Gene Therapy – An American Innovation Worth Fighting For

Authors: IGT Chairman Erik Paulsen, Scientific Academic and Medical Council Chair Donna Christian-Christensen, M.D.

As our nation’s healthcare infrastructure evolves, we must ensure that the voices of rare disease patients are not lost in the shuffle. These families – who often face years of misdiagnosis, fragmented care, and limited treatment options – cannot afford delays or disruptions. They need bold leadership, thoughtful policies, and a healthcare system willing to embrace innovation.

Gene therapy offers a rare and remarkable opportunity: to cure – not just manage – serious, often fatal genetic diseases. These are typically one-time treatments designed to correct the root genetic cause of disease. That’s not just medicine, that’s hope.

The U.S. has emerged as a global leader in gene therapy development. We hold over 40% of the global market share and support one of the world’s largest portfolios of clinical trials. Our research institutions, from coast to coast, are advancing science that will transform lives, the gene therapy pipeline is considerably robust which not only translates into jobs across the U.S. but also incredible potential for people and families navigating the challenges of genetic conditions.

But scientific progress means little if patients can't access these treatments. We’re at a crossroads. Decisions made today will determine whether gene therapies reach families who need them – or remain out of reach due to outdated policy, regulatory uncertainty, or financial barriers.

To seize this moment, there are concrete actions that our nation’s decisions makers can and must take:

1. Expand newborn screening (NBS): Early diagnosis is essential to ensure that children with genetic diseases have access to life-saving therapies as early as possible. With enhanced screening, we can identify treatable conditions sooner and intervene when it matters most.

   
   

2. Remove outdated legal barriers: Recent court decisions, like preventing companies from covering fertility preservation for patients, highlight how legal frameworks written decades ago are out of step with today’s science. These impediments may be invisible to most, but for rare disease families, they can mean the difference between cure and continued suffering.

   
   

3. Broaden access through Medicaid: The Cell and Gene Therapy (CGT) Access Model, piloted under the first Trump Administration, should be expanded to cover all FDA-approved gene therapies immediately upon approval. This would not only improve access and affordability but also reward innovation through value-based purchasing arrangements, providing other payers with a model to explore implementing for their own enrollees.

   
   

4. Protect the Accelerated Approval Program: This vital FDA pathway brings treatments to patients years faster than traditional models – especially critical for rare diseases, where conventional trials are difficult due to small patient populations. Weakening this program threatens to delay, or deny, access to life-saving therapies.

   
   

Gene therapy is the ultimate form of preventive medicine. These treatments don’t just improve symptoms; they offer long-term health benefits, dramatically reduce healthcare costs, and restore the quality of life for patients and families alike. Rather than managing disease over a lifetime, we have a chance to eliminate it at its genetic source.

Yet access to gene therapies remains uneven, and the pipeline for new therapies is vulnerable. If we send the wrong signals to investors or create uncertainty around coverage and approval pathways, we risk stalling innovation. And when innovation stalls, it is patients – especially those with rare diseases – who pay the highest price.

Some state Medicaid programs, and commercial insurers are limiting coverage of gene therapies approved under the accelerated approval pathway. Patients are rightly concerned that the Centers for Medicare and Medicaid Services may follow suit. If we don’t address these challenges head-on, we risk wasting the scientific momentum and investment that have brought us this far.

This is not just a policy issue; it’s a moral one. Are we willing to tell a parent that a cure exists for their child, but they can’t have it because of outdated rules or red tape? Are we willing to let breakthrough therapies gather dust while patients suffer?

The healthcare system must evolve to reflect what science can now deliver. That means protecting the regulatory and coverage frameworks that enable innovation, expanding screening and early diagnosis, and ensuring equitable access to therapies for every patient – regardless of where they live or how much they earn.

We urge policymakers, regulators, and health leaders to act with courage and vision. Let’s double down on American leadership in science and medicine. Let’s create a future where diagnosis leads not to despair, but to hope, healing, and cures.

The future is bright – but only if we choose to act.