Nonprofits selling access to research data a growing movement

Simon's Fund, the local nonprofit that highlights the dangers of sudden cardiac death in children, has invested about $200,000 to build the first national registry for adolescent hearts. HeartBytes will collect data and imaging at screenings of students and young athletes nationwide and make it available for research into the puzzle of sudden cardiac arrest.

Current plans call for academics to have free use of the data. But if "someone comes along who does have funds, like Pharma, I would entertain a fee for access," said Darren Sudman, cofounder of the fund named for his late son, Simon. "This will help ensure the sustainability of the registry."

It is known as venture philanthropy - a growing movement that includes the idea of a nonprofit selling access to its data for profit. Nonprofits make investments that may pay dividends down the road, somewhat like what big academic institutions have been doing for years. It differs from traditional sources of funding for nonprofits, which most often depend on donations from foundations, corporations, and wealthy individuals.

This form of venture philanthropy garnered attention last year when the Cystic Fibrosis Foundation sold its rights to royalties from Kalydeco - a drug that helps about 4 percent of patients with the inherited disorder - for a landmark $3.3 billion.

To develop a treatment, the foundation had invested $150 million over 15 years in Aurora Biosciences of San Diego. Eventually, Aurora was acquired in a $600 million deal by Vertex Pharmaceuticals in Boston, which continued to develop what was then called VX-770 and in 2012 won regulatory approval to market the drug.

The Cystic Fibrosis Foundation still owned the rights, which it sold two years later - for a record gain on investment by a health nonprofit - to Royalty Pharma in New York, which buys intellectual property and other rights to drugs.

National nonprofits involved in venture philanthropy include the Multiple Myeloma Research Foundation, Muscular Dystrophy Association, and JDRF (formerly the Juvenile Diabetes Research Foundation; the disease is now called type 1 diabetes).

Though experts agree venture philanthropy is likely the wave of the future, some harbor concerns about ethical conflicts that could arise from the investments.

"I do see this as a trend," said Arthur Caplan, director of medical ethics at NYU's Langone Medical Center. "Anytime you have a huge hit" - say, $3.3 billion - "it attracts others with the same dream, particularly in the area of orphan diseases, which traditionally don't attract as much investment. But remember that the odds of making it from mice to the pharmacy shelf is 40- to 50-to-one."

Caplan notes that, as business partners, nonprofits may be investing with entities whose goals include helping patients but who are in it mainly to make money.

"Another question for nonprofits is privatizing research: how much to spend on research and how much on services for current patients. And then there are tax issues: Nonprofits say they are not-for-profit, but here they are acting like investors. And what happens to the money?" Caplan asked. "People are going to be looking more closely at nonprofit directors' salaries, the size of their buildings."

Katherina Rosqueta, executive director of the University of Pennsylvania's Center for High Impact Philanthropy, agrees that deals like the Kalydeco one will likely encourage other foundations to invest.

"But whether nonprofit boards are capable of doing due diligence and making these investments well and in an informed way is open to question," Rosqueta said. "You need to have both the patience and the ability to take those kinds of risks and the capital to take those risks." Few nonprofits have that kind of money.

There is also the danger of blurring the lines between financial success and medical and service missions.

"What matters first is why the nonprofit goes into the investment: not to make money, but to treat a disease," said Denis Hadjilidis, a physician who directs Penn's cystic fibrosis program for adults.

"The second most important thing is that the majority of profits made should go back into the same idea," he said. "If it's used for a different purpose, say to grow the foundation, that's not right."

Yet another complicating issue: how to price drugs that were produced using nonprofit investments. Kalydeco (generic name: ivacaftor) costs $300,000 a year and must be taken for life.

Though insurance and drug companies pick up much of the tab, some have questioned why a drug funded with nonprofit investment is not more reasonably priced.

"Patients may say, 'A lot of money is coming back to the foundation, but that doesn't help me,' " Caplan said.

Still, Rosqueta sees venture philanthropy as a good example of investments that yield positive social impact.

"Change has always been achieved between the private, philanthropic, public, and commercial sectors," she said. "In the case of Kalydeco, a medical treatment exists today that didn't exist before. Most people would say that is a very good thing."

Simon's Fund was started nine years ago when Darren Sudman and his wife, Phyllis, who live in Plymouth Meeting, lost their 6-week-old son to sudden cardiac arrest. The likely cause was Long QT syndrome, an undiagnosed heart rhythm disorder that causes chaotic, accelerated heartbeats.

It is unclear whether an electrocardiogram could have saved Simon's life, but the search for what killed him led to the discovery that Phyllis had the genetic anomaly and probably passed it on to her son.

The couple emerged from their grief - and considerable research - convinced that EKGs are the most effective way to screen for conditions that can lead to sudden cardiac arrest and death in children. (Although it is far more common among the elderly, sudden cardiac death in children and young adults can come as a shock, and the issue has gotten attention after the deaths of some well-known athletes.)

Over the years, Simon's Fund has sponsored screenings - including EKGs and heart ultrasounds when other findings suggest a need - for 10,000 students in and around Philadelphia. About 100 heart problems have been uncovered. All of those records are now in the fledgling computer registry, funded so far by donations.

HeartBytes is an opportunity for the nonprofit to be "entrepreneurial," said Darren Sudman, a lawyer who is now the organization's executive director.

"Instead of following the path of raising money and giving it to research," he said, "we saw the chance to do something different."