Insights From the 2017 World Orphan Drug Conference

Our rare disease team recently attended the World Orphan Drug Congress and Chris Komelasky, senior director sites and patients, Rare Disease and Pediatric Center of Excellence, shares his thoughts on what he gained from this year’s conference and why working to advance treatments for rare diseases is important to him.

What was your favorite part of the conference this year and what did you learn while attending?

Having taken part in previous World Orphan Drug Congresses, I had a sense of what to expect and was excited to be attending again this year. There are three things that stood out about this conference, and all are anchored to the people and their mission:

• Focus and alignment: The participants, some of the world’s most visible and active experts, share a common understanding of rare diseases and know the importance of working together to achieve the goal of bringing the right medicines to the right patients, at the right time. This laser focus and understanding of the common challenges faced in this area create a unique environment that unifies people from different companies, functions and parts of the rare disease ecosystem. Understanding the importance of working together towards a common goal is powerful.
• Energy and passion: The energy is contagious when you walk into the plenary, the exhibitions and the networking zone. There is a constant buzz that drives people to want to learn more and explore new ideas. There is also an authentic passion that comes through around the patient voice and how valuable a perspective that is throughout the drug development and clinical research process.
• Drive for change: The collective conference group is not one that settles for the status quo, whether the path forward has been charted or not. They will exhaust all channels, explore every possibility and persevere through challenges to push things forward. This group understands that change will only happen when we put our heads together.

Coming in, I was hoping to get a chance to meet some new people who are tackling different aspects of the rare disease challenge, share PPD’s vision for this space and learn how we can partner in different ways to solve some of those challenges. While I enjoyed hearing some of the latest scientific, medical and regulatory advances, in my role targeting the development of strategies for site and patients, meeting some of the data and service providers proved to be the most rewarding. This group is pushing the areas of innovation and enabling new approaches to find, connect and engage with patients. If we as a collective group can constantly improve our planning and delivery, we will have the ability to bend the cost and time curve in the drug development process to better deliver life-changing rare disease therapies.

Tell us about the roundtable that you hosted, “Where in the World is my Patient? Next Generation Strategies for Global Patient Recruitment”.

Every patient, in every study, matters. In rare disease studies, it’s magnified to the nth degree given how few patients are available and how many patients are typically needed for these trials. This dynamic puts tremendous pressure on patient identification, recruitment and retention, which can be the lynchpin to successfully delivering a rare disease trial.

In our roundtable, Horacio Plotkin, M.D., F.A.A.P., Vice President, Medical Lead, Rare Disease and Pediatric Center of Excellence, and I explored the reality of looking throughout the world to find that one patient who will make the difference in a trial. We talked through what it means to be conducting successful studies in the rare disease space and how it differs from traditional, larger patient populations. We focused on the different approaches that can be taken, the data sources and tools used to do this, the challenges seen and ways to mitigate them, and ultimately the regional differences around the world that can affect these approaches.

Our group of table participants brought global representation from patient advocacy groups, big pharmaceutical companies, smaller biotechs, CROs and various service providers. All brought their ideas as we quickly narrowed in to discuss the latest trends and challenges in working with online patient communities and social media as a mechanism to find and connect with patients. It is clear that there is not a single proven channel or approach that will work in all situations, so having a flexible framework, customized to the particular disease, that exhausts every possible channel is the only way we can sustain success in these studies.

We know this is a rapidly evolving space and enjoyed hearing how each stakeholder group can bring a different perspective and valuable contribution to the table.

Why are you passionate about working in the rare disease space, especially in clinical trials?

Every patient has a story and it’s our personal connection to these that helps bring out the passion for the space. Whether it’s a friend, a neighbor down the street, a family member – the realities of those situations make rare diseases very tangible to people. For me, it’s my father. Diagnosed with a rare cancer, I watched for two years as he fought through various treatments, and the pain and all the emotions it brought out. Along that journey, there were some targeted treatments that really helped, which also gave us hope when there wasn’t any otherwise – and that medicine was only possible because of the focus on rare disease drug development. Experiencing that firsthand and being a part of the journey have made my work in the rare disease space have such a deeper meaning.

If you close your eyes and think about living with a rare disease or having your child live with a rare disease, it’s a scary thought. One likely filled with challenges in large part driven by the lack of available treatment options. Participation in clinical trials attacks this head-on. The impact of just one more person in a rare disease trial touches the lives of all those around them, and those following them. The impact can’t be understated.

Chris Komelasky is a senior director sites and patients, Rare Disease and Pediatric Center of Excellence.