Rare Leader: Debra Miller, CEO and Founder of Cure Duchenne
November 16, 2017
Name: Debra Miller
Title: CEO and founder
Organization: Cure Duchenne
Disease focus: Duchenne Muscular Dystrophy
Headquarters: Newport Beach, California
How did you become involved in rare disease: My son Hawken was diagnose with Duchenne in 2002
Previous career: Magazine advertising sales
Education: BA Communications Studies; University of California, Los Angeles
Organization’s mandate: To cure Duchenne and to extend and improve the lives of those living with this disease.
Organization’s strategy: We focus on four areas: research, care, community, and access.
Funding strategy: For research in two ways: We raise funds through traditional nonprofit fundraising where we work with Duchenne families and co-host events. We work with celebrities, athletes, and we’ve been able to tap into the wine community. Our second way of funding is through our venture funding, our impact investing, where we will, instead of just giving money away, we thought there was a better way to be good stewards of that money and invest that money if there’s potentially a gain. That way if there’s a potential of a gain, we participate in the financial upside. That’s proven to be very successful. We’re on our third generation of leveraging these funds. For care and community together, we work closely with pharmaceutical and biotech companies for funding for educational outreach. For access, we’re able to get grants to pay for delivering drugs to kids in Canada, who were participants in the Drisapersen trial.
What’s changing at your organization in the next year: We are the leading organization working on CRISPR, and we will continue to expand our efforts with CRISPR and look for more impactful ways to maximize the potential of this particular science for Duchenne. We will continue to improve the standard of care for patients, and the outreach we have to the Duchenne community to offer better services and reach more patients. We also will be expanding our help to industry to help them be more effective in clinical trial design, regulatory issues, and access to drugs.
Management philosophy: We all have a consensus on the mission. That is the overriding theme of everything we do in this organization—cure Duchene and take care of the families. After that, I engage the team members to create their own paths to achieve their individual goals that will collectively achieve this mission.
Guiding principles for running an effective organization: For me, the most important thing is trust and communication. That communication starts even before I hire someone, by communicating the most important guiding principles for our organization, which is integrity and work ethic. We’re a small organization. We have to have a very committed team to achieve our goals. One thing that is important is hiring the right people. They have to be self-starters. I travel a lot. There’s nobody here who needs hand-holding. They need to be solution-oriented.
Best way to keep your organization relevant: As a mom, I live with this disease daily and live with the hope for a cure. My first-hand experience of the need keeps it very relevant. The most important thing for me is a sense of urgency that our organization has. We are not about growing our organization. We’d like to be out of business really. Our focus is not on the organizational structure, white papers, or platforms. It’s about getting drugs to kids and keeping them going as long as possible.
Why people like working for you: They clearly understand our mission and there’s no doubt about my commitment and my dedication to the families impacted. We have pretty good longevity here with employees because it’s really clear what we’re here to do.
Mentor: There was a gentleman, Bill Losse, I worked for at PC Magazine. He had the biggest impact on my professional life. He came in with what we all felt as a team were unrealistic expectations. A vail had been lifted from my eyes about what I thought I could do before and what was possible. I was able to see how magic happens by working together and setting a collective goal that’s really high.
What inspires you: His name is Hawken Miller (son)—and all the boys with Duchenne.
What makes you hopeful: The accelerated speed at which research is moving. We started a company with Dr. Eric Olson at UT Southwestern. The speed with which it is moving is incredible. He’s accomplishing in less than a year what it took several years with the early research we funded. We have more tools and we’ve learned so much about what not to be doing.
Best organization decision: To take a venture philanthropy model and be good stewards of the donor dollars. It’s painful to watch nonprofits work so hard to raise money from people who give them their hard-earned money and then to see it gone, especially if there’s a company that makes millions and millions of dollars off that. Also, hiring professionals with business experience. One of our best hires has been Dr. Mike Kelly as our CSO. As important as it has been that we’ve made really smart investment decisions and funding decisions, he’s also been at least equally important helping us avoid disasters.
Hardest lesson learned: Collaborate when there’s a shared objective and not to spend a lot of time entering into collaboration for the sake of collaboration, and when the objectives are different, or perhaps self-serving. Just stay focused on collaboration where we share a true objective.
Toughest organization decision: Passing on research and advocacy collaborations where the entire Duchenne community was supporting it, but our experts were advising against it. There was a lot of pressure put on us. It turned out we made the right decision, but it was tough to stand strong on that.
Biggest missed opportunity: Donating to Sarepta instead of investing in it.
Like best about the job: It keeps me sane. I think I would go stark raving mad if I had a son with this disease and I wasn’t doing everything I could to find a cure for it. Also, the variety. I never know when I walk into my office what opportunities are going to come up and they are all different. The constant learning is stretching me every single day. Most of all, the families we serve.
Like least about the job: Managing people.
Pet peeve: Dishonesty and lack of integrity.
First choice for a new career: I’d probably choose another really bad disease to try to help.
Most influential book: The Bible – everything you need to know is in there.
Favorite movie: The Imitation Game.
Favorite music: I love Country, even the sad songs are upbeat.
Favorite way to spend free time: Spending time with my husband and son.
November 16, 2017
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