Industry leaders, patient advocates, researchers unite to maximize the incredible potential of transformative gene therapies
The Institute for Gene Therapies (IGT) launched today with a focus on advocating for a modernize the U.S. regulatory and reimbursement framework so that gene therapies can deliver their significant potential to patients. IGT will educate stakeholders across the healthcare community about the transformational nature of gene therapies and advocate for policies that help ensure patients who need them can benefit from them.
Gene therapy is poised to change human health as we know it. By altering non-functioning genes or replacing absent ones, gene therapies have the potential to reshape the way thousands of diseases are treated with long-lasting effects for patients. The first of these transformative therapies have already been approved by the U.S. Food and Drug Administration (FDA) and hundreds more are currently being studied in clinical trials for rare and common diseases, including many types of cancer, neuromuscular diseases, blood disorders and infectious diseases, among others.
“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,” said Susan Ruediger, CEO of the CMT Research Foundation (CMTRF) and member of the IGT Patient Advocacy Advisory Council. “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.”
Gene therapies are fundamentally different from traditional pharmaceutical and biologic medicines in that they target the cause of the disease at the DNA level to create a change in the body. Further, some gene therapies are designed to be one-time treatments that offer life-long benefits. Today, the vast majority of medicines help manage the symptoms of disease over time rather than address – or halt – diseases at their root. The U.S. healthcare system – from the drug approval process to the way treatments are paid for – reflects this reality. The existing regulatory and reimbursement structures, which were established and adjusted over time to accommodate pharmaceutical and biologic medicines, need revisiting in light of gene therapies and their significant potential.
“The incredible scientific advancements in this space present unique opportunities to directly improve and save the lives of patients suffering from debilitating diseases,” said IGT Chairman, and former Congressman Erik Paulsen. “This is not some far-off future – patients are already benefiting from the first FDA-approved gene therapies. 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.”
As part of IGT’s effort, experts from across the healthcare system will work together to ensure health policies reflect the latest medical advances, remove barriers that hinder patient access to gene therapies and advocate for sustainable, long-term solutions. IGT will work to ensure a greater understanding about the value gene therapies bring to patients, families, the healthcare system and our society so that gene therapies can achieve their full potential.
About the Institute for Gene Therapies
The Institute for Gene Therapies (IGT) works with stakeholders across the healthcare system to advocate for a modernized regulatory and reimbursement framework that encourages the development of transformative gene therapies and promotes patient access. Members of our advisory councils include Johnson & Johnson, PTC Therapeutics, Sarepta Therapeutics, Spark Therapeutics, Patient Advocate Foundation, Cure SMA, CMT Research Foundation, American Autoimmune Related Diseases Association (AARDA), Khrystal Davis, Founder of Texas Rare Alliance, Jenn McNary, Founder of One Rare, Seth Rotberg, Co-Founder and Head of Strategy & Engagement of Our Odyssey, Rolf Benirschke, Patient Advocate for Crohn’s disease, ulcerative colitis, colorectal and bladder cancer, Friedreich’s Ataxia Research Alliance, and Foundation Fighting Blindness. For more information, visit gene-therapies.org and follow us on Twitter @gene_therapies
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