Science is hard, but rare disease research and development (R&D) stands apart. Because individual patient populations are small and many rare diseases are not well understood, research in this space can often bring added complexity. Mary Kunjappu is a Medical Director in our Medical Affairs team in the US. We spoke with her about the importance of following the science, expecting the unexpected, and the endless possibilities in rare disease research.
How did you get involved in medical affairs?
I'm a bench scientist by training, and towards the end of my PhD, I got very interested in clinical research and the potential for things at the bench to have therapeutic effects. And after graduate school, I started working in medical communications because I was very passionate about talking about the science itself. I started working in rare diseases…and I got the opportunity to come in-house with Alexion. I’ve been here for almost seven years now.
What led you to a job in rare disease, specifically?
[When] I started working in rare diseases, I really fell in love with the impact it can make and the work that we do for patients and families...Because many rare diseases are not well known, we get an opportunity to start educating healthcare providers and really, at the early stages, defining different aspects of the disease and what are the symptoms.
What about your work excites you?
I think it gets back to the impact we can make in rare disease…where there is often nothing previously existing to help some of these patients, and we’re able to fill that gap. It’s a very unique position to be in.
What’s one lesson you’ve learned from working in rare disease R&D?
It’s all about following the science, even if it takes you to unexpected places. You need to be able to say, “this is where this is leading us, this is what we're learning about this, and the best way for us to understand this molecule,” and be open to making a change.
I’ve learned to really focus on what the information is, and what it is telling us, rather than having preconceived notions of where things should be, and really tailoring and figuring out what the next step should be.
Do you have any advice for someone considering a career in rare disease R&D?
There’re so many new things happening in science that if it doesn't exist today, it may exist tomorrow. Whatever different combination of interests you are thinking about is probably going to happen in the future. Don't limit yourself to what is possible today.