Rare Leader: Joslyn Crowe, Executive Director, National Niemann-Pick Disease Foundation

The Basics
Name: Joslyn Crowe

Title: Executive director

Organization: National Niemann-Pick Disease Foundation

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Disease focus: Niemann-Pick disease is a rare genetic metabolic condition that impedes the ability to breakdown, transport, and use fats and cholesterol in the body. As a result, lipids accumulate in various organs and cause damage over time. There is currently no effective treatments for this condition and those who are affected generally do not survive past early childhood.  

Headquarters: Fort Atkinson, Wisconsin

How did you become involved in rare disease: I was working with Pfizer for many years on blockbusters and across all therapeutic areas. They were launching a product for a rare disease, which was Gaucher disease. And they were the third to market in this space, but they needed some internal and external stakeholder education because 50 percent of the Gaucher patient population was Ashkenazi Jewish. They needed to reach out to that community and to understand it. They brought me in to work with Pfizer’s executive leadership and to work with the national leaders of Jewish community organizations throughout the U.S. to talk about Gaucher disease, to talk about Pfizer, and to talk about this product. It was the first experience I had in rare disease, and it opened my eyes to the possibilities of when a large company invests resources into small patient populations and how great the outcomes could be. After that, my interest was piqued and I started following what was happening in the rare disease world and doing a lot of reading. I was speaking with a colleague who worked at a different pharmaceutical company and who was on the board of the NNPDF and she let me know that they were looking for an executive director. All the pieces came together. And that’s how I got involved with Niemann-Pick and rare diseases.

Previous career: Patient and professional relations for pharmaceutical companies

Education: B.A. in clinical psychology from Tufts University, MA in non-profit management from Hebrew Union College, and an MSW from the University of Southern California

The Organization
Organization’s mandate: We’re the leaders for Niemann-Pick advocacy and family services. We also offer research in a meaningful way, through fellowships for early stage investigators. One of the strategies that we use is to weave advocacy into all of our work and all of our programs so that we’re continually supporting the community for where they’re at in the present, but also looking forward for what will be needed down the road.

Organization’s strategy: Our strategy is to continually stay on the pulse of the patient community. We look  at where we are now, which is a place of great importance in terms of advocacy for therapies and advancing therapies through the pipeline, working with regulators and key opinion leaders, but also maintaining that focus on families, because while there are other organizations in the Niemann-Pick world who are raising money for research, no one else is working with families on their day-to-day challenges. When we look at our strategy, it’s just staying in line with what’s meaningful to families, what’s needed for families, and delivering programs that meet those goals from the day to day challenges to the bigger picture of bringing medicines to market.

Funding strategy: Our funding strategy is a mix of corporate and industry and grassroots. We’ve been fortunate to have continued support from many of the industry partners in our space, which is essential because grassroots fundraising is challenging. We’re an ultra-rare disease. It’s a small population. And there are other organizations contributing towards research. We’re all looking for shared dollars, but our realities, which is similar to many rare disease communities, is that Niemann-Pick disease is a severe disease. It’s a neurodegenerative disease and the consequences of that are that oftentimes one family member needs to stop working to become a full-time caregiver and our families are stretched to capacity. We do have a grassroots strategy and we do viral fundraising. We have two major campaigns a year that are direct mail. We do a little fundraising through our annual conference, though the goals of our conference are not predominantly fundraising from families in any way. We’re fortunate, again, to have good relationships with industry in our space.

What’s changing at your organization in the next year: Hopefully, we will be in an environment where we’ll have an approved therapy for Niemann-Pick type C and a standard of care. While we’ll still need to encourage research and search for a cure and encourage other development in the space, what will be changing is the way that our families engage and what will happen once they receive a diagnosis. For our ASMD families (Neimann-Pick type A and type B), we’re still progressing there, and there’s still lots of regulatory work to do down the road. Given the space that we’re in, next year we will focus on access and payment for future therapies. One area, which will be even more important in the coming years, is our focus on newborn screenings. We are doing work with both NPC and ASMD to work towards adding both of these types of Niemann-Pick disease to the screening, which could lead to earlier diagnosis and earlier treatment decisions, which would be incredibly beneficial to our families.

Management Style
Management philosophy: I lead by example. I don’t ask my staff or my team to do something that I wouldn’t do or that I wouldn’t want to do. It’s important to be deliberate and to be think things through. In nonprofits, especially volunteer-based organizations like ours, it’s important to nurture your volunteers, to be respectful of their time, and to be in tune with their skill sets, so we are working with volunteers and families in ways that are engaging and inspiring, but also maximizing what talents they can share with the organization.

Guiding principles for running an effective organization: Keep forward focused, not looking backwards. Look at where we want to be in the next year, two years, in five years, pivoting as needed along the way, but keeping your eyes on the big picture.

Best way to keep your organization relevant: It’s to show up. It’s to be present. Say “Yes,” when speaking opportunities come up. Say “Yes” to participating, to collaborating. Pay attention to other rare organizations, collaborate with organizations in your disease space, and check in with your members and your community to make sure that the content that you’re delivering is what’s needed. One of our strategies says, deliver content, deliver programs, but if you’re not delivering programs and content that the community needs, then there’s no purpose.

Why people like working with you: People like working with me because I am committed. I’m very committed to the community. I’m committed to following things through to the end, and to making sure that we do have treatments for families. Families find that my team and I are accessible and my professional colleagues find that I do what I say and will step up and be present anytime that is needed.

