GTTs

Genetically targeted technologies (GTTs) target diseases at the genetic level going beyond treating symptoms and can be life-saving and transformative for patients and caregivers. GTTs can improve quality of life, ease the burden of disease, and address adherence challenges for people managing various diseases as well as their caregivers. There are promising GTTs across several disease states including cardiovascular health, rare diseases, neurological conditions, and pediatric health, all of which directly and indirectly impact people who have limb loss and limb difference. Genetically targeted technologies are not gene therapies, though are often confused as the same. GTTs do not permanently alter proteins but rather turn them on or off for a period of time, which addresses diseases at their root cause, which can transform outcomes. The MINI Act would protect GTTs by incentivizing manufacturers to continue developing these therapies, making them available to more individuals who need them.

There are GTTs for Peripheral Arterial Disease (PAD), which is the leading preceding diagnosis of amputation in the US today. Cardiovascular disease remains the leading cause of death among Americans, yet it is often underdiagnosed and undertreated. GTTs as a treatment are taken once or twice per year, instead of the more commonly available daily medication that requires more frequent consumption, which can improve adherence, which is a critical factor in managing cardiovascular diseases.

According to the Government Accountability Office (GAO), 61% of Medicare beneficiaries who had amputations in 2016 were dead by 2019, which is an atrocious mortality rate. Further, 33% of all individuals who have amputations will need a second amputation due to complex health conditions. We also believe there is a future where GTTs could address congenital conditions that lead to and cause limb differences and/or amputations after birth, such as muscular dystrophy. We want to make sure everyone is able to access those treatments.

The limb loss and limb difference community also experiences a multitude of comorbidities at any given time, including diseases such as Alzheimer’s, Parkinson’s, multiple sclerosis, familial hypercholesterolemia, and spinal muscular atrophy. Veterans face higher rates of cardio, neurological, and rare diseases, often linked to service.

Amputee Coalition has not weighed in on the IRA (Inflation Reduction Act), however does continue to advocate for a technical fix, related to GTTs called the MINI Act. MINI stands for “Maintaining Investments in New Innovation” and has been reintroduced in the 119th Congress as HR 1672. In the 118th Congress, the MINI Act was bipartisan and bicameral, though it did fail to pass in the end of year healthcare package. If passed, the MINI Act will ensure that the pipeline for innovation remains viable by appropriately designating Genetically Targeted Technologies for purposes of price negotiation.

April 8, 2025 – Ashlie White, then Chief Strategy & Programs Officer and current Policy Advisor, spoke on a panel hosted by HealthyWomen on the impact of GTTs on the limb loss and limb difference community.