Know your value. — There are many nonprofits out there — thousands in fact. Even within the same disease there can be many nonprofits pursuing a similar mission. It’s important to not be a copycat organization and understand what unique value you are trying to introduce into that world; we all have a role to play. Cure Rare Disease clearly defined our niche: developing tailor-made therapeutics for rare genetic diseases. To my knowledge, we are one of the only nonprofit organizations in this space, so we are able to move it forward in a targeted way. Equally important is understanding what is outside of your mission; mission creep is very real and can threaten to derail your organization as you grow. Make sure to know where to draw the line. (And it’s okay if it changes as long as it makes sense as related to the overall mission!)
As part of my series about “individuals and organizations making an important social impact”, I had the pleasure of interviewing Richard Horgan.
Rich Horgan is the Founder and President of Cure Rare Disease. He has a deep passion for Duchenne muscular dystrophy (DMD) and other rare diseases. With a younger brother impacted by DMD, Rich has a strong interest in accelerating promising treatments for the disease. He has formed a collaboration with world-class researchers and clinicians to pioneer the rapid development of customized therapies for Duchenne and other rare diseases. Rich holds a BS from Cornell University where he graduated summa cum laude, and an MBA from Harvard Business School where he was awarded the Blavatnik Fellowship for Life Science Entrepreneurship.
Thank you so much for doing this with us. Before we begin our readers would like to get to know you a bit more. Can you tell us a bit about your “backstory”?
Of course. My story starts in a small town in upstate NY where I was born and raised. My family owned a small convenience store and my younger brother, Terry, and I were heavily involved in my family’s business growing up — mostly stocking shelves and helping out during the summer. It was a relatively conventional upbringing in a tight family, but for the fifth presence in our house: Duchenne muscular dystrophy (DMD). My younger brother, Terry, was diagnosed with the disease when he was three years old, and that would eventually be the catalyst for Cure Rare Disease. DMD is a cruel disease, progressively robbing boys of the muscle needed to walk, breath and live. The diagnosis of a fatal rare disease like DMD usually lands with desperation: families like my own are told the same, sad, canned line: There is no treatment or cure; go home and love your kid for as much time as the disease allows. Most boys and young men succumb to DMD in their twenties, despite the seemingly limitless potential of modern science, faced with limited to no options due to the slow pace of change.
The gambit of emotions shared by family members — uncertainty, fear, anger, sadness — evolve over the years, underpinned in large measure with the desire to do something, anything, to change the fatal outcome that currently colors DMD. For me, the entrepreneurial spirit that grew through my family’s small business brought me to Harvard Business School, where I found that “something” I could do, in the fall of 2016.
Can you tell us the story behind why you decided to start your nonprofit?
My education in entrepreneurism at HBS began to make my “impossible dreams” for Terry suddenly feel like realistic goals. After a particularly tough fall that Terry, 23 years old at the time, suffered, I told myself I wouldn’t let Terry be another victim to this faceless killer. I had no idea what I was going to do or what would come of it, but fear and urgency are good fuel for risk-taking. Immediately, I began googling all the relevant DMD researchers, systematically reaching out to them in hopes of starting a fruitful conversation. Fortunately, that HBS email address was a key conversation-opener with researchers from Boston, Canada, Australia and beyond. What I lacked in science knowledge was supplemented by business training, which pushed me to understand as much as possible about the research and development ecosystem — what researchers were doing what, which companies were focused on which technology, etc. The next year was spent immersed in an unfamiliar environment (think semester abroad). As my network grew, so too did my understanding of the ecosystem. Unfortunately, that understanding came with the stark realization that nothing in the drug development ecosystem — from preclinical research to clinical trials — was going to help my brother in time.
And yet: Determined efforts frequently yield serendipitous leads. A chance encounter led to Dr. Timothy Yu, whose groundbreaking work already underway at Boston Children’s targeted a tailor-made treatment for a little girl suffering from a highly progressive and fatal disease called Batten disease. The idea of developing a drug for one person seemed like a fairytale — but Dr. Yu had the story figured out. He successfully dosed his patient, and through these conversations I came to understand the potential to rapidly create and test a drug designed for one person. My hopes grew. Rather than the traditional “10 years and 2 billion dollars” trajectory, a more reasonable timeline — 2–3 years, 2 million dollars investment — emerged. Since Tim is not a Duchenne-specific researcher, I formed my own team of cross-functional collaborators — researchers, clinicians, manufacturing experts and regulatory guidance — all focused around building and advancing a therapeutic to ameliorate Terry’s unique genetic mutation. And with that, Cure Rare Disease was born.
