February 20, 2020 -The Institute for Gene Therapies (IGT), a new advocacy foundation focused on policy issues surrounding gene therapies, recently launched with the goal of modernizing US regulatory and reimbursement framework for gene therapies.

According to IGT, the US health system currently spends about 85 cents of every healthcare dollar managing the symptoms of chronic diseases over a patients lifetime. With gene therapies, providers could alter patients non-functioning genes or replace absent ones, leading to long-lasting effects and potentially reshaping the way thousands of diseases are treated.

While traditional biologic and pharmaceutical medicines help manage the symptoms of disease over time, gene therapies target the cause of disease at the DNA level, creating lasting changes in the body. Some gene therapies are also designed to be one-time treatments that offer lifelong benefits.

IGT will work to educate stakeholders across the healthcare system about the potential for gene therapies to treat and cure common and rare chronic diseases, and advocate for policies that help ensure patients who need gene therapies can benefit from them.

Existing regulatory and reimbursement structures were established and adjusted over time to support pharmaceutical and biologic medicines, IGT noted, and need refining to accommodate the potential of gene therapies.

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Many crippling conditions like Charcot-Marie-Tooth (CMT), 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.

The FDA has already approved four gene therapy products, and researchers are studying hundreds more in clinical trials for rare and common diseases, including many types of cancer, neuromuscular diseases, blood disorders and infectious diseases, and other conditions.

The growth of innovative research and product development in the field of gene therapy is exciting to us as physicians, scientists and regulators, FDA Commissioner Stephen M. Hahn, MD, said in a statement.

We understand and appreciate the tremendous impact that gene therapies can have on patients by potentially reversing the debilitating trajectory of diseases. These therapies, once only conceptual, are rapidly becoming a therapeutic reality for an increasing number of patients with a wide range of diseases, including rare genetic disorders and autoimmune diseases.

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The FDA has also released six final guidance documents on gene therapy manufacturing and clinical development of products. These documents incorporate input from stakeholders across the healthcare industry and make important strides in designing a modern structure for gene therapy development.

As the regulators of these novel therapies, we know that the framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field and further enable innovators to safely develop effective therapies for many diseases with unmet medical needs, said Peter Marks, MD, PhD, director of the FDAs Center for Biologics Evaluation and Research.

Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review; including how these products work, how to administer them safely, and whether they will continue to achieve a therapeutic effect in the body without causing adverse side effects over a long period of time.

IGT will being together experts across the healthcare community, including corporate leaders, patient advocacy groups, and academic and scientific stakeholders, to ensure health policies reflect the latest advancements in gene therapies. The institute will also work to remove barriers that limit patient access to these therapies and promote sustainable, long-term solutions.

Experts will learn more about the value gene therapies bring to patients, their families, and the healthcare system as a whole. IGT will help advance chronic disease treatments and get to the root cause of some of the most debilitating, expensive conditions that affect patients throughout their lives.

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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.

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New Gene Therapies Institute Aims to Address Regulatory Barriers - HealthITAnalytics.com

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