By Regina Hartfield, President and CEO, Sickle Cell Disease Association of America, Inc.
This year, we celebrate a landmark step forward in minority health. The December 2023 Food and Drug Administration approvals of the first two cell-based gene therapies for patients 12 and older to treat sickle cell disease (SCD) marks a huge advancement in the fight against this devastating disease. Now more than ever, it is time to have a national conversation to build consensus on how to make cutting-edge medical innovations more accessible to those who need them.
Sickle cell disease is a serious condition that poses lifelong challenges to approximately 100,000 Americans and their families. Sickle cell “warriors” face episodes of intractable, crippling acute pain and are at elevated risk for strokes, damage to affected tissue, and all too often, an early death. A recent study showed the median age of death of those suffering from chronic sickle disease complications was only 43 years.
The gene therapies approved by the FDA modify the body’s blood stem cells and, in clinical trials, have demonstrated extraordinary effectiveness in preventing excruciating pain and organ damage. Although more research is needed to understand the long-term impacts of the treatment, it is a potentially curative therapy. For thousands of people, a pain-free life finally seems within reach.
But who will pay for this, and how?
The Biden Administration recently announced a new program through which Medicaid will negotiate rebates with the manufacturers of these treatments to make them more affordable for states. It is then up to the states to decide whether they will participate in the program and help make the gene therapies accessible for enrollees. This should lead to a broader conversation about how to build a bridge between new, transformative health care solutions and the patients who have rare, complex, and life-shortening diseases.
There is the uncertainty that comes with innovation. Our current health care coverage system simply isn’t built for the cell and gene therapies that are entering the pipeline with increasing frequency. As opposed to conventional treatments for other diseases like cancer, heart disease or diabetes that are taken for months, years, or even over the course of a lifetime, cell and gene therapies are often administered only once and can change the body’s internal makeup to prevent the symptoms of a disease or even eradicate the disease itself. Stakeholders must find a way to ensure delivery to the intended patients, while also ensuring reimbursement for these one-time treatments in a way that enables those who create them to see a return on their considerable investment and incentivize more research into life-changing gene and cell therapies.
As the president of the leading national advocacy organization for individuals with sickle cell, I believe it is our collective moral responsibility to find a way forward. Generations of Americans have lived painful, shortened lives because of sickle cell. For many, the idea of living without pain was just a dream. Now that it can be a reality, it would be unconscionable to leave those treatments out of the reach of families in need. This will require some creativity on the part of manufacturers and health care payers in both the government and the private sector. There have been initial efforts which reimbursement is tied to the therapy’s success in improving the patient’s health. There is also exploration in how to extend payments for these cell and gene therapies over time to make them more affordable. At the end of the day, curative therapies are expensive, but maintaining the status quo is more expensive – for both the individual and our society at large. Beyond the stark reduction in health costs, gene therapy offers many warriors and their caregivers a chance to live a normal life. No more missing class due to extreme fatigue. No more calling out of work because your child had a pain crisis. The impact of removing or greatly reducing these barriers cannot be understated.
We are at a critical turning point for thousands of American families, the vast majority of them people of color. No one with sickle cell disease or other insidious diseases should continue to suffer when our society has the means to improve their health and lives. Nothing would make Minority Health Month more meaningful than advancing a more sustainable infrastructure for access to these lifesaving solutions.
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