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2019 Annual Letter
Dear Community,
2019 was a year of discovery and progress for TREND. Our communities nearly tripled. We forged strong bonds with patients and caregivers, learning not just about the diseases that impact their day-to-day lives, but about their aspirations, hopes, and dreams.
When our child was diagnosed with Prader-Willi syndrome (PWS), we felt so much relief when we found other parents in our position. Becoming part of a community of people facing similar challenges gave us hope when we needed it the most, and it literally changed our child’s life for the better. It was also the moment we realized there were significant gaps in the knowledge of doctors and researchers—and we believed that our community’s shared stories and experiences could fill in the missing pieces.
Drawing upon our expertise in digital health and clinical research, we set out with our team to build innovative technology to turn community conversations into powerful data. In 2017 we launched our flagship Community Voice Reports (CVRs). We are grateful that our success over the last three years has allowed us to gift our CVRs to communities at no cost. We are proud of the breakthroughs our work has already inspired. Here are a few of the most notable:
Together with members of the community, we identified pitolisant as a treatment option for PWS and created a road map to bring this drug to patients in the U.S. The data collected by the caregivers inspired the FDA to approve a novel clinical protocol design. Due to the efforts of brave pioneers and a truly patient-centric company, Harmony Biosciences, one day pitolisant could benefit the entire PWS community—setting a precedent for rare disease therapy development and approval.
In 2018, we partnered with the pituitary tumor community and Cydan, an orphan drug accelerator, to demonstrate the urgent, unmet need for a therapeutic option among people living with non-functioning pituitary adenomas. In 2019, Cydan launched Tiburio Therapeutics, a company that aims to advance novel treatments for serious neuroendocrine disorders.
In collaboration with members of the recurrent pericarditis community, TREND contributed patient perspectives to Kiniksa Pharmaceuticals’ application for breakthrough therapy designation, which the FDA awarded for rilonacept. We applaud Kiniksa for its ongoing commitment to listening to and supporting patients as they await this promising treatment.
This summer we were invited to participate in the Global Skin Rare DERM Forum in Italy where delegates from 13 countries representing rare and uncommon dermatological diseases came together to build a cohesive community. We presented our cross-analysis of two rare skin diseases, Epidermolysis Bullosa (EB) and Chronic Urticaria (CU), to stimulate discussion about overlaps in challenges and identify opportunities for collaboration.
TREND’s new vision is to revolutionize the world’s understanding of diseases. As we scaled our technology to look across rare disease datasets, it became evident that by looking at diseases more broadly, we could:
• Advance the development of therapies more rapidly,
• Positively impact outcomes,
• And expand the knowledge of doctors and researchers.
Our new mission is to inspire solutions that accelerate treatment discovery and improve quality of life by listening to our communities and uniting stakeholders around them. This is how we will achieve our vision, because discoveries come faster when we are all working together.
We spent a large portion of 2019 understanding how the data we are collecting can inform the FDA’s regulatory decision-making process. The 2016 21st Century Cures Act is a pivotal piece of legislation that underscores the FDA’s commitment to incorporate the patient perspective into the development of drugs, biological products, and devices. This bill validated our belief that the anecdotes of our communities are invaluable. To date, TREND data has been used:
• As evidence of disease impact and unmet needs to obtain breakthrough therapy designation,
• In support of an orphan drug designation request,
• To inform a novel clinical protocol design,
• And as supporting evidence for a patient-focused drug development meeting
It is our hope for 2020 that every disease group engaging with the FDA will start with a Community Voice Report to help guide their path forward. We intend to extend our reach to regulatory agencies around the world.
Every victory has been a team effort involving hundreds of people. Community members, patients, caregivers, stakeholders, and the TREND organization have all come together to do the work, and we all share in the reward
In many cases citizen scientists are leading the way. To shine a light on the incredible achievements of these bold trailblazers, each year we recognize one citizen scientist who has made a substantial impact.
This year, we are presenting the TREND Community Citizen Scientist Award to Lara Pullen, for her tireless work on behalf of the PWS community and her relentless commitment to improving the quality of life for everyone living with the disorder.
Moving forward into 2020, we plan to collaborate with even more amazing communities, while strengthening the partnerships we’ve already established. We aim to discover new unmet needs and inspire our stakeholders to create solutions. We will work more closely with our clients and regulators to ensure the patient voice is heard.
May this year bring you happiness and hope,
Maria Picone, Co-founder & CEO
Christopher DeFelice, Co-founder & CTO