After a few years of ruminating over the growing problems that slowed or prevented the development of treatments for the rarest of diseases, this is the year I decided I needed to do something about it. Naïve and optimistic, there is no other way to conceive tackling what many think is an impossible task: reforming development for rare disease treatments. In the nearly 12 months from the time I sat in a coffee house with John Ditton, now our COO, and told him about the foundation and the need to change the world in this way, we have bolted out into the open and are driving a new agenda around improving how rare disease treatments get developed. People have heard us, and the discussion is beginning.
Starting a foundation in a crashing economy might seem foolish because it is hard to get the funding for established charitable activities, let alone those just beginning. But I made the decision to just pay for it (ok and my wife agreed too) and press hard for an answer. Financing solved. A few have begun to help, but I won’t stop to wait for money. Just pay it and go.
I visited and tested a number of my early ideas with those most knowledgeable about the current situation like Frank Sasinowski, Marlene Haffner, Steve Groft, and others, and honed and focused the goals for the foundation to the 3 most critical and achievable goals. With the help of our first creative support team at Naganuma Design, we named the campaign for change, CureTheProcess, applying the right medicine to a development process for medicines. The first team of 4 people, John, Julia, Louisa and me, worked out of a hotel room for 3 months, and then finally settled into space here in Novato, and picked up steam. We added support from Steve Smith, for patient group endorsements; Phil Hays for our Harley riding, part time controller; and Brigitte Miyamoto, our first CalTech trained intern doing research finding drugs that have been blocked in development. Annie now has replaced Louisa and added her non-profit experience to the team. A small team but effective and I thank all of them for their dedication to a cause that earlier this year, was mine alone.
We burst on the scene in the May 14, 2009 Rare Disease Summit organized by the National Organization of Rare Diseases (NORD) where I had the chance to put our 3 goals in front of patient group leaders, patients, industry professionals and most notably senior FDA officials. No doubt, the concerns I raised put tension into the discussion and while no one likes tension, sitting still and not carrying forward the urgency to our fight to do the best for our patients and families is also not right.
Our goals we set then were to get the best people in place at FDA, and give them the best tools for studying and approving drugs for the thousands of rare disease that are still left out. You can read more at www.CureTheProcess.org.
Our goals and our messages have resounded with others including 70 patient organizations that have now formally endorsed our campaign, and the numerous patients, physicians, parents, and others that have provided us positive feedback and endorsement of the need to do more. A number of others have supported our issues in their public statements, and the issues around rare disease drug development policy are now the focus of an Institute of Medicine report commission, the Brownback/Brown amendment to the FDA appropriations bill, and a new interagency task force being formed to look at regulatory issues. Many at the FDA have also been receptive to looking at what we are doing and figuring out how to do better. They want to get it right too. Of all the feedback and support, the one that matters to me the most is what the patients and parents are saying who have nothing right now. They want something done, and they want it now, and they are happy we formed the foundation to do it.
2010 is the year to make things happen. Health care reform should be behind us. I have had some detractors tell me that say that we should wait, take our time, and that change won’t come easily or that the FDA won’t respond to suggestions for change from the outside. I have started enough things from scratch to know that everything can seem impossible at first, but bit by bit, step by step, you can win, working hard and keeping a clear sense of direction as to what is right, and having the data to prove it. The FDA will listen because there are people there that want to get it right and know how complicated these diseases are. We also have 70 patients’ organizations telling me to get it done. We have supporters in Congress that want improvements. I won’t slow down as the people that matter most to me, patients and their parents cannot slow down their disease to wait for us. It goes on, unabated and unrepentant.
My father was a neurologist who had lots of patients with incurable progressive problems. He told me once about managing a teenage patient with a terminal brain tumor, that sometimes, the best you can do is hold their hand. Well, holding hands is not good enough anymore. We can do better. CureTheProcess.
