Rare Leader: Margie Frazier, Executive Director of the Batten Disease Support & Research Association

The Basics
Name: Margie Frazier

Title: Executive director

Organization: Batten Disease Support & Research Association

Disease focus: Batten disease, all 14 forms of it. It is one of about 50 lysosomal storage diseases. It’s primarily a pediatric neurological disease related to genetic mutations, autosomal recessive, and it’s ultra-rare. There are varying features of the disease, but in time, all children will experience seizures, vision loss, cognitive decline, and at end of their lives, are typically wheelchair-bound and die way too soon.

Headquarters: Columbus, Ohio

How did you become involved in rare disease: This is the first position I’ve had working in rare disease. A friend of mine saw the posting for this position more than five years ago and sent it to me and said, “You ought to do this job. You’d be perfect.” I wasn’t convinced that I would be, but I applied, and six interviews later the board asked me to join them. It was one of the best career decisions I ever made.

Previous career: I was working for the Ohio Attorney General in the grants administration area. I was responsible for bringing in funding for victim services, human trafficking programs, and opioid abuse in the state.

Education: B.S. from Ohio State, MSW from the University of Kentucky, and Ph.D. from University of Chicago all in social work.

The Organization
Organization’s mandate: Fund and facilitate research, support all families through the life cycle of the disease, and advocate for them at the federal level.

Organization’s strategy: Always building relationships and looking for opportunities to partner, and staying on top of the latest research that can help our families.

Funding strategy: What we do is tried and true. It’s build relationships, look for opportunities to partner, make the ask, and steward the gift to make sure you show people that what you do has value. It’s a tried and true funding cycle. It’s arduous, and rewarding, and important. We want to invite people into a larger vision. That’s really what we are trying to do to—to show them how their hard-earned money can make a difference to very vulnerable children.

What’s changing at your organization in the next year: In 2017 we went through a strategic planning process that was incredibly valuable. At the same time, we are working with Fundraising Council. Those two consultant groups have helped us focus on some things we really think are going to be valuable in the next year. We’re going to be doing a lot of convening, a lot of asking of questions across our stakeholder groups about what’s important, what’s on the horizon for research. How families and children can be more involved in the research process by voicing what’s important to them. Working much more steadily and in close contact with the NIH, funding opportunities with the FDA, continue working with industry. We’re very excited that 2017 brought us our very first FDA approved drug for one form of Batten disease. It was only one form, but it was a very important milestone.

Management Style
Management philosophy: I would say, in terms of the organization, there are four full-time people. I’ve hired some incredibly smart, funny, young women in the office, who are very talented and committed. I start there with a high standard. You’ve got to be smart, and you have to have a sense of humor. That’s the kind of people I like to work with. Then I make sure they have the tools they need, and the encouragement and support to use them. It’s not just hiring people. You have to create an environment where they feel they can ask questions, use their imagination to develop new ideas and new ways of doing things, and engage our families. I think we do it pretty well.

Guiding principles for running an effective organization: Highly ethical and transparent nonprofit management. There’s nothing magically from these ideas. The magic comes because we have a firm underpinning that allows us to engage. When everything at the office feels firm and in place, then you can deal with any chaos that’s happening, new ideas and opportunities, and all of that. But you have to have your house in order.

Best way to keep your organization relevant: Continue to ask what matters of all our stakeholders—families, researchers, our clinicians. We are constantly out there in the world of rare. We’re going to meetings. We’re exhibiting at conferences. We are talking to people and continuing to envision what’s next. That’s how we find out—by being out there.  

Why people like working for you: I think I’m generous. I’d like to be more generous about what we pay them, but we are a nonprofit. I think I’m generous in spirit to their requests, to their ideas, and what they want to accomplish. I encourage their personal growth. I like to have as much fun as I can at work with them.

Mentor: I am a mosaic of a lot of people. Family members, professors, and bosses. I’ve been fortunate to have a number of really fine supervisors in my past. I feel I’m a mixture of all of my past experiences. Currently, I feel like I’m mentored by my peers. I have several groups of nonprofit directors I talk to on a regular basis to share ideas and get encouragement. I love what I do, but this is hard work. You have to be a nonprofit expert. You have to be somewhat of a research expert. You have to be a management expert. You have to understand how government systems work, how drug development works. This is a varied group of skill you have to have. Then, in the midst of all of this, you are working with families that are often going through the worst period of their lives. Children are being diagnosed with a devastating disease. We are deeply affected by what’s happening to our families. It’s very tough to go to a funeral of a 12-year-old, and we do it pretty frequently. In a nutshell, I have my people I talk to all the time for encouragement.

