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by Monica Coenraads


This past November the Cystic Fibrosis Foundation (CFF) announced an unprecedented windfall: $3.3 billion from selling royalty rights to drugs that it helped develop to treat Cystic Fibrosis. The payout is the largest financial return ever secured by a disease non profit. The CFF is the gold standard for anyone working in the disease non profit world.

Years ago I had the good fortune to meet the founder of CFF, Doris Tulcin. She now serves on our Professional Advisory Council.  I consider myself lucky to count Doris as well as the CFF CEO, Bob Beall, among my mentors. Their $150 million investment for this particular drug has paid off handsomely and I couldn’t be happier for them and for the entire CF community. It’s been interesting however to see the spectrum of opinions regarding this windfall. Below are two polar opposite commentaries on the subject.  I encourage you to read them both.

In case you are wondering whose view I agree with…it’s Peter Kolchinsky!

Rethinking Venture Philanthropy After the Kalydeco Windfall
by Peter Kolchinsky

KolchinskyThe Cystic Fibrosis (CF) Foundation’s big win in venture philanthropy can fuel constructive competition among companies developing innovative CF drugs, benefiting both patients and the healthcare system by increasing future treatment options and reducing their cost.

CF is a fatal genetic disease affecting around 30,000 people in the U.S. that is caused by mutations in the cystic fibrosis transmembrane receptor (CFTR) gene. These mutations disrupt either the expression or function of the CFTR protein, causing mucus buildup in the lungs that can impair breathing and lead to infection. Although the most severe symptoms of CF impact the lungs, the disease also leads to a shortage of the pancreatic enzymes needed for digestion.

The vast majority of drugs marketed to treat CF address the symptoms, and not the cause of the disease. Ivacaftor (Kalydeco), a drug from Vertex Pharmaceuticals (NASDAQ: VRTX) that was developed with an investment from the CF Foundation, is the only therapy available that addresses the underlying cause of CF, though currently only for a small fraction of patients with particular mutations. Vertex is developing other drugs, so-called CFTR correctors, that can be combined with ivacaftor to address more CF patients.

Royalty Pharma’s $3.3 billion purchase of the CF Foundation’s roughly 10 percent royalty on Vertex’s CF drugs last month sparked some controversy. Articles in the New York Times and Xconomy suggested that the foundation had somehow failed patients by allowing Vertex to price its drugs so high that a 10 percent royalty could be worth so much. These criticisms echo those directed at the foundation when ivacaftor hit the market in 2012.

Ivacaftor’s price tag, about $300,000 per year, per patient, shocked the market. Critics declared that the CF Foundation should have done more to ensure an affordable price for patients. They further insinuated that the drug’s price was evidence that the foundation had a conflict of interest; it could not simultaneously serve patients and fund biotech companies. In the wake of the multibillion-dollar royalty sale, critics are now repeating these same accusations.

These critics are missing an important part of the CF Foundation’s strategy. While the foundation could not possibly have any leverage over how Vertex priced its drug, by harvesting $3.3 billion now, it will be able to speed the development of over a dozen early competitors. This will usher in an era of competition that will help make the CF therapies of the near future not only better, but also less expensive—long before Vertex’s drugs go generic.

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Stop Subsidizing Big Pharma
by Llewellyn Hinkes-Jones


Robert J. Beall, the president and chief executive of the Cystic Fibrosis Foundation, called his recent decision to sell the royalty rights to his organization’s research a “game changer.” Indeed: Deals like this, in which an investment company paid the foundation $3.3 billion for its future royalties from several cystic fibrosis drugs it helped finance, could revolutionize the way medical research is funded. Rather than the staid model of government-funded institutions handing out grants to academic research facilities, a new breed of “venture philanthropies” like the Cystic Fibrosis Foundation could corral private investment into developing lifesaving drugs quickly and cheaply.

The problem is that venture philanthropy is, essentially, another term for privatizing scientific research. Instead of decisions about the fate of scientific funding being made by publicly oriented institutions, those decisions are being put in the hands of anonymous philanthropists and ostensibly benevolent nonprofits.

At the risk of oversimplification, biomedical research divides into two categories: private and public. The former is the constellation of big pharmaceutical companies and start-up labs. The latter comprises government agencies and the universities and philanthropies that rely on government support — directly, through grants, or indirectly, through tax policy. The former can charge whatever it wants for its products; the latter is limited by government rules and price controls.

Venture philanthropy complicates this picture by introducing a tax-exempt loophole. An organization like the Cystic Fibrosis Foundation will take in tax-exempt donations to invest in a pharmaceutical company — in this case, Vertex Pharmaceuticals — to develop drugs based on publicly funded research. Venture philanthropies can then sell the results of that research to private industry to deliver drugs to the market.

