Sarah Jenssen had been waiting for a ray of hope in her battle against Duchenne muscular dystrophy, a degenerative neuromuscular disorder. The approval of a gene therapy by the FDA earlier this year seemed like a promising breakthrough for her. The therapy, Elevidys by Sarepta Therapeutics, was authorized for most patients with Duchenne, regardless of their age or disease progression.
However, the Jenssens’ joy was short-lived when their insurer denied coverage for the $3.2 million treatment for Sarah. The reason cited was that Elevidys was only approved for patients who could still walk independently, which put Sarah, who had started using a wheelchair, at a disadvantage. The family was devastated by the denial, feeling like they had hit a dead end.
After Sarah’s medical team appealed the decision, there was a glimmer of hope as the insurer eventually agreed to cover the treatment for her. With a few more steps to go, Sarah could potentially receive the therapy as soon as November. The road to access life-changing treatments like Elevidys is often fraught with challenges, as seen in Sarah’s case.
The controversy surrounding Elevidys’ approval added fuel to the fire, with concerns about the treatment’s efficacy and safety in older patients who use wheelchairs. Critics questioned the FDA’s decision to approve the therapy despite mixed results in clinical trials. The debate highlighted the tension between providing access to innovative treatments and ensuring their effectiveness and affordability.
Insurers, facing the dilemma of covering high-cost gene therapies, sometimes align their policies with clinical trial criteria, leading to access issues for patients like Sarah. The complex interplay between regulatory standards, insurance policies, and drug pricing creates barriers for families seeking life-changing treatments.
See also Eliminating Waste, Fraud, and Abuse in Medicaid My Administration has been relentlessly committed to rooting out waste, fraud, and abuse in Government programs to preserve and protect them for those who rely most on them. The Medicaid program was designed to be a program to compassionately provide taxpayer dollars to healthcare providers who offer care to the most vulnerable Americans. To keep payments reasonable, billable costs for such care were historically capped at the same level that healthcare providers could receive from Medicare. The State and Federal Governments jointly shared this cost burden to ensure those of lesser means did not go untreated. Under the Biden Administration, States and healthcare providers were permitted to game the system. For example, States "taxed" healthcare providers, but sent the same money back to them in the form of a "Medicaid payment," which automatically unlocked for healthcare providers an additional "burden-sharing" payment from the Federal Government. Through this gimmick, the State could avoid contributing money toward Medicaid services, meaning the State no longer had a reason to be prudent in the amount of reimbursement provided. Instead of paying Medicare rates, many States that utilize these arrangements now pay the same healthcare providers almost three times the Medicare amount, a practice encouraged by the Biden Administration. These State Directed Payments have rapidly accelerated, quadrupling in magnitude over the last 4 years and reaching $110 billion in 2024 alone. This trajectory threatens the Federal Treasury and Medicaid's long-term stability, and the imbalance between Medicaid and Medicare patients threatens to jeopardize access to care for our seniors. I pledged to protect and improve these important Government healthcare programs for those that rely on them. Seniors on Medicare and Medicaid recipients both deserve access to quality care in a system free from the fraud, waste, and abuse, that enriches the unscrupulous and jeopardizes the programs themselves. We will take action to continue to love and cherish the Medicare and Medicaid programs to ensure they are preserved for those who need them most. The Secretary of Health and Human Services shall therefore take appropriate action to eliminate waste, fraud, and abuse in Medicaid, including by ensuring Medicaid payments rates are not higher than Medicare, to the extent permitted by applicable law. This memorandum is not intended to, and does not, create any right or benefit, substantive or procedural, enforceable at law or in equity by any party against the United States, its departments, agencies, or entities, its officers, employees, or agents, or any other person. DONALD J. TRUMP
Despite the challenges, the approval of Elevidys for Duchenne patients marked a significant milestone in the fight against the disease. For Sarah, the therapy offers a glimmer of hope in preserving her muscle function and potentially extending her life. The journey to accessing Elevidys has been a rollercoaster for the Jenssens, underscoring the complexities of navigating the healthcare system for rare diseases.
As Sarah awaits the treatment that could change her life, her family remains hopeful that Elevidys will provide her with the opportunity to live a longer and more independent life. The battle for access to innovative therapies continues, but for now, there is a flicker of hope on the horizon for Sarah and others like her who are fighting against Duchenne muscular dystrophy.
