Drawing on her own experience of being a teacher, a parent and a rare disease mother, Sarah Krieger, Director of community engagement at Cure Rare Disease tells us how she views her role as the bridge between patients who are feeling isolated and scared and whatever that family needs to thrive.

“In my experience, nothing in the rare disease space is easy. Things worth doing, however, rarely are.”

PEO series: meeting the beating hearts behind the RARE brands

1.

How new is the patient engagement role in your organisation, how has it evolved and what are your hopes for the role in the future? 

I have worked as the director of community engagement at Cure Rare Disease for a year and a half. Cure Rare Disease is a Boston, Massachusetts-based non-profit transforming possibilities for people with rare diseases by developing customised therapeutics to save lives.

My role, and the demands of this position, have evolved in many different ways over the past 18 months, and continue to change as Cure Rare Disease grows. When I started, we were only interacting with a few families in the space, and it was necessary to figure out what our role with families was going to look like moving forward. As we’ve grown and now communicate with more families, we’ve really developed our ability to direct and support them within our goals as a non-profit organisation. My goal for this role in the future is to continue to educate families about our approach to drug development, in unique and honest ways, while finding the best approaches to involve and educate them in the process. I want to always improve our infrastructure to continually help our families understand, in real time, where their loved one is in the research and development process, but also assist in scaling and growing the personalised gene therapy programme as a whole.

2.

What does a typical week entail for you, and are there any specific projects you can tell us about?

Currently, I spend time responding to families who want to learn about Cure Rare Disease, as well as those with new diagnoses who simply need help navigating the world of rare disease and the options available, often specifically to Duchenne. Although the diagnoses can be the same, these conversations always look very different as people approach the diagnosis of their child very differently. Ultimately my intent is to provide families with an empathetic ear, answer any questions they may have, connect them with someone that can answer the questions that I cannot, and help support them in whatever decision they deem best for their child and family.

I also work with families once they decide to pursue a customised therapeutic for their loved one. This involves a lot of coordination; getting various documents read and signed, scheduling bloodwork and having it sent to the correct locations, sharing the results of blood work with families, scheduling a muscle biopsy and having the specimen shipped to the correct location, and communicating with families about what they can expect next in the process of developing a customised therapeutic. I also help families’ fundraising efforts, providing them with materials, ideas or general support they may need to run a successful fundraiser in their community.

An upcoming project I am excited about is a lab tour we are offering for many of our involved families. My children and I were able to visit the lab at Yale last year and have lunch with the scientists and researchers of our lab this year and it was quite impactful. Not necessarily because the lab itself was so state-of-the-art, but because often the idea of science and research seem more like a futuristic thing happening in a faraway place with high walls, locked doors and ‘smoke’ coming out of beakers. It was so cool to personally step foot in the lab, not see any proverbial smoke, but see the actual faces of the researchers who are doing such important work, and to personally ask them questions about what a certain machine does or where materials are stored and what they are used for. We are excited to offer families the same opportunity, to meet with the people that are working on life-changing therapies for their loved ones and educate them on what it takes to develop a customised therapeutic.

3.

What were your personal motivations to taking up a role in patient engagement?

This is an easy one. I have a son who lives with Duchenne. He is almost six, but was diagnosed at just 10 months. Because he was diagnosed so young my husband and I had quite a bit of time to explore and learn about the space before his needs became greater. I met with a variety of patient engagement types, never with the intent of becoming one myself, but as the person on the other side. I was (and still am) a parent trying to figure out what this diagnosis means and what to do next. Naturally, I started to realise what was and was not helpful for me as I navigated life as a rare disease parent. There were things I appreciated, but also things I felt were missing, in addition to ideas I thought could be helpful based on my own experiences. These personal experiences have laid the foundation for how I approach my role as director of community engagement at Cure Rare Disease.

4.

What makes the role of patient engagement officer important to your organisation?

I like to view my role as a bridge. Often a rare disease diagnosis leaves one feeling alone and isolated; there are so many decisions to make, yet one can feel stranded when making those decisions. My job is to connect the patient and their family to whatever it is they need, because no one should feel alone when navigating rare disease. Sometimes, it’s connecting a newly diagnosed family with a clinician, sometimes connecting one family with another that have kids the same age, or a family who is starting the process of building a wheelchair-friendly home with a family that already has done that, or it might be connecting a family in our development process with the researchers who are working on a therapy for their loved one.

5.

In your role, how do you ensure the patient voice remains central?

I think this comes naturally for me because I was a patient (or mother of a patient) long before I took this role. My motives for stepping into this role were rooted in my belief that patient voices should be at the forefront of drug development. I was frustrated in feeling this was not always true, and so instead of just accepting and complaining, I felt accepting this role was my chance to bring some of the change I had wanted to see in the world of rare disease. I am in the trenches with so many of these parents and by having a son with Duchenne, making the patient voice a priority is a relatable and natural practice for me.

At Cure Rare Disease, we are also constantly asking for feedback. Our approach is a very new one, so checking in with families on what they need and learning what is and is not helpful via surveys and conversations is a daily part of our work.

6.

How do you reconcile operational business needs with elevating the patient voice?

The patient voice triggered the start of Cure Rare Disease. It is the foundation and inspiration for the work we are doing. As a non-profit organisation, we exist to provide a chance at life-changing therapies to those who otherwise would be forgotten. We would be doing a disservice to the rare disease community if the patient was not at the forefront of all we do. Since we are a non-profit directly controlling the drug development process from bench to bedside, the patient voice is naturally elevated and the focal point.

7.

What are the most rewarding aspects of your role?

I love learning, and often my job is best done when I am listening. Being able to provide people with a safe place to share their rare disease story and often comforting them through their fears and challenges isn’t always easy, but is so needed. The world of rare disease is very diverse, and I continue to be inspired by all the parents and families I meet that are doing all they can to bring change to the current prognosis of varying diseases. It’s always rewarding to know that no one is alone in this fight, and we all desire and are working toward one thing – to bring life-saving treatment to the ones we love.

8.

What is your proudest moment in your career thus far?

I am incredibly proud of Cure Rare Disease’s work to continually tweak infrastructure that not only helps families understand in real time where their loved one is in the research and development process, but also assists in scaling and growing the personalised gene therapy programme. Customised therapies are a new and innovative way to approach drug development, and I am proud of the ways we at Cure Rare Disease have collectively worked to bring the opportunity of life-changing therapeutics to those who previously did not have any treatments available.

9.

What advice would you give someone considering working in the rare disease space?

In my experience, nothing in the rare disease space is easy. Things worth doing, however, rarely are. The ability to listen to, learn from and empathise with people is key. The rare disease community is filled with passionate people doing meaningful work. But working in the rare disease space is more than just that – it’s an investment in people who are fighting to simply have the same opportunities as those who do not find themselves with a rare disease diagnosis. It’s not always easy, but so worth it considering the difference the work makes!

10.

If you weren’t Patient Engagement Officer of [insert company], what was Plan B?  What did your 10-year-old self want to do as a job?

In what I like to refer to as my ‘former life’, before I worked as a PEO and even before I was a mom, I was a teacher. I very much enjoyed breaking down new and difficult concepts for students in a creative way for them to not just understand, but apply. It is always surprising to me how many aspects of my teaching career directly relate to what I do in my current role as director of community engagement for Cure Rare Disease. Breaking down often difficult science in approachable ways for families is something I very much enjoy.