Unlocking Hope with Improved Access to Gene Therapy

Few breakthroughs have been as transformative as cell and gene therapies (CGTs). For people living with sickle cell disease (SCD), gene therapy offers the promise of a pain-free life. Despite the promise, access to CGTs remains out of reach for many.

The story of Kyle, a young man whose life was reshaped by gene therapy, underscores both the power of these treatments and the need to make them more accessible.

A life transformed by science

Kyle’s journey began with a dream: to fly. From the age of 5, he was captivated by airplanes, determined to become a pilot. But at 16, that dream was grounded by sickle cell disease — a genetic disorder that causes red blood cells to become misshapen and block blood flow, leading to excruciating pain, organ damage, and shortened life expectancy. For Kyle, even mild exertion could trigger a crisis. He spent countless days in the hospital, battling pain so severe he once told his father he wanted to die.

Kyle holding a toy airplane.

Then came a breakthrough. Kyle entered a clinical trial for a new gene therapy at Children’s Healthcare of Atlanta. On his 19th birthday, his stem cells were collected, genetically repaired, and reintroduced into his body.

The result was nothing short of miraculous. His body began producing healthy red blood cells, and the pain crises stopped. Today, Kyle holds a private pilot license and is training to fly commercially. “It completely changed my life,” he said. “And I want everyone out there with the disease to have the same access and same hope.”

The promise of gene therapy

Kyle’s experience shows CGT's potential to change the lives of an estimated 100,000 Americans living with SCD. In late 2023, the FDA approved two CGTs for SCD. These one-time treatments correct the genetic mutation responsible for SCD.

However, the promise of these therapies is tempered by their staggering cost, which can be as much as $3.1 million for the gene therapy drug alone. Beyond the price tag, the treatments require extended hospital stays, specialized care teams, and complex logistics. These barriers make access nearly impossible for many families, especially those relying on Medicaid.

Bridging the gap: The CMS Access Model

Recognizing the urgency of the situation, the Centers for Medicare & Medicaid Services (CMS) launched the Gene and Cell Therapy Access Model in January 2024. This initiative brings together CMS, state Medicaid agencies, and pharmaceutical companies to negotiate outcomes-based payment agreements. Under this model, drug manufacturers offer rebates tied to patient outcomes, making these CGT therapies more affordable for states and accessible to more patients.

The Children’s Hospital Association (CHA) has applauded this move, emphasizing that nearly half of the nation’s children are Medicaid beneficiaries. As of July, 33 states and the District of Columbia have indicated they will participate. Of these, seven states and the District of Columbia have already been awarded Cooperative Agreement funding.

Children’s hospitals are at the forefront of delivering these therapies, but they face challenges, including inconsistent insurance coverage, administrative burdens, and limited commercial availability. The CGT Access Model is a critical step forward to ensure no child is denied treatment.

Looking ahead

The future of pediatric medicine is being written in the labs and clinics pioneering CGTs. As Kyle’s story shows, gene therapy can restore dreams and a new outlook on life. Now, the challenge is to ensure that every child with sickle cell disease has the same opportunity.

Kyle is flying high today, not just because of medical advancement, but because someone gave him access. It’s time to do the same for every child still waiting on the ground.