New US Institute for Gene Therapies Seeks to Modernize Regulatory, Reimbursement Framework

The Institute for Gene Therapies (IGT) has launched, with the aim of maximizing the potential of gene therapies in genetic disorders such as hemophilia.

Comprised of industry leaders, scientists, and patient advocates, the IGT’s overarching goal is to set the foundation for a modernized regulatory and reimbursement framework that fosters the development of transformative gene therapies, and patients’ access to them. Its mission is educating stakeholders across the healthcare community.

Human genes are made up of DNA, which influences how the body functions on the most basic level. By modifying non-functioning genes or replacing missing ones, gene therapies can prospectively alter the way that many diseases are treated, and with long-lasting results. Some treatments have already been approved by the U.S. Food and Drug Administration (FDA), and many more are in clinical trials for a wide spectrum of diseases, including different types of cancer, neuromuscular diseases, blood disorders — including hemophilia type A and hemophilia type B — and infectious diseases.

“Many crippling conditions — like Charcot-Marie-Tooth, which I was diagnosed with before the age of two — take hold at a very young age, cut lives far too short or cause ongoing daily suffering,” Susan Ruediger, CEO of the CMT Research Foundation (CMTRF) and member of the IGT Patient Advocacy Advisory Council, said in a press release.

“Like so many diseases, CMT currently has no cure. I am proud to stand with other leading patient advocates, members of the research community and companies that are developing gene therapies to help ensure patients can fully realize the benefits of these giant leaps toward treatments and cures,” Ruediger added.

Rather than addressing the underlying causes of different disorders, most medications merely help manage their symptoms. Gene therapies are inherently different from conventional treatments in that they tackle a disease’s root cause in order to create lasting change. In addition, some of these therapies have the benefit of being one-time treatments that effectively cure the disease.

In hemophilia, gene therapies use a harmless virus to introduce a functional copy of a gene that encodes for the missing clotting factor that prevents normal blood clotting. Several clinical trials are currently underway to test potential gene therapies for the disease.

The FDA recently accepted — under priority review — a marketing application for Valrox (valoctocogene roxaparvovec), BioMarin Pharmaceutical’s investigational gene therapy for hemophilia A. The action marks the first time the agency has accepted an application for a gene therapy specifically designed to treat hemophilia. A decision on BioMarin’s Biologics License Application for Valrox, regarding its approval, is expected by August 21.

“The incredible scientific advancements in this space present unique opportunities to directly improve and save the lives of patients suffering from debilitating diseases,” said Erik Paulsen, IGT chairman and former U.S. congressmen.

“This is not some far-off future — patients are already benefiting from the first FDA-approved therapies,” Paulsen said. “But we need policy to move faster toward this new reality where we can treat the causes of many diseases. The Institute for Gene Therapies and our members believe unique regulatory and reimbursement structures need to be established, novel development pathways need to be embraced and new value-based arrangements need to be tested.”

Despite a growing focus on gene therapy in general, current regulatory and reimbursement structures — from the drug approval process to how treatments are paid for — were built over time around pharmaceutical and biologic medicines. Part of the IGT’s mission is to change that paradigm.

The effort calls for stakeholders to collaborate to ensure health policies mirror cutting-edge medical advances, remove hindrances to patient access to gene therapies, and promote long-term remedies. The IGT will work to raise awareness of gene therapy and its potential value. For more information, please visit gene-therapies.org.