There are many patient advocacy organizations. Most are more interested in getting a "seat at the table" of the Washington policy community. A few, like Everylife Foundation for Rare Diseases, are focused on removing the barriers to faster and broader access. The group was the leading voice for reforms that accelerate the development of drugs for rare disease.. You can join it's movement for medical progress by voting for a Rare Voice Award honoree. Here's a link to the nomination page: http://rarevoiceawards.org/nominate/
The number of deserving awardess are growing. Just nominating someone can shine a light on their efforts.