top of page
  • Writer's pictureD. Maurice Kreis

Good News: No Room at the Inn in 2020 for Rose

Among other things, 2020 was a great year to stay out of the hospital. And that's exactly what my daughter Rose managed to do this year -- the first in recent memory. In honor of that feat, I'm back again with my perennial pitch to make an end-of-year gift in Rose's name to the Annual Fund of the Cystic Fibrosis Foundation.

Ever the strategist and schemer, Rose actually tried a few days ago to talk her way into Boston Children's Hospital for a couple of weeks. But her pulmonologist, Dr. Alicia Casey, was hearing none of it. Yes, is would have been the perfect time for such an interlude, given

Rose's plan to spend the winter in Florida, training with her horse Diego and studying remotely at Bowdoin. [The picture is of Rose and her roommate Victoria in Brunswick a few weeks ago]

But there was that pesky problem of Rose's FEV1 . . . of 100. You don't have to know that FEV1 means "forced expiratory volume over one second," the standard measure of lung performance. You can just guess, correctly, that 100 means perfect. And perfectly healthy people don't get admitted to the hospital. It's that simple.

Here's another simple truth -- a familiar one in our family. Without the work of the CF Foundation, Rose would almost certainly never have made it past Kindergarten. When the CFF got started in 1955, most CF patients died as infants or toddlers. Today, Rose and her fellow PwCF (that's my personal abbreviation for "people with CF") can and do expect to live well into adulthood. In due course, they will see in their lifetimes the CFF reduce cystic fibrosis to a fully controllable medical condition if not a disease that has been cured outright. The graph of the CFF's drug development pipeline tells the story in compellingly visual terms.

This being 2020, I've got a few more numbers to throw at you, related to the pandemic still raging around us.

According to the CFF's national patient registry, as of November 19 there have been just 431 cases of COVID-19 among the nation's more than 30,000 PwCF, and only three deaths. All three who paid the ultimate price (for the nation's failed pandemic response) were either post-lung transplant or had what is euphemistically referred to as "advanced lung disease." (One of those deaths, I am sad to say, was veteran Chicago undercover cop Marco DiFranco, whom COVID-19 claimed in April after his department refused to grant him medical leave.)

Those registry numbers are remarkable for several reasons, not the least of which is that the national CF patient registry exists in the first place. The CFF started the registry back in the 1960s. In today's data-driven world the existence of such a database is a no-brainer, but 50 years ago it was an astonishing leap of insight to conclude that keeping detailed track of how nearly every CF patient in the country is doing would yield enormous benefits. Thanks to the registry we know what works and what doesn't among all the ideas that have been tried over time. [Picture = Rose outside her Bowdoin dorm, Winthrop Hall.]

We did not hesitate to consent to Rose's participation in the registry the day after her diagnosis in 2002, and I am proud to say that Rose's data is part of this vast trove of insight. I never hesitate to roll that out whenever, in my day job as a public utility ratepayer advocate, people get all freaked out about using customer data to address our energy challenges.

But I digress.

A COVID-19 rate of less than 1.5 percent is also a tribute to the CF Foundation, which works so hard in partnership with its national network of accredited care centers to arm PwCF with the right infection control protocols. PwCF, after all, spend every day of their lives working to keep opportunistic bacteria out of their lungs. And as for the almost unbelievably small COVID-19 death rate, given that both the novel coronavirus and CF are respiratory diseases -- well, there are lots of theories bouncing around, so I will just say this blissfully reassuring phenomenon will be studied and then studied again and studied some more in the years to come.

Meanwhile, I've got another number for you: $3.8 billion.

[Crossing Longworth Avenue in style, thanks to the new walkway at Boston Children's]

For me, that's the elephant in the room whenever I ask my friends and loved ones to express their solidarity with Rose by donating to the CF Foundation. Because $3.8 billion is the sum the CFF has collected so far, from a company known as Royalty Pharma, in exchange for the Foundation's rights to royalties associated with the breakthrough "modulator" medications (Kalydeco, Orkambi, Symdeco and, as of last year, Trikafta) owned by the highly successful Big Pharma company Vertex Pharmaceuticals.

The original Royalty Pharma deal netted $3.3 billion in 2014, but the CF Foundation retained the rights to 50 percent of the royalties if global sales of the Vertex drugs went above $5.8 billion. Apparently that benchmark was approaching so Royalty Pharma bought out the CFF's remaining rights this fall for another $575 million.

The sum of $3.8 billion is one hell of a return on an initial $150 million "venture philanthropy" investment by the CF Foundation. So why am I asking you to throw your modest contribution onto this pile?

Here's why. Truly conquering cystic fibrosis is going to cost upwards of $9 billion according to one estimate I've seen. Rose, in particular, needs the benefit of the CFF's "Path to a Cure" initiative because she's among the 10 percent of CF patients who don't benefit from the Vertex modulator drugs in light of her rare mutations. Rose is part of an orphan population within what is known in the health policy realm as an "orphan disease."

Moreover, I am a transparency maven and thus I was impressed when Foundation CEO Mike Boyle and COO Marc Ginsky convened a live webcast and answered every single question from the CF community (including a pointed one from me) about the latest development in the Royal Pharma deal. Then, a few days later, the Foundation's senior vice president for policy and advocacy, Mary Dwight, reached out to me directly to update some factual information that appeared in my blog post about all of this.

In other words, I can put my personal reputation as a cynic, crank, troublemaker, iconoclast, advocate, and journalist on the line here because I know these people personally. I don't know what Mike, Marc, or Mary (or any of the many other CFF employees I've met) think when they see me coming, but in them I see integrity, focus, and kindness. You can and should send them some money even though they already have a lot of it in the bank.

Finally, and thinking especially of Mary Dwight and her CFF public policy team, I have to add that above and beyond what the Foundation is doing to conquer CF, this organization is fighting the good fight when it comes to saving civilization. And it's the threat to civilization rather than any lack of medical progress that is my biggest worry when I think of my daughter and her future. [Here's a selfie I took with Mary Dwight a year ago.]

How proud I was to see the CFF join a pile of other health-related and patient-focused charities in defending the Affordable Care Act by filing an amicus brief at the U.S. Supreme Court in the Texas v. California case that was recently argued. How pleased I was to call on the New Hampshire and Vermont congressional delegations in late February -- just before everyone stopped traveling and meeting in person -- as part of the CFF's annual "March on the Hill" lobbying event.

And how reassured I was to see Mary and her colleague J.P. Clancy (the Foundation's vice president of clinical research and another longtime friend) speaking and actively participating in a big virtual meeting convened in April by the Institute for Clinical and Economic Review (ICER) to pore over its finding that at $312,000 a year Trikafta, though a breakthrough, is not worth the price. I was there too, at ICER's invitation, to participate as a representative of the CF community. ICER is unfairly vilified by many of my fellow CF activists, so by their mere presence Mary and J.P. were making an important statement. Plus they had a lot to say about the importance of not allowing people with CF to get caught in the cross-fire between Big Pharma and the health insurance industry. [Photo = getting ready to board the Senate subway at the Capitol.]

So, as you ponder where to deploy your end-of-year, giving season and tax deduction season gifts among all of the worthy charitable nonprofits out there, please consider adding the Cystic Fibrosis Foundation to your list. The other cynic and iconoclast in the family -- Rose -- will appreciate it. So will I.

101 views0 comments


bottom of page