Helping Those with Rare Diseases, Strengthening Government Transparency Would Improve Pennsylvania in 2020

By Rep. Marcy Toepel


Who knew that the day a local constituent came to my office to talk to me about an issue he was having would send me on a new journey in the Legislature?


New Hanover resident Joe Coyne came to talk to me about his son, who had been diagnosed with Batten Disease. This is a rare disorder of the nervous system, and he was having difficulty finding information and services to help his family navigate his son’s health care.


After this conversation, I started to do research and learned that more than 1.2 million people across the Commonwealth are coping with one of more than 7,000 rare diseases. However, because so little is known about the various diseases, they are having trouble dealing with insurance and finding medical care, services and support.


It was then I realized that although they were all dealing with different diseases, they were still dealing with the same basic issues. Together, these folks were a community unto their own. They just needed a little help to make the connections.


That was when I had the idea of creating a Rare Disease Advisory Council. This council could act as a hub of information and support for those afflicted with a rare disease and their caregivers, while also working with those in the medical and insurance fields to highlight the need for greater focus on rare diseases and advocate for those individuals.


The council became a reality due to the passionate and hard work of various stakeholder groups, such as the National Organization for Rare Diseases, along with the current chair of the advisory council, Marie Conley, and the late Craig Tucker with Life Sciences. The rare disease community also advocated strongly for the bill to be signed into law, which made a big impact.


By working together, we were able to make the council a reality.


Now one year in, the council, of which Joe Coyne is a member, has released a report about the issues they need to tackle and what steps need to be taken in order to make progress for those with a rare disease.


I also began talking with my colleagues in the House and Senate and started the Rare Disease Caucus which is currently headed by myself, Rep. Mark Longietti, D-Mercer and Sens. Judy Ward, R-Blair and John Blake, D-Lackawanna.  The caucus gives us an opportunity where both Republicans and Democrats can work together for a common cause and raise awareness about the rare disease community amongst our colleagues. During meetings we discuss ways in which state government can help these individuals, many of whom are children.


The council’s report is a culmination of countless hours of research, surveys and interviews. It will act as the basis for what the council will be focusing on moving forward, as well as what the Rare Disease Caucus can work on to address from a legislative perspective.


Research and development companies and drug developers in the Commonwealth that deal with rare diseases will also benefit from a relationship with the council. A collaboration with this business sector will help with the progress of treatment for those with rare diseases.


The creation of the Rare Disease Advisory Council is one of the most rewarding legislative endeavors of my career. I have met so many amazing people throughout the process. And being able to help people and be of service in some small way is why I ran for office in the first place. Seeing these people connect with one another and share their stories, as well as being able to put them in touch with potential resources that could help them, is an amazing feeling.


To top it off, the council is quickly becoming a national model that other states are working to emulate.


As we enter into 2020 and look toward the future, I am hopeful the council continues to make great strides. However, it is not my sole focus. I have also been working on legislation to bring greater transparency to how taxpayer dollars are being spent.


I have authored legislation in collaboration with Reps. Donna Oberlander, R-Clarion, and Sheryl Delozier, R-Cumberland, that would include information on the state’s PennWATCH website about settlements paid out as a result of an action taken by a Commonwealth agency employee.


The PennWATCH website was created for user-friendly monitoring of all state department and agency budget expenditures and investments. However, the current website does not include information on settlements paid based on action taken by a state employee, such as a sexual harassment settlement.


This legislation is the result of a Joint State Government Commission report on workplace harassment/sexual harassment settlements that were never disclosed to the public or the Legislature. Including this information on the website will provide residents with an easier way of monitoring where their taxpayer dollars are going.


House Bill 1803 was unanimously approved by both Democrats and Republicans in the House of Representatives and is currently before the state Senate for consideration. This is yet another example of how working together with other colleagues has strengthened the push to get this legislation through the legislative process.


When looking ahead to 2020, the Legislature as a whole will continue to look at legislation to improve health care for all Pennsylvanians, ensure greater educational opportunities for students, and look at ways to attract more businesses to the state.


Representative Marcy Toepel, R-Montgomery, serves as House Republican Caucus Chair. She represents the 147th House District.


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