Book Excerpt from The Power of Rare

In chapter 4, “Building a Cure Machine,” author Victoria Jackson reveals the challenges in launching a foundation focused on funding research on a rare disease.

May 1, 2018
Victoria Jackson, Michael Yeaman

Regan Arts, September 2017Curing a disease is not for wimps.

Even before the ink was dry on our foundation charter, that was something I knew instinctively. And it has served as a mantra ever since—a reminder that has kept me on my feet, especially on those fearful days when all I’ve wanted to do was curl up in a fetal position in the corner. Now that we’d gotten everyone into the room and had created incentives for sharing, the greater challenge was figuring out how to translate all that brilliance and life-saving science and patient insight into the moving parts of a real-world, practical delivery mechanism for change.

Such was the vision for our cure machine.

Questions and challenges abounded. Not just what had to be done—but how. How did we connect and synchronize our many different moving parts? How would we overcome the challenges of rare—to map the “where” and “when” and “who” of NMO around the world? How could we get enough blood and data to our researchers when the patient population was so small? How could we help educate the medical community to end the nightmare of misdiagnosis? How could we constructively rattle the cages of the medical establishment to speed more accurate diagnoses for new patients? How could we best respond to the needs of the newly diagnosed? How would we engage industry to invest time and resources in treating and curing this rare disease? And until then, what were the needs of those who had been living with NMO for much of their lives?

These were only some of the challenges before us. What we had to build couldn’t be any kind of conventional system or process. Why so ambitious? Because if our machine worked, we knew that the field of NMO would change radically and rapidly. So we had to invent and then put into practice a streamlined system of strategies and actions that could respond to change and overcome obstacles we didn’t even know existed yet—as they arose. We had to reshape time. Our cure machine had to be progressive and adaptive.

Or put more simply—necessity had to become the mother not only of invention but also of implementation. When lives are at stake, you rise to the challenge or you fall down. No passes given to the faint-hearted. Don’t get me wrong. There was nothing about what we were up against that was easy or that didn’t scare me most of the time. No question. My solution, rather than pretend this was going to be easy, was to take all that fear and uncertainty and distill it into my personal Rx to keep going. The fire inside of me would have to fight the fire outside of me. On a quest to stop a killer on the loose, I could not be in denial that I was afraid. But I still couldn’t be a wimp. Problem-solving, fearful though I was, would show me the way to picking up my sword and putting on my armor.

In the process, there would be many lessons to be learned from what went right and what had room for improvement. We wouldn’t have the option to stop and pat ourselves on the back. We would have to keep pushing forward, adjusting and adapting the blueprint in response to challenges met on the ground. And there would be plenty of those. Innovations don’t always appear in the middle of the night as a brainstorm out of thin air. Luckily, one of our first steps for creating connections was something that did sort of happen that way.

Excerpted from The Power of Rare: A Blueprint for a Medical Revolution, by Victoria Jackson with Michael Yeaman. Copyright © 2017 Victoria Jackson. Used with permission of the publisher, Regan Arts. All rights reserved.

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