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Rett Syndrome Research Trust Website

Doris Tulcin – A Mother’s Love Raises the Bar For All Non-Profits

Half a century ago, a mother whose baby daughter was diagnosed with a life-threatening genetic disorder decided to fight it. Doris Tulcin is that mother, and Cystic Fibrosis is the disease against which she went to war. First identified in 1938, Cystic Fibrosis affects roughly 30,000 children and adults in the United States and is therefore, like Rett, classified as a rare disease. In 2010 the Cystic Fibrosis Foundation generated donations in excess of $300 million, $120 million of which was raised from the general public. Now, Vertex Pharmaceutical’s VX-770, the first drug that treats not just symptoms but the underlying genetic cause of Cystic Fibrosis, has been fast-tracked through FDA approval.

Doris Tulcin has been a valued advisor to RSRT since our inception three years ago. The following in an excerpt from a recent conversation between Mrs. Tulcin and RSRT Executive Director, Monica Coenraads.

MC:  Congratulations! You must be thrilled with the early FDA approval—it’s been a long road and I know CF families are rejoicing today!

DT:  It’s mind-blowing!  VX-770 is for a small group of patients that have a rare mutation but will literally change the disease and these kids completely.  And that’s what we are working on now for 90% of the patients with the major mutation.  Things are moving really well.

MC:  CFF provided substantial scientific, financial and clinical support for the development of VX-770, including the investment of $75 million.  When I think about the vast amount of money CFF raises, with a patient population similar to Rett, I can’t help but ask myself, How have you done it?  Are there specific decisions that you think have been pivotal?

DT:  Well, you must remember that we have a long history; we’ve been in business since 1955.  So it took over fifty years to get to where we are today.  Going back in time, it is so hard to pinpoint any one thing.  But we really focused on research, as you are doing.

MC:  Of course, the scientific tools and knowledge that were available fifty years ago are so very different from research today.  Everything has accelerated.  Yet I know that getting to this point with Cystic Fibrosis has been a process with many components.

DT:  From 1980 on is where we really took off with developing research programs with major universities, and by the 1990’s we became increasingly business-oriented and
focused on working with the biomedical community and pharmaceutical companies for drug development.

MC:  As with Rett syndrome, the Cystic Fibrosis population is small.  You know RSRT has a PSA that’s running in Times Square, the same screen that was used for the CFF.  So 1.5 million people will see it every day for three months.  How important do you feel it is to raise general public awareness, in terms of the ability to raise research funds?

DT:  Name recognition is very important.  The general public doesn’t have to understand the disease but they certainly have to recognize the name.  You know, people will say “I’ve heard of Cystic Fibrosis”— well, what do you think it is?  They can’t describe it.  They haven’t the vaguest idea, but they’ve heard of it.

MC:  And you think that correlates to the amount of money you raise?

DT:  I do. Our chapters raise roughly $100 million a year. But keep in mind that it took decades to get where we are today.

MC:  For RSRT, at the three-year mark, most of our revenue comes directly from affected families and their networks.  Do you think the same applies to you, or do you have people just spontaneously donating to CFF without any personal connection?

DT:  I think now we do, yes. But what we started with and built on was indeed that personal commitment and involvement to develop networks of family and friends.  The activism of those who are personally affected is crucial.

MC:  How would you compare the current philanthropic landscape to what you’ve seen in past decades?  These are difficult economic times for many.

DT:  Yes, we are still in a bad economy now and that reflects on contributions, there’s no doubt. It’s tough, and there’s always competition and distraction.

MC:  On a personal note, how is your daughter doing?

DT: Oh, thank God, she’s doing well.  She’s a grandmother!  My grandchildren have introduced me to Facebook, so we communicate in that way, because they’re all grown up and don’t have time for other things. But we do that too with the foundation, we’re on everything you can think of— Facebook, Twitter, YouTube.  Everything now is sent electronically.  The website is being updated all the time, you can get every bit of information at any time off that website.

MC:  Yes, the fact that electronic media is now ubiquitous worldwide has really changed the flow of information in important ways for families dealing with something like a rare disease. The ability to be in instant communication with others who really understand relieves some of the isolating strain of constant and intense caregiving, as well as keeping people informed of current developments. That families can so easily connect and learn from each other is vital and sustaining.  The day-to-day struggles can be so overwhelming sometimes for Rett parents that there’s not always a lot of energy left for what I feel is truly going to change our children’s lives—the research.  What are your thoughts?

DT:  There is no doubt that you really have to find ways to stay focused, and I think that was one of our biggest successes.  Care has been important but it is research that’s absolutely critical; it’s the only way to move forward.

MC:  Research has always been my commitment, and I hope the work of RSRT will prosper as brilliantly as that of the CFF. Congratulations again on VX-770 and
thank you for your guidance and inspiration.

DT:  Monica, I know how tremendously devoted you are to this fight. The Rett community is very blessed to have you.

MC:  Thank you, Doris.