Global Genes, a leading rare disease patient advocacy organization, today released NEXT 2021: A Time for Resilience and Ingenuity report, highlighting forward-looking developments, trends, and progress across the rare disease landscape.
Among the trends to watch in the year ahead, the report highlights:
- Data as a Driver of Progress
There is a growing movement to break data free from silos and foster data sharing to improve the diagnosis of rare disease and accelerate the development of new therapies. 2021 marks an important juncture as we look to overcome obstacles, cultural barriers, and convince data holders of the value of sustained and open collaboration within and across disease areas.
- Nimble Development, Tailored Solutions
The year ahead will also be an important one for advancing the feasibility of individualized therapies and therapies for ultra-rare populations. There are several efforts underway to test new models for development and delivery of treatments to individual patients and ultra-rare populations. While some achieved proof of concept, the feasibility and scalability of these approaches remains as yet unproven, but worth further exploration.
- Aligning Policy With Paths to Progress
As the new Congress takes office, the seventh incarnation of the Prescription Drug User Fee Act (PDUFA) is already taking shape. One issue rare disease advocates are expected to push for in PDUFA VII is the creation of a Rare Disease Center of Excellence at the FDA, similar to what the agency did when it created the Oncology Center of Excellence. The center would provide a ready source of expertise to weigh in on issues ranging from clinical trial design to drug reviews and work across the different centers and offices of the agency.
- Leveling the Playing Field
While the challenges of finding a diagnosis and treatment for a rare disease are well-documented and widespread, racial and other disparities can further hinder the ability to achieve an accurate and timely diagnosis, gain access to support services and specialized care, and can lead to underrepresentation in research, clinical trials, and advocacy efforts. The rare disease community is increasingly getting behind efforts to better understand gaps and develop solutions to help ensure that progress is equally accessible and beneficial to all members of each rare community.
“2020 taught rare disease organizations a number of important lessons,” said D. Craig Martin, CEO of Global Genes. “In the race to develop vaccines for COVID-19, we’ve seen researchers, government and industry work together in unprecedented ways and at an unprecedented pace. If we can work as collaboratively and rapidly in addressing rare diseases, imagine what could be accomplished! Yet we also saw how the prioritization of pandemic response affected rare disease patients’ access to care and trials. As we move beyond the pandemic, we will need to redouble our efforts to call attention to the collective impact and urgency of rare disease and act with resilience and ingenuity to further accelerate the development of treatments and cures.”
About Global Genes®
Global Genes is a 501(c)(3) nonprofit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. In pursuit of our mission, we connect, empower, and inspire the rare disease community to stand up, stand out, and become more effective on their own behalf – helping to spur innovation, meet essential needs, build capacity and knowledge, and drive progress within and across rare diseases. We serve the more than 400 million people around the globe and nearly one in 10 Americans affected by rare diseases. If you or someone you love has a rare disease or are searching for a diagnosis, contact Global Genes at 949-248-RARE, or visit our resource hub.