On the Job
What inspires you: People who are often going through the worst time as they help their children and they remain optimistic. And our bereaved parents continue to fund us year after year after year. They believe that there’s something brighter our there for kids. It’s just amazing how many people are still in touch with us year after year after their children are gone. I’m very inspired by my staff too. These young women are very special, and I’m very proud of them.

What makes you hopeful: Science. Science. To go to some of our research conference and see the dedication of these scientists and all they are working on is thrilling. That makes me very hopeful. And I have to say humankind. We are constantly surprised by the kindness of strangers and our community. There are some amazing stories of people helping people out there.

Best organization decision: Our organization is 30 years old. I’ve been at the helm five-and-a-half of those years. So many great ideas were already in the works before I came on the scene. There was an ethos built along the way, and at a very early stage, about families stepping up to the plate and participating in research either as funders, providing blood and tissues samples, and making some very difficult decisions at end of life to donate brains to a brain bank, which has been very important to us. Every year at our conference, families will line up and say, “What do you need us to do this year? Do you need blood samples? Do you need swabs? Do you need questionnaires? How can we be helpful? It’s a constant cycle of giving back into that research. Sometimes we are the envy of other groups. Sometimes I’ll get calls and they’ll say how did you do that? For me, it’s a regular thing. It’s this 30-year legacy that continues.

Hardest lesson learned: There are hard lessons every day. I think one of the things I wished we all had done much earlier is just accept the idea that we weren’t going to solve this quickly. It’s hard to develop endowments and invest in long-standing and expensive test systems when you think you are going to solve this quickly. We are 30 years down the road. Things are looking much brighter on the horizon, but the truth is we are a long way away from a cure. We continue to invest in sustainability so we can be productive for families in the future. I’m not convinced that we’ve done that as well as we could have. I always tell members of new organizations, those that are one or two years old and think they haven’t done enough yet. Thirty years ago, our families were asking the same questions. I always say, “Keep doing it. Keep investing. Keep moving forward.”

Toughest organization decision: I’ve had to let people go in the past and that is always heart-wrenching for me, but the families and their mission is what is most important.

Biggest missed opportunity: We all miss opportunities. You have to prioritize. You can’t do everything all the time. Our antidote is to stay true to our mission. Look for as many good opportunities

Like best about the job: I’m really into job variety. This is a really good job for me. I’m kind of a border collie when it comes to work. I truly enjoy being helpful to people and doing something that makes a difference. I enjoy that. I’m very active in the job.

Like least about the job: The hardest part about the organizational/family part is having to realize next week, and the next week, we will continue to talk to new families that are stunned and terrified about the diagnosis they just received. We want to talk to them because we don’t want them to feel isolated. We’re just sorry that it’s happening. It’s an existential sorry. The other thing I don’t like is the time away from my family and parts of my community. I’m on the road a lot and travel all over the world. Because the populations of these families are so spread out and conferences so spread out, we are in planes a lot. It takes away from times away from my family and parts of my community that are important to me.

Pet peeve: O’Hare Airport cancellations. When you want to be somewhere, and you’ve taken a week away from work, and your flight is cancelled, it’s not good.

First choice for a new career: There are days when I would tell you that my next career—I have this title in my head—chief officer of encouragement in an urban high school. I don’t want to walk the halls, I don’t want to teach, I don’t want to be a principal. I just want these kids to move on, get a good education, and do well. I live in a neighborhood close to some great urban high schools. I see these kids walk to school every day. I wave and say, “How are you doing?” I love that. I get my five minutes with them. They tell me what’s going on in school, and I say, “Hang in there and keep going.” That really is it, but I’d like to be paid exceeding well to do that in retirement. That would be a perfect job.

Personal Taste
Most influential book: Mountains Beyond Mountains by Tracey Kidder, a book about Partners in Health and Paul Farmer
Favorite movie: It’s a tie between The Bourne Identity and You’ve got Mail. It depends on your mood.
Favorite music: Classic rock and roll—Beatles, Rolling Stones, The Who, The Clash.
Favorite food: Italian
Guilty pleasure: Playing hooky and going to the movies
Favorite way to spend free time: With my husband and my dog. I’m thrilled to be married to such a nice man for 23 years.

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