There are many ways that people living with rare diseases and their loved ones can be advocates! We connected with Leah Svoboda, Director, Government Affairs and Public Policy, on what it means to advocate on behalf of legislation in the U.S., the impact of this kind of advocacy and the ways PTC is advocating for patients.
What does it mean to advocate on behalf of legislation?
Leah: Advocating for policy or legislative change means seeking to shape the system of federal, state, and local laws, rules and regulations which govern everything from how we vote to how we access healthcare. Petitioning our government for change is one of the fundamental First Amendment rights we have as U.S. citizens.
What can people with rare diseases and their loved ones do to advocate on behalf of legislation?
Leah: Successful advocates are not only familiar with the policy issue they are focused on, but also the process and the people who are best suited to help make change. Become a resource for information on your issue to build a relationship with your key stakeholders. Policymakers seek advocate partners who clearly communicate the issues and suggest workable solutions they can help advance.
What is the impact this type of advocacy can have on the rare disease community?
Leah: From difficulties obtaining a diagnosis, to a lack of available therapies and access barriers, rare disease patients face many serious challenges. By sharing their experience and expertise with policymakers, people living with rare diseases can help shape development incentives, coverage guidelines and a host of other policies which will have a huge impact on their lives and future.
How does PTC help advocate for patients?
Leah: PTC’s Government Affairs and Public Policy team has a presence in Washington, DC and across the U.S. focused on eliminating barriers to access and promoting innovation. We continuously analyze the impact of current and proposed legislative and policy changes on our patient communities and our company. We partner with patients and patient advocacy organizations to better understand the issues affecting them and collaborate to increase education and advance solutions in these areas.
Learn more about advocacy in our Insightful Moments™ Advocacy Works! roadmap.
If you have questions or want to learn more, please call 1-833-PTC-HOPE (1-833-782-4673) or email us at InsightfulMoments@ptcbio.com.