis Foundation. Just as the March of Dimes cured polio, so too is the CFF leading the inexorable march to reducing Cystic Fibrosis to a curious medical footnote -- a controllable condition if not a disease that has been vanquished outright. That's really something for a condition that was, before 1938, an unidentified and mysterious reason why certain babies (the ones whose skin was salty to the taste) died before even becoming toddlers.

It is no mere academic interest for me. I'm a cystic fibrosis dad and, thus, as the landing page of my blog proclaims, a militant CF heterozygote. My sweet baby girl, born in 2001 with CF, is today a happy and healthy young woman who is in her first year at Bowdoin College. Without the CFF, I'm certain she would be 15 years dead. So, yes! I think you should follow the "share this hug" link and make a generous donation to the CFF.

But . . . I've got some questions after reading this message received today from Mike Boyle, the CEO of the Cystic Fibrosis Foundation:

Dear Friends,

More than 20 years ago, the Cystic Fibrosis Foundation took the pioneering step of providing research funding to Aurora Biosciences (now Vertex Pharmaceuticals, Inc.) to identify and develop the first treatments for the underlying cause of CF. Driven by the relentless determination of the CF community, those investments led to the development of multiple transformative therapies to treat CF. In 2014, the CF Foundation sold rights to royalties related to those therapies to a third party group called Royalty Pharma -- generating critical resources to reinvest in the Foundation's mission at an unprecedented rate.

The funds from that sale are being used to accelerate the next generation of transformative therapies, support high-quality care and programs for people with CF and their families, and pursue bold new opportunities to nd a cure for all people with CF. We have made incredible progress, but our work is far from done.

Today, I am pleased to share that the CF Foundation has successfully completed a sale of its remaining stake in royalties related to Vertex's CFTR modulators for an upfront payment of $575 million and a potential future payment of $75 million, bringing additional resources to the fight against CF.

This sale comes at a time of great change and opportunity in CF. Our goal in pursuing this agreement -- and for all resources we receive through our continued fundraising and venture philanthropy programs -- is to maximize the funds available to fuel our mission. With these resources, we are better positioned to address the challenges that people with CF and their families continue to face today and move faster and further down the path to a cure. 

The speed and thoroughness with which we can pursue better todays and add tomorrows for people with CF are only possible because of successes like this and the resources the CF community has entrusted to us over the years.  We want every person to have access to the best therapies for their disease and are committed to ensuring that people with CF have what they need to live the longest, healthiest lives possible. Thank you for your enduring commitment and your continued support of our shared goal: a day when CF stands for Cure Found.

With gratitude,

Michael Boyle, MD

President and CEO

The 2014 sale of royalty rights to which Mike Boyle refers resulting in the CFF cashing a check in the amount of $3.3 billion. That immediately catapulted the Foundation to nearly the top of the list of wealthiest charities in the world dedicated to fighting a single disease. Add today's $575 million to the 2014 cash-out and that makes $3.8 billion dollars the Foundation has received as the reward for its "venture philanthropy" related to the miracle "modulator" drugs that are now sold on a monopoly basis by Vertex Pharmaceuticals. We're talking here about Kalydeco, Orkambi, Symdeko and, especially, Trikafta. As for each of those drugs, the Foundation's venture philanthropy is the reason Vertex (or its predecessor, Aurora) was willing to pursue bringing these important medications to market even though CF is a so-called "orphan disease" with only 30,000 patients nationwide.

There is much controversy these days about the outrageously high retail price of the modulators -- especially the $312,000 annual per-patient pricetag for Trikafta. The Institute for Clinical and Economic Review (ICER) recently determined that Trikafta is not cost-effective at that price and wouldn't even be worth that price if Trikaftka were a cure for CF outright (which it isn't).

But the CFF has been stolidly silent when asked to weigh in on questions related to the prices of the Vertex modulators. Basically, the CFF has said two things: First, it's better to have these drugs, and argue about their cost, than not have them. (I agree.) Second, the CFF plays no role in determining the price of the drugs.

Today's announcement seems to undermine the latter claim. Since the 2014 sale of royalty rights, the Foundation has allowed people like me to think that the CFF had completely divested its rights to any part of the income stream from these drugs. Turns out my assumption is mistaken. If, prior to this most recent sale of royalty rights, the Foundation still had the rights to some portion of the modulator-related income stream then surely it had some role to play in determining what patients and their insurers would pay for these drugs.

I've been clamoring for Vertex to be transparent about its modulator prices so that the public can know whether the prices it is charging are reasonable. Now, in a similar spirit, I call on the CFF likewise to get transparent about its past and present financial stake in CF drugs that are currently being prescribed to patients. And I reiterate my call for the CFF to use its moral authority to pressure Vertex to stop charging outrageously high prices for its modulators, both here and around the world (including all of the countries where modulators are simply out of reach because Vertex refuses to reach agreement with government payors).

Meanwhile, I sometimes get asked: Why, if the CFF has billions of dollars in the bank, should we continue to send our small donations to this organization? Well, here's why: The CFF is not like Princeton or Middlebury or Bowdoin (to name three ultra-rich academic institutions has educated or is educating my family). Those institutions build up huge endowments with the objective of keeping them, and their schools' luxurious campuses, forever. By contrast, the CFF may have taken in $3.8 billion in exchange for royalty rights but it has been estimated that curing CF will cost at least $9 billion.

So, yeah, as the end-of-year giving season approaches, and you want to show some solidarity with my family, do keep the CFF on your list.

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