Our Priorities

IGT aims to translate the value of transformative therapies so that their potential can be realized and patients with serious genetic disease can thrive. We seek a modernized regulatory and reimbursement framework that encourages the development of transformative gene therapies and promotes patient access. We will focus on the following priorities: 

Educate stakeholders about the potential of gene therapy to transform human health

While gene therapies have been studied for many years, they are just beginning to gain approval by the FDA. As a result, most people don’t know that they differ from traditional medicines in significant ways. There are also many misconceptions about gene therapies due to the complexity of these therapies and a lack of access to current information. IGT will clarify the important differences and incredible potential of gene therapies.

Address regulatory and reimbursement hurdles to speed near-term access to gene therapies

The FDA has prioritized the acceleration of gene and cell therapy approvals and expects to receive over 200 investigational new drug applications (INDs) each year. The current reimbursement system will not be able to accommodate the influx of approvals, which will adversely impact patient access. To help ensure these transformational therapies can be approved in a timely way and patients can access them when they are available, novel development pathways need to be embraced  and new value-based arrangements must be tested. IGT will work with policymakers to advance these solutions.

Ensure a greater understanding about the value gene therapies bring to patients, families, the healthcare system and our society 

Gene therapies will address diseases at their source, rather than simply mitigate signs and symptoms. Their potentially long-lasting effects will greatly reduce – and in some cases eliminate – the need for ongoing treatments and procedures over a patient’s lifetime. We believe  gene therapies will reflect the value they bring to patients and the healthcare system over the long term. We are committed to helping stakeholders understand this dynamic.

Advocate for sustainable, long-term solutions that ensure gene therapies can achieve their full potential for patients 

If we do nothing, the current federal healthcare system, including price-reporting methodologies administered by the Centers for Medicare and Medicaid Services, will prevent patients from realizing the value of access to gene therapies.

 

We will engage stakeholders across the healthcare community to modernize the reimbursement pathways within these systems so that they keep pace with medical science.

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The Institute for Gene Therapies advocates for a modernized regulatory and reimbursement framework that encourages the development of transformative gene therapies and promotes patient access. 

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