You may be aware of the IPPF’s advocacy efforts. Now, we need you to advocate, too. Your story is important and shows that rare disease not only affects Americans, but Americans in your district. Your story proves to congressional members that their decisions have a great impact on human life and well-being.
As a NORD IAMRARE™ Registry Partner, the IPPF is proud to share that a new book, “The Power of Patients: Informing Our Understanding of Rare Diseases” was published today by the National Organization for Rare Disorders, Inc. (NORD) and Trio Health.
The IPPF had the pleasure of interviewing Lindsey Horan, AADR’s Assistant Director of Government Affairs about their 2018 Advocacy Day and important legislative issues.
For pemphigus and pemphigoid patients, there are several events that can trigger stress and exacerbate disease activity, even simply being diagnosed with a rare disease. The time it takes to be diagnosed, the medication itself, and how it all affects our families and friends can trigger stress and depression.
Although there has been a huge public outcry over the cost of prescription drugs, there has been little public discussion about the need for innovation for patients who don’t yet have an effective therapy. It is estimated that 1 in 10 Americans suffer from a rare disease, and only 5% of rare diseases have a treatment approved by the Food and Drug Administration (FDA).
In addition to our general request for NIH funding, I stressed the importance of cutting edge research on rare diseases and emphasized the significance of the Open Act, which incentivizes pharmaceutical companies to make treatments available off-label.
The purpose of advocating in Washington, DC is to spread awareness and lobby for favorable legislation that affects the entire IPPF community. At Rare Disease Week on Capitol Hill 2016, we had 7 advocates from the IPPF community.
In November 2014, I used the chemotherapy drug Rituxan off-label for my rare disease, Immune Thrombocytopenia (ITP). The decision was made after careful consideration of all the possible outcomes. I was desperate for relief since ITP causes internal bleeding that can be fatal. This thought stayed in the back of my mind as I watched my platelets drop and my bleeding episodes increase in severity.
The First Amendment to the United States Constitution guarantees the right of all citizens to communicate with their elected officials. So you might be asking yourself, “Does my Congressional Representative really care what I think”? Well, the answer is YES!
When the email landed in my inbox last month asking for pemphigus and pemphigoid patients and caregivers to advocate on Capitol Hill, I didn’t hesitate.