Thriving On Ambiguity: What I've Learned Leading Rare Disease Teams
By Melissa Leichter
We live in a time of hope for millions around the world, as technological advancements and research breakthroughs hold the promise of transformative treatments and cures. And with social media at our fingertips, it’s never been easier to raise awareness for those living with illness and advocate for progress.
In recent years, the rare disease sector has boomed. In 2020, nearly half of all novel drugs approved by the FDA were intended for rare diseases, a significant leap from the 23.5% reported in 2012. The total market size to treat rare diseases around the globe is projected to rise to $255.4 billion by 2028.
But for every research leap and successful social media campaign, many vulnerable people still suffer from diseases so rare that they are largely unknown and overlooked. The fact remains that patients often spend years, sometimes decades, in the dark, grappling with symptoms that confound even the most seasoned physicians. By some estimates, as many as 30% of children with a rare disease don’t live to see their fifth birthday.
For people working in rare disease, the playbook that you find in more well-trodden paths in medicine and pharmaceuticals doesn’t exist. That means professionals in rare need to be comfortable with ambiguity. It’s a bit like being handed a jigsaw puzzle with no box cover to guide you.
But it’s that lack of a consistent roadmap that makes working in rare so interesting—and so rewarding when you’re successful.
Over the course of the last 14 years at Novo Nordisk, I have had the deeply satisfying experience of leading teams to commercialize treatments for people living with rare diseases. I’m proud of what we have accomplished over this period of significant growth for this segment of the pharmaceutical industry.
I use that word “we” very purposefully. One of our executive leaders is fond of quoting Margaret Mead, the anthropologist: “Never doubt that a small group of thoughtful, committed citizens can change the world. Indeed, it is the only thing that ever has.” My team always reminds me of that essential truth.
They bring a wide range of skillsets, but all share some common traits. Here are three lessons I’ve learned on my leadership journey in rare diseases:
Passion trumps all. I’ve learned over time that you can hire based on skills or experience from a resume, but there’s an intangible factor that’s even more crucial: how passionate people are about this work. I know that if a healthcare provider calls about a challenge with supply, I can very quickly rally highly committed team members from various functions across the organization to come together and devise a solution that will get patients the treatments they need.
The shared mission extends beyond my team. A big part of our success hinges on building connections to the rare community we serve. That includes nurses and doctors, pharmacists, individuals working in advocacy groups and, of course, people living with rare diseases and those that care for them. It’s relationships across that entire community that help us collaborate and communicate with one another to meaningfully serve patients and the broader community.
We have to be comfortable working with the unknown. It bears repeating: One of the distinguishing features of working in rare disease is the complexity inherent in the field. Unlike the more data-rich environment in mass-market health conditions, rare diseases require a wider set of capabilities in order to move the needle; namely, puzzle-solving skills and an inordinate amount of patience.
A condition like Glanzmann Thrombasthenia (GT) tells the whole story – why rare is so challenging but why it’s simultaneously incredibly rewarding. This ultra-rare bleeding disorder affects fewer than 300 people in the U.S. You don’t have access to the traditional data you’ll find in the retail drug world. Because of these gaps, we have to generate insights from conversations with numerous different groups.
It’s an outside-in approach that has led to a comprehensive understanding of unmet needs for people living with GT. We have learned that these patients can go years without an accurate diagnosis and can face life altering and life-threatening bleeds. This has led our team to develop diagnostic algorithms and deploy AI with the goal of reducing time to diagnosis.
I have found that rather than scare off great talent, the complexity of rare disease actually resonates with individuals who relish challenges: extraordinary people who blend entrepreneurial spirit and intellectual curiosity with immense empathy for patients.
There’s nothing more gratifying than working with a community of passionate people who want to make a difference for patients who have struggled with a disease for years and need hope that better days are on the horizon.
About The Author:
Melissa Leichter is the Vice President and Commercial Lead, Rare Disease, at Novo Nordisk.