The COVID-19 pandemic has led to many changes, disruptions, and setbacks. For rare diseases, it did delay research for new treatments. However, there were a few positive things that came from this public health crisis. For instance, through research on COVID-19, rare disease research has been improved.

Researchers, through learning more about the biologics of COVID-19 and focusing so much on how current medications could be repurposed for COVID-19, also gained a greater understanding of repurposing in general. This alone could aid their research for other conditions. Particularly, this newfound knowledge could be helpful for rare diseases.

While researching COVID-19, NCATS tested more than 10,000 different compounds. They have around 3,000 molecular entities, 2,900 of which are in their screening collection.

They learned about the importance of rapid response and how to look at and understand a lot of data very quickly. A lot can be done in a short amount of time if you have the dedication, funds, and mindset to do so.

Rare Disease Research

Most rare diseases have a genetic cause. This means that many can be treated with similar genetic tools such as gene therapies, gene editing, and even antisense oligonucleotide technologies. This is huge because it means that one treatment can be widely applicable.

Although there will always be hardships in regard to rare disease treatment, such as a lack of diversity in clinical trial participants, and recruitment for trials in general, working with research for other conditions could improve outcomes.

Rare disease research in regard to COVID-19 is also critical. NCATS worked with the Rare Diseases Clinical Research Network, the University of Pittsburgh, and the rest of the NIH, to study how COVID-19 has affected rare disease patients. They studied both COVID-19 cases in the rare population as well as vaccination rates. As a part of this effort, they surveyed patients. 

You can read more about rare disease research during COVID-19 here.