Can you describe how you or your organization aims to make a significant social impact?
Cure Rare Disease was born purely from desperate necessity to save people impacted by diseases too rare to attract traditional drug development efforts (read: investment). What we are building is an ecosystem, with drug development at its core, supported by insurance coverage. We want to democratize this process to all families who are impacted by a rare, genetic disease with no treatment options.
We are a nonprofit and measure our success by the number of patients treated. Within that model, we partner with our families to raise awareness and support to enable our mission. Our researchers, clinicians, patients/patient families and supporters all play collective roles in enabling our shared vision to be made real (finally).
Without saying any names, can you share a story about an individual who was helped by your idea so far?
Our website and social media feeds feature the many faces who could be helped by our work. These individuals come from a wide range of backgrounds and experiences, brought together by shared hope for people we love. We are on track to dose my brother as our first patient later this year, and behind him are two other patients with tailor-made medicines in waiting, as our vision moves closer to reality. Cure Rare Disease is a relatively young organization still attempting to prove our model, so we are currently limited by the number of people we can help — but our imaginations are limitless. We hope and believe that as we prove ourselves in these early days, new opportunities will emerge and let us continue to expand the scale of our mission so no family ever again has to fear a rare disease diagnosis. It will take time to get to that point, but we have the team necessary to get us to that point.
Are there three things the community/society/politicians can do to help you address the root of the problem you are trying to solve?
Yes! Awareness is one of the most important facets of our job. Some believe that if a disease is rare, few people are impacted. While that is true for some rare diseases, others are more common than you’d think, ALS and Muscular dystrophy among them. All told, rare diseases impact more than 30,000,000 Americans — that’s 10% of the entire population! That’s why it’s important that communities and policymakers alike understand the challenges that people and families impacted by rare disease face. The emotional, economic and physical costs associated with these diseases is very real and support is limited within our current systems. Education is important to elicit change.
That leads me to my second message for politicians and the voting American public: We need to reimagine how insurance reimburses life-saving therapeutics. The status quo? Insurance companies, both private and public, will only reimburse a drug that has been shown to be effective in a large group of patients before deciding to pay for it. This model simply does not work for rare and ultra-rare diseases that require individual solutions. That’s my intent in building an ecosystem: it is here where we need society and politicians to understand that not one reimbursement mechanism will work for every case. Ultimately, the question we seek to collectively answer is, how do we make tailor-made therapeutics sustainable in the long term, by working with insurance companies to get these much needed medicines to patients who otherwise have no hope? In advocating for this cause, I strongly believe that it aligns with economic incentives of insurance companies — if we can deliver lifesaving medicines to patients earlier in life, before significant symptoms have manifested, we can ideally offset or eliminate costs of care that are realized as the patient progresses further into the disease.
Case in point: the need for a bipap or cough assist for muscular dystrophy patients. Currently, my brother’s insurance pays 12,000 dollars a year to lease a cough assist and bipap machine — the purchase price of both of these units is well below this amount, yet insurance will pay magnitudes more for this equipment. I’m no actuary, but that makes no logical sense on its face. This is where we need to reimagine the insurance mechanism to include consideration for rare diseases — and potentially save money in the process for all involved! Doesn’t the combo of improved patient quality of life and reduced costs seem like a win/win?
This will all require communities and politicians to get involved. Rare diseases often impact too few individuals to attract celebrity attention or significant government funding. But that doesn’t mean that the individuals it impacts are less worthy of help than people impacted by well-understood diseases. Rare disease patients hold an incredible potential for societal health as a whole: By learning what can work to fix a disease that impacts 10,000 people, we can leverage that knowledge to tackle far more common diseases. To realize this vision we need people to get involved and champion these causes. I encourage policymakers to think deeply about this potential, and encourage them to consider unique funding vehicles that award grants and support to those teams and researchers who have shown tangible progress in helping these patients.
