As we commemorate the fifth anniversary of the Institute for Gene Therapies (IGT) today, I want to take a look back at the strides we've made in advancing patient access to innovative gene therapies.
Gene therapies are unique – targeting the root cause of disease – providing patients with chronic, debilitating, and often fatal diseases critical hope. With nearly 20 FDA-approved therapies today, patients with rare diseases, including hemophilia, sickle cell disease, and spinal muscular atrophy now have the potential to live longer, healthier lives. This allows not only patients but also their caregivers and families the ability to enjoy more meaningful time together, contribute more to their communities and save money on long-term healthcare costs.
In February 2020, IGT was established. We recognized that while gene therapies hold immense promise, significant challenges threatened to prevent patients from accessing these treatments, as the science had advanced more quickly than our healthcare system.
IGT realized we need a modernized regulatory and reimbursement framework that encourages the development of transformative gene therapies and promotes patient access.
Central to our efforts has been the creation and expansion of our advisory councils. Our Patient Advocacy Advisory Council critically ensures that patient perspectives remain at the forefront of our initiatives. From panel discussions on Capitol Hill to PSAs shared across the country, our patient advocates have lent their voices to policy discussions and helped to demonstrate the urgent need to help deliver these therapies to patients who stand to benefit.
IGT advocates have supported the introduction of the Medicaid VBPs for Patients (or MVP) Act, recognizing that patients' ability to access gene therapies is imperative to realizing the promise of these transformative treatments. We have encouraged Congress to advance bipartisan bills like the Accelerating Kids’ Access to Care Act that represent hope for the rare disease community and will meaningfully improve access to life-changing and lifesaving treatments. We have pushed back against harmful regulatory policies and restrictive actions that put patient access at risk.
With a new administration and new Congress, we remain committed to educating policymakers about the value of gene therapies and the importance of accurately assessing their impact.
The journey is ongoing, but with the collective efforts of our partners, advisors, and supporters, I am confident that we will overcome the challenges ahead. Thank you to everyone who has been part of this incredible journey. Together, we are realizing the promise of gene therapies and transforming healthcare for generations to come.
Sincerely,
Erik Paulsen
Chairman, Institute for Gene Therapies
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