Rare Disease Day is February 28th. This year, it caused a lot of reflection among healthcare professionals and families impacted by rare conditions. How has the pandemic shifted progress in 2020? What can we expect for 2021?
Progress Continued
Thankfully, although the pandemic cancelled, postponed, and shifted many events, rare disease research was not affected. Drug development progressed throughout the year of 2020. In fact, more progress was made than in 2019!
During 2020, The FDA issued 476 Orphan Drug Designations. In 2019, just 340 were issued.
Another huge development which occurred in August of 2020 was a public meeting for the COA Endpoints Pilot Program, to discuss outcomes for their grant programs. COA has existed since 2019 and there are three standard core sets. These are for migraines, acute pain in children, as well as physical function for a wide array of rare conditions. The standard cores work to develop which factors matter the most to patients, and therefore, where research should concentrate.
Policies
One policy implemented by the FDA during the pandemic to help ensure research continued, and continued safely, was the guidance issued regarding clinical trials. Although this guidance for conducting trials during COVID-19 was not specific to rare disease, it was vital to ensuring rare disease research continued.
This guidance was published on March 18, 2020, quite quickly after the pandemic began. It was then continued throughout the pandemic to ensure that the guidance matched the current state of the pandemic stricken world. They addressed new questions and continued to provide clarity on issues such as trial enrollment and remote monitoring.
The community is hopeful that these changes will continue long into the future, providing increased flexibility in how we conduct research. Through the pandemic we have seen that virtual trials are possible. Telehealth is possible. Virtual follow up is possible.
This is not to say that all trials should be conducted this way. But by providing researchers more options in how they conduct research, we will be able to conduct more research faster.
As of November of 2020, it was found that 55% of current trials had been able to successfully transition to some virtual and remote means, particularly through the use of wearable devices and other technology.
Another way the FDA has helped to simplify the process during the pandemic is by allowing email submissions to the orphan drug development office as opposed to simply through the online portal.
Additionally, Congress authorized an extension in December of 2020 for the Rare Disease Priority Review Voucher program.
Minimizing COVID-19 Impacts
The FDA also acted quickly in effort to minimize the impact of COVID-19 for rare disease patients. For instance, when it became clear that Hydroxychloroquine could be a therapeutic option for COVID, the FDA created a priority review process to ensure that Lupus patients were still able to access the medication they needed as demand increased.
You can read more about the progress in rare disease research in 2020 here and how this progress may be maintained throughout 2021, even as the pandemic continues.
