The STAT Act is a necessary piece of federal legislation to develop new and accessible treatments for rare-disease patients. The bill (in full, the Speeding Therapy Access Today Act of 2021, HR 1730 and S. 670) streamlines therapy development for small patient populations. It also builds upon the work of HIV/AIDS activists to ensure that the FDA becomes more responsive to patients' needs.

The bill was introduced to the US House and Senate in March 2021 by Sens. Klobuchar (D-MN) and Wicker (R-MS) and Reps. Bilirakis (R-FL) and Butterfield (D-NC). The legislators also serve as co-chairs of the Rare Disease Congressional Caucus.

There are over 7,000 recognized rare diseases, each occurring in fewer than one in 200,000 Americans. However, rare diseases in general are common — about one in ten Americans lives with at least one of them. These patients lack legislative support to access necessary healthcare and face unnecessary barriers to therapies. Delays and the inaccessibility of treatments disproportionately impact already marginalized people, including the BIPOC and LGBTQIA+ communities.

What the act would do
The STAT Act would create an FDA Rare Disease Center of Excellence and a Rare Disease and Condition Drug Advisory Committee, both of which would aid in delegating resources to and better serving small patient populations. These programs would inform how the FDA makes decisions regarding rare diseases, including how they interact with the Accelerated Approval pathway.

Today, it takes an average of 15 years to develop and approve rare disease treatments. Waiting 15 years is far too long for patients with fatal or degenerative diseases. The Accelerated Approval pathway, created in 1992 following the work of HIV/AIDS activists, streamlines the research of certain diseases and is currently used in many rare disease therapies.
The STAT Act would continue their advocacy, saving even more patients' lives by developing and distributing treatments sooner.

The STAT Act would also create an ALTITUDE (Accelerating Lifesaving Therapies in Treating Ultra-Rare Disease Entities) program for ultra-rare disease patients, who face additional barriers to treatment. There are often not have enough patients to qualify for treatments via the Accelerated Approval pathway. Ultra-rare diseases also pose greater obstacles to collecting clinical data due to such small patient populations. The ALTITUDE program would focus on manufacturing standards, flexible regulations, and best practices for clinical trials.

Finally, the act would put resources toward making treatments more accessible to all rare disease patients through a Rare Disease Therapy Access Program. This would inform policies regarding coverage, which is often a barrier to treatments. Some patients currently do not qualify for coverage because of factors like age, other health conditions, or using Medicare or Medicaid, which the STAT Act would review to remove unnecessary barriers.

Some patients have had treatments denied because they do not meet strict criteria set by clinical trials. This excludes children and older adults or people with other rare diseases or disabilities. These patients may benefit from therapies but cannot receive them solely based on coverage policies. Both private insurance and public insurance like Medicare, Medicaid, and CHIP would be brought together by the Rare Disease Therapy Access Program to promote greater access and coverage for patients.

As a rare disease patient, I strongly support the STAT Act. I was born with Ehlers-Danlos syndrome, a kind of connective tissue disorder that impacts my entire body. It still has no FDA-approved treatment, like about 95% of rare diseases. Untreated, it has led me to develop other rare diseases and chronic illnesses. Rare disease impacts most of my body and every part of my life. For people like me, the STAT Act could be life-changing or even life-saving, by supporting new therapies.

Community members can support rare-disease patients by sharing information on social media, contacting legislators to co-sponsor the bill, or signing an online community letter of support at STATact.org.

For more information, including whether your legislators co-sponsor this act and suggested phone/email scripts, see STATact.org

Photo credit: Rare Disease Week on Capitol Hill in early 2020 — Photo courtesy of EveryLife Foundation for Rare Diseases