Great Strides . . . Toward Drug Company Profits?

If you looked up the word "virtuous" in my dictionary, or that of any parent of a little kid with cystic fibrosis, you would find a picture of Dr. Robert Beall, president of the CF Foundation. He has a reputation, among everyone connected with CF, as savvy and indefatigable, obsessively dedicated to overcoming the disease. During the 13 years he has led the Foundation, CF has gone from being an incurable scourge that killed most people in childhood to a disease that is on its way to being cured. The median predicted survival age is now 37 years, and growing.

A hallmark of Beall's tenure at the helm of the Foundation has been its Therapeutics Development Program. According to Business Week magazine, the CF Foundation has paid drug companies $175 million, and counting, to work on developing new treatments for CF and its symptoms. In recent weeks the program has received a wave of publicity because one such CF Foundation-supported drug company, Vertex Pharmaceuticals, may have a winner on its hands. Specifically, for the first time, there is at least preliminary evidence of a drug that could, in at least some patients, fix the biochemical cell-wall defect that causes CF.

There is a dark cloud that goes with this silver lining, however. The CF Foundation is a big, mainstream charity that was started by CF families but which relies in no small part on the public at large -- hundreds of thousands of people who have no direct connection to the disease, do not automatically view Bob Beall as the visionary he truly is, are familiar with the scandals that have plagued other mainstream charities, and, most importantly, might be inclined to think that drug companies are faceless and greedy corporate behemoths bent on extracting windfall profits from sick people.

These people don't automatically think it's a great idea that the CF Foundation is taking their donations and turning them over to venture capitalists whose ultimate purpose is to get richer. I know this because, as a leader of Team Rose 66, I'm getting ready to take part in the Foundation's annual Great Strides fundraising walk on May 17 -- and the people I am asking for money are asking me some tough questions in return.

Here's the heart of the explanation of the program that appears on the CF Foundation's web site:

Current estimates suggest that it costs more than $800 million to move a drug from its concept stage to the market place. There is a critical need to help provide support to pharmaceutical and biotechnology companies that conduct drug discovery and early-stage clinical evaluation studies in small population diseases such as CF. Even with incentives, such as the Orphan Drug Tax Credit (that encourages investment in orphan diseases like CF, which affect patient populations of less than 200,000), the fact remains that pharmaceutical companies must first secure the financial resources to invest in these diseases.

Further, with increasing demands being placed on pharmaceutical and biotechnology companies, especially the small “start-ups,” investors are often hesitant about making major capital investments for orphan disease-classified drugs. The Therapeutics Development Program attracts researchers to the CF drug development process and shows a level of commitment unrivaled by any other voluntary health organization.

Fair enough. I'm delighted to be able to tell the donors to Team Rose 66 that the Therapeutics Development Program is a fundamental reason my daughter is likely to die, in old age, of something other than CF. But this allides certain ethical questions, ones that are at the core of the fundamental flaws in our health care system. Among them:

1. In a market economy that relies on investor-owned businesses to produce medical innovations, super-high returns on investment are supposed to compensate venture capitalists for the ultra-high risks of trying to develop new drugs. That's why so many prescription medications, including ones my daughter uses like Pulmozyme and Tobi, are so expensive. If those big bucks are inadequate -- which is the premise of the Therapeutics Development Program -- then, for the purpose of curing disease, shouldn't we consider alternatives to venture capitalism instead of propping it up?

2. If the problem is that CF is an "orphan" disease -- i.e., one with too few patients to make curing the disease profitable -- then isn't the Therapeutics Development Program just a mechanism for diverting societal resources away from curing truly widespread ailments in favor of working on a condition whose relatively small cohort of patients happens to be more wealthy and empowered than others?

"I favor all efforts to give people like Rose and those with similar diseases a better shot at a long, healthy life, and I understand that sometimes creative approaches and strange bedfellows are part of the process," said the friend who first raised concerns about the Therapeutics Development Program to my attention. "I just don't want to see the drug companies profit unduly."

It would help if the Cystic Fibrosis Foundation could offer the public reassurance that such undue profits are not where their donations to the Great Strides are ending up.