For years, veterans with polyneuropathy caused by amyloidosis have faced barriers to receiving the right treatment. The Veterans’ Administration formulary required patients to try medications intended for cardiomyopathy before they could access therapy indicated for polyneuropathy.
The delay meant unnecessary suffering for many, as polyneuropathy and cardiomyopathy represent two distinct presentations of amyloidosis, each requiring unique treatment approaches. With the recent update to the VA formulary, this “fail first” restriction has been removed, offering veterans access to the right treatment sooner.
Understanding Amyloidosis
Amyloidosis causes abnormal proteins to be produced, and eventually build up in tissues and organs, interfering with normal function. The disease takes different forms, with hereditary transthyretin-mediated (ATTR) amyloidosis often affecting the nerves – called polyneuropathy – or the heart – called cardiomyopathy. Other presentations can impact the kidneys, liver, brain, bowel and lungs.
Early diagnosis and intervention are essential to protect patients. Left untreated, amyloidosis can lead to progressive disability and worsening symptoms, driving down quality of life.
Accessing New Treatments
The FDA, in 2022, approved vutrisiran, a subcutaneous injection for hereditary ATTR amyloidosis with polyneuropathy. The same medication has recently been approved for cardiomyopathy of wild-type or hereditary ATTR amyloidosis. The inclusion of this treatment in the VA formulary marks an important step in improving patient care.
Advocates have long urged the VA to update this restrictive policy. Clinicians active with the Alliance for Patient Access and a variety of patient organizations weighed in to the VA, advocating for reforms that would allow access to the full range of therapies.
“We are thrilled about the long awaited and highly anticipated news regarding increased access to treatments for our veterans suffering from ATTR,” said Muriel Finkel of the Amyloidosis Support Groups. “This development marks a significant step forward in ensuring that those who have served our country can receive the medical care they need and deserve.”
Early Treatment Leads to Better Outcomes
Delays in treatment of amyloidosis can lead to irreversible damage and increased disability. When patients receive the right medication early, they have a better chance of slowing disease progression and maintaining their quality of life.
The VA’s decision to update its formulary represents progress, and AfPA is proud to have advocated on behalf of patient-centered amyloidosis care. Every step toward timely, appropriate care makes a difference in the lives of those affected by this challenging disease.
