The $3.3 billion question: Why donate to the Cystic Fibrosis Foundation?

[Here's the link to my 2017 CFF Annual Fund campaign.]

An interesting factoid about the Cystic Fibrosis Foundation (CFF):  It has reserves of approximately 12 times its operating budget.  That makes the CFF among the wealthiest, if not the wealthiest, healthcare charities in the country.

Since I have smart friends, it’s hardly shocking that one of them would ask about this in response to my customary end-of-year pitch on behalf of the CFF annual fund.  Here’s my take on the full story, as I understand it.

The CFF is a dynamic and innovative organization.  It has to be.  The disease the Foundation is striving to conquer is a so-called orphan disease.  Even though one in 26 people in the U.S. carries a genetic mutation that causes CF, fewer than 40,000 of them actually have CF because that requires a CF mutation on both ‘sides’ of chromosome 7.

One innovation pioneered by the CFF is known as “venture philanthropy.”  Basically, the CFF partners with drug companies – co-investing, alongside big pharma, in the research needed to develop new drugs that will be effective against the disease.

Among the drug companies with which the CFF has partnered is Vertex Pharmaceuticals.  And, a few years ago, two Vertex drugs developed for CF patients hit paydirt.  The drugs – Kalydeco and Orkambi – are breakthroughs in the sense that they are the first FDA-approved therapies that actually treat the root cause of CF at the cellular level – the failure of a particular protein (called CFTR) to emerge from the mitochondria inside cells, migrate to the cell wall, embed itself in the cell membrane and thereby open the chloride channels that allow salt water to pass through the membrane.

This was such a big deal – such a huge breakthrough – that the financial implications alone were staggering.  In 2014 the CFF was able to cash out its share of the revenue stream associated with these drugs to the tune of $3.3 billion dollars.  That’s billion with a B.  Cashing that check is the reason the CFF has such a big reserve fund.

So why not just let that candle burn itself down a few years before people like me resume asking our friends to donate their hard-earned cash to the CFF?

Well, let’s start with the selfish reason.  It’s on my license plate: N1303K.  That’s the CF mutation I ‘contributed’ to my daughter – half the reason she has CF.  N1303K is a relatively uncommon CF mutation and it is, so far, not among the mutations for which Kalydeco and Orkambi have been proven effective.  I need the CFF to press onward until it has come up with therapies that work for people with all CF mutations.

According to the CFF, it’s taken expenditures of roughly $3 billion to get us from where we are today – the journey from an unknown reason for babies with salty skin to die, to a condition discovered in 1938 (by the chain-smoking lesbian pathologist Dorothy Hansine Andersen at Columbia Presbyterian, who noticed odd cysts on the pancreases of infants whose bodies she was conducting autopsies) to a disease whose median life expectancy is now in the early 40s.  I have a feeling it is going to take us more than $3.3 billion to get us the rest of the way.

That journey – and here we come to my less selfish reasons – will help more than just my daughter, more than just people with exotic CF mutations, and, indeed, more than just people with CF.  The CF Foundation has pioneered reforms that have changed healthcare.

My favorite example is the CF Patient Registry, started by one of my great heroes, the late Warren Warwick, a pulmonologist from the University of Minnesota.  The registry keeps data on every CF patient in the U.S. (at least those who don’t opt out) and we thereby have a deep mine of information about what works and what doesn’t work in treating the disease.  It seems like a no-brainer – but nobody was doing this as to any disease until Dr. Warwick started doing it for the CFF.

Another great CFF idea:  accrediting care centers.  Every hospital that treats cystic fibrosis must meet rigorous care standards set by the Foundation on behalf of the people who have the disease.  You don’t need a PhD in health care quality improvement to grasp the value of this.  When the bean-counters at the hospital that cares for my daughter start hassling the head of the CF care center, he can (and does) look them back in the eye and tell them that to stay accredited they can’t cut corners.

The CFF has been a pioneer in patient and family empowerment.  Right about when my daughter got her CF diagnosis in 2001, the Foundation acknowledged the reality that CF patients spend 98 percent of their time away from the care team at the hospital – ergo, it’s the patients and their families who are the real experts in treating the disease and maybe deserved a voice in how the standards for CF care are developed.

I could go on; the Foundation’s web site has many more examples – and an impressive pipeline of new therapies that are working their way through the FDA approval process.

Still, I must admit, donating to the CFF is a bit like donating to my alma mater (the very well-endowed Middlebury College).  Except it isn’t – not completely.  The CFF is not building edificies and adding institutes and satellite campuses so that it has an increasingly impressive and permanent presence.  The CFF is paying cool people – PhDs with bizarre specialities, doctors with deep insight, nurses who deliver the real healthcare – to do what their life’s passion is calling on them to do.

Oh, and did I mention that the CFF is also devoting resources to principled and aggressive advocacy?  The Foundation has been stalwart in its ongoing effort to thwart repeal of the Affordable Care Act, and it will be there to defend Social Security Disability because it knows these things are essential to the survival of people with chronic illnesses like CF.

Donating to the CFF is not going to be for everyone.  Even if you prepared for college at the prestigious St. Paul’s School – as did the friend whose inquiry prompted this essay – and even if you love the school (as even I do, living nearby and enjoying its campus), you might reasonably conclude that it doesn’t need any more money.  I guess you could draw the same conclusion about the CFF – but I do not.  I send the CFF enough money each year so that I notice the ‘hit’ not because the CFF needs my cash, but because I need to feel connected to its work.  I ask others to donate because I know that some of them will likewise take real joy in having that bit of palpable connection to my family and its challenges.  Some of them won’t – and for those folks, there are other worthy charities (some of which I donate to as well).  That’s okay too.

I’d be the last one to say it’s a blessing to have CF, or to be the parent of a person with CF.  But it’s a blessing to be connected to all of the insight, and passion, and plain-old human persistence and achievement that emanates from the community of people affected by this disease.  The big reserve fund notwithstanding, inviting others to share in that blessing is something I do without hesitation.

[If I've convinced you, here's that link again.]