Your Turn — Virginia needs to guarantee access to innovative treatments

Published 12:00 pm Tuesday, December 13, 2022

Advancements in innovative medical treatments are revolutionizing care for many Virginia patients and their families. As a Farmville resident living with Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness, I know well the hope that these treatments offer to a broad range of patients, particularly those living with chronic, rare, or complex conditions.

However, investment in the development of new drugs will not have the intended impact unless lawmakers prioritize health system readiness and pathways to access for patients who need these treatments most. Virginia legislators must support policies that ensure patients, especially those living with rare diseases, are able to access and afford lifesaving health care when new medications come to market.

About 1 in 10 Americans are living with a rare disease, or a disease or condition that affects fewer than 200,000 people. In 1998, when I was 3 years old, I was diagnosed with Duchenne muscular dystrophy. DMD is one of the most serious genetic diseases in children. It is one of the nine types of muscular dystrophy and affects almost exclusively males, with a birth prevalence of about 1 in every 3,500 male births. More than 90% of kids diagnosed with DMD end up in wheelchairs by age 11.

Gene therapy is a type of medical treatment that involves adding, removing, or changing a person’s DNA. The future of gene therapies is especially important for rare diseases, because these types of treatments focus on correcting the root cause of a disease, rather than just a solution to help patients manage symptoms. As a result, gene therapies can offer patients a better chance at long-term health improvements and quality of life.

I have dedicated years advocating for the support of Duchenne-related legislation to state and federal legislators. Now, I am urging Virginia lawmakers to ensure that when new gene therapy medications and treatments that address the world’s toughest diseases come to market, Virginia’s health systems are prepared to enable access for all eligible patients.

Increased investments in innovation, research, and development continue to push critical cell and gene therapies forward. In May 2021, there were 1,745 gene therapies in development, an over 30% increase than 2020. These treatments have the possibility to transform patients’ lives and bring much-needed personalized health solutions to the horizon.

The gene therapies that are being researched and developed to treat DMD could have changed my life when I was a child. Treatments like these enable patients to live healthier lives without the need for ongoing treatments or daily disease management.

As Virginia policymakers continue to explore avenues to improve health care, policies that foster a stronger statewide system to support the next generation of transformative medications for patients are crucial. Gene therapy holds promise for people like me, all adults living with Duchenne, but to realize its full potential, policymakers must take action now to ensure systemic preparedness and improved treatment access for those who need it most.

COLIN WERTH is a patient advocate living with Duchenne muscular dystrophy in Farmville. He can be reached at cjrailstar@gmail.com.