Fostering Change: A Scientist's Path to Impact in Rare Disease Research

Posted by Megan Cala Pane on Feb 29, 2024 6:00:00 AM

IMG_9769I joined Metrum Research Group in the Fall of 2022 as a research scientist in PKPD, driven by a passion for modeling and simulation analyses of clinical trial data. My journey into this field was profoundly shaped by my experiences during a post-doc at a nonprofit organization, where I had the chance to collaborate closely with patient advocacy groups in the rare disease community. Their unwavering determination and resilience have inspired me to continue my work towards finding cures and creating innovative drug development solutions. In honor of Rare Disease Day, I am grateful to have the opportunity to share my journey of fruitful collaboration that has shaped my perspective.

Individual rare diseases affect a small percentage of the population, but collectively, they affect roughly 1 in 10 people worldwide. Rare diseases usually have few or no treatment options and often no cure. These diseases can be overlooked due to their rarity, leaving patients and their families feeling isolated and desperate for answers. It is within this community that I have found a great source of inspiration and motivation. 

Prior to joining MetrumRG, I worked on an initiative that was striving to provide a centralized and standardized infrastructure to support and accelerate rare disease characterization. I had the opportunity to witness collaborations between patient advocacy organizations, regulatory agencies, and clinical disease experts. A valuable lesson I learned from those interactions was the importance of the patient’s voice and how meaningful progress can begin only after understanding the impact of the disease on the people who have it. Those interactions also solidified my belief that collaboration lies at the heart of significant advancement in drug development research. Pooling knowledge from cross-disciplinary teams can concentrate efforts toward the unmet needs of patients.

In drug development research, the power of modeling and simulation cannot be overstated. Through advanced computational techniques, we can create virtual representations of biological systems (models), allowing us to simulate the effects of therapeutics. The models enable us to optimize dosing, safety, and efficacy and inform clinical trial design decisions. Individual patient-level data are at the core of these models, providing a direct link for the patient’s perspective to be considered during their development. 

With a desire to strengthen my technical skills as a means of generating a greater impact as a research scientist, I feel very fortunate to now be working at MetrumRG where I have the privilege to do just that. In my short tenure at MetrumRG so far, I have already been able to work on a few projects in the rare disease space and I find it truly gratifying being able to contribute to an area with such great need, even if it’s only moving the needle. 

Rare Disease Day is an important reminder of the power of collaboration and a call to action for the broader scientific research community to continue to work together to improve the lives of individuals impacted by rare disease.

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