Mentor: I don’t know that I have a mentor right now. I think that I’ve had role models in my professional development and professional life, especially from earlier in my career, who have been examples of behaviors or ways to position myself that I’d like to emulate. I did have a mentor in my previous career in pharma, but unfortunately that outcome was a little disappointing and turned out to be a disingenuous relationship. At the moment, I’m more focused on just looking at some of the women and men who have inspired me along the way.

On the Job
What inspires you: The families in NNPDF are incredibly inspirational. The stories and challenges that I hear weekly make me feel fortunate to have a healthy family and to be in a position to help. The biggest inspiration is hearing from families who are newly diagnosed or in the Niemann-Pick journey, or even families who’ve lost loved ones and are still very engaged and want to continue to tell their story and to help others and to lead the organization, to make sure that there is a strong, solid foundation for the future.

What makes you hopeful: We’re in a very hopeful space right now. We’ve come from a community with no hope of treatment where families were being told to go home and enjoy your child or your loved ones, to now being in a position that’s got a lot of interest from industry and the possibility of approvals on the near horizon. I’m hopeful that the face of Niemann-Pick disease will change in the coming years. While we won’t have a cure, families will be in a position when they receive a diagnosis, as awful as Niemann-Pick disease or as awful as any devastating, rare disease is, to know that now there are possibilities and hopefully we’ll have multiple treatment options so that families will be able to use combination therapy, or find something that works to lessen symptom impact and extend life.

Best organization decision: Rebranding was one of our best decisions since I’ve come on board. When I came on board, the organization had just been through a challenging time. It was looking to professionalize and step up in terms of leading the community and step up its presence. Rebranding allowed NNPDF to have a new look, and it was a fresh start and a reset. Our new logo had a lot of thought put in. We were able to continue the hopeful nature of the original design, but really show that we are now in a new phase. And we were in a new phase in Niemann-Pick leadership and in rare disease leadership. Carrying that through to everything that we have visually made a big change. We put a lot of time into our communications. We have a lot of external communications with information going out throughout the week through a variety of sources. Everything’s tied into this new brand. It’s very discernible as far as where information is coming from. As the national patient organization, families know that when they see our materials, they’re being given information that is up to date, accurate, and reliable.

Hardest lesson learned: Community-wise, the hardest lesson probably was learning that everyone needs to work together and not be fractured—not encourage drama and encourage interpersonal or organizational distrust and challenges. I came in following a challenging period in our community. That was a real lesson for everyone involved. Not working together sets everyone back. Looking for the big picture and a united community is the best front you can have when speaking with regulators, or to key opinion leaders. Researchers and industry people want it. Everyone wants to see that the community is working together and that the steps forward are going to be possible rather than having a big distraction from community drama. Ultimately, unity is what’s best for patients and for families.

Toughest organization decision: Reducing our scientific advisory board to focus only on the U.S. was a tough, but deliberate decision that we made. We had a scientific advisory board that was large. It was probably 20 professionals, but it was the creme de la creme of the Niemann-Pick space. We had Niemann-Pick disease pioneers and experts from all around the world. But in terms of our new focus, what became important was to have our focus just be on the U.S. and to have our board members just be from the U.S. It allowed us to be more effective and more efficient. Moving some of these amazing international superstars in this space to emeritus status was a tough decision, but one that has paid off.

Biggest missed opportunity: The biggest missed opportunity is right now. It’s the lack of in-person dialogue due to travel restrictions and COVID. While virtual conferences have allowed us to deliver content, we can’t replicate the in-person experience a hundred percent. For our conference this year, we doubled the attendance that we typically had in the past. It was great for keeping the community informed with an update on current events and what’s happening. We had a lot of programs that allowed for some connections, but it’s impossible to replicate that feeling that families get when they get to be together on site. Some of our families don’t see other Niemann-Pick patients at all during the year. What we hear a lot is that it’s the only place that our families can go, that everyone else just gets it, and they don’t have to explain, and everyone just understands, and they can relax and be themselves and let their guard down. That, for me, is one of the missed opportunities. And then in terms of relationships with industry, it’s challenging not being there in person at conferences, because a lot of the work that goes on at these events are the meetings you have on the side with your industry partners. It’s when you get to share your updates and talk about your plan and build those relationships and talk about funding. This year has been a missed opportunity for all of that.

Like best about the job: I’ve got a dedicated team and staff and board members. It’s nice to work with people who are so passionate about everything that they do.

Like least about the job: It would be great if we were fighting a different battle. It would be great if we had treatments and we were now saying, “Now we need a cure.” It is emotionally challenging to face the reality every day, that new families are being diagnosed and that some of our family members will pass during the year. Everyone’s trying their best, but there are still patients who are going to degenerate and who next time you see them their disease will have progressed. And that’s hard.

Pet peeve: Small talk that goes on at the beginning of meetings. I want to get right to it.

First choice for a new career: I would be a diplomat or a travel photographer.

Personal Taste
Most influential book: The Wal-Mart Effect: How the World’s Most Powerful Company Really Works–and How It’s Transforming the American Economy by Charles Fishman

Favorite movie: Love Actually, The Holiday, and The Talented Mr. Ripley

Favorite music: I like pop music

Favorite food: Publicly I say French food, Thai, or something with a lot of flavors. In truth my real favorite is thin crust pizza.

Guilty pleasure: House Hunters International

Favorite way to spend free time: Tennis. I just started playing golf. I like to try to get my kids outside, but a lot of times we will end up watching movies together. And in the winter, I like to ski.