How do you define “Leadership”? Can you explain what you mean or give an example?
Leadership means providing the voice that continually rallies and inspires the people around you to relentlessly pursue a shared vision until it is a reality. The journey to find and build a highly effective organization while advancing the most groundbreaking science out there, in parallel, has been the most challenging journey of my life. I am certain that it would never have been possible if not for the team that we have built — both on the R&D side, as well as the organizational development side. To me, a great leader finds the best people for the needs and then focuses them in the right direction.
Based on your experience, what are the “4 things a person should know before they decide to start a non profit”. Please share a story or example for each.
There’s a number of lessons I have learned, and continue to learn, throughout this journey.
#1: Know your value.
There are many nonprofits out there — thousands in fact. Even within the same disease there can be many nonprofits pursuing a similar mission. It’s important to not be a copycat organization and understand what unique value you are trying to introduce into that world; we all have a role to play. Cure Rare Disease clearly defined our niche: developing tailor-made therapeutics for rare genetic diseases. To my knowledge, we are one of the only nonprofit organizations in this space, so we are able to move it forward in a targeted way. Equally important is understanding what is outside of your mission; mission creep is very real and can threaten to derail your organization as you grow. Make sure to know where to draw the line. (And it’s okay if it changes as long as it makes sense as related to the overall mission!)
#2: Teamwork: It is the dream.
We are so much stronger together than we are divided. It’s important to be collaborative with synergistic organizations and work together when and where it makes sense. I built CRD almost entirely through partnerships and collaborations. This let me keep the headcount lower (so people management is less of a time commitment) and still be nimble and effective in the efforts we pursue. However, not everyone who claims to be open to collaborating is actually open to collaborate. I find that the term collaboration is thrown around very loosely yet rarely acted upon as it relates to nonprofits. This is a disappointment, but the antidote is to find partners willing to not only talk the talk but actually walk the walk — as the saying goes. Above all, we can collectively do more together than we can individually, proven time and again.
#3: Leave Your “I” at the Door
It’s important to understand that your proverbial village needs to be curated carefully and recognized. I see my job as being primarily centered around finding the best possible talent for the role and putting that talent in a position to win. Whether that means acting as a tackler to clear the path of bureaucracy for researchers, or facilitating one connection to work with another, the job is to let the scientists (or fill in the blank) do their work. And be sure to recognize everyone! Thank you is free but it’s one of the most powerful phrases out there. Don’t be afraid to use it often and make sure to recognize the people who usually don’t get recognized.
#4: Be a Storyteller
This point ties into understanding your value proposition: It’s important to understand how best to communicate with your audience(s). Each audience will react differently to certain messages, and crafting the message that resonates most with your respective audience is critical. For complicated subjects that sometimes means using analogies to bring home concepts. For instance, we compare the virus used to deliver our CRISPR-based therapeutic to a FedEx truck, which helps people understand the complex subject better without a PhD in hand.
We are very blessed that very prominent leaders read this column. Is there a person in the world who you would like to talk to, to share the idea behind your non profit? He or she might just see this, especially if we tag them. 🙂
For many years I’ve followed the work of Dr. Priscilla Chan. Her work with the Chan Zuckerberg Initiative parallels incredibly closely with our mission, to develop tailor-made therapeutics for untreated, rare, genetic diseases. It would be a once in a lifetime opportunity (for so many lives) to have a conversation with her.
Can you share your favorite “Life Lesson” Quote? How is that relevant to you in your life?
The quote that has always resonated deeply with me, for obvious reasons, is one by Margaret Mead: “Never doubt that a small group of thoughtful committed individuals can change the world. In fact, it’s the only thing that ever has.”
How can our readers follow you online?
To learn more, readers can go to cureraredisease.org and also follow us on social media platforms (@cureraredisease) to stay up to date with our progress. We welcome everyone to join the fight — individuals, companies and teams all play critical roles in winning the war against rare disease. We are so close to solutions, so now is a great time to get involved!
This was very meaningful, thank you so much. We wish you only continued success in your mission.