Talking with my Congressman about Rare Disease

About a month ago I reached out to my congressman's office hoping to talk to him concerning a serious drug shortage. Representative Burchett's office was very responsive. While I continue working on the one specific issue with his office and other advocacy groups, I have also scheduled an in person meeting with the Congressman for next week to discuss rare disease in broader terms for Tennessee and beyond.

When I first reached out to Rep. Burchett's office the aide who I spoke with was the person who communicates with the FDA. I spoke with her about the problem we are having with reduced production of our emergency injection of solu-cortef for adrenal insufficiency, In addition to setting up a phone call between me and the representative, she also immediately wrote the to FDA concerning this situation.

Within a few days I not only heard from Representative Burchett directly, but they also heard from the FDA concerning this issue. CDER (Center for Drug Evaluation and Research) is the part of the FDA that ensures safe and effective drugs are available in the United States responded to the aides inquiry. Their response was that they were aware that there was no medication currently available which scared me and that they were continuing to monitor the situation which should have production back to "typical" levels by June. According to the FDA website the recovery from this shortage will not be until the end of the year - that means that it will take that long for them to have on hand extra inventory. The FDA has also extended expiration dates on vials of this medication already produced (how does that happen?).

As for my conversation with the Representative, he said that he would write a letter and asked me for a draft so that the ask would be clearly stated. I am bringing that draft with me to the in person meeting I have scheduled with him for next week. I have stories from people directly affected and data to support the urgency of this issue.

As for the larger conversation, I want this problem with our emergency injection to highlight how a problem like this within the supply chain and the reality of how this affects the rare disease population. You see, in the rare disease community, especially with an invisible rare disease that has a treatment that does not cost a lot of money (therefore does not make a lot of money for the medical industry), it is easy for these issues to become invisible to the rest of the world. We need assurances from people in power that, though money talks, peoples' lives will not be put at risk by drug shortages based on financial decisions by large pharmaceutical companies. We need to know that when there are interruptions in the supply chain in the future that patients and caregivers have control in what happens, in the current situation our mercy was with our local pharmacies and doctors filling out and submitting more paperwork on our behalf. We need more options for medications that save lives, even if they do not have large profits - to have only one life saving medicine for an injectible steroid, which is actually used for many rare diseases could put many more lives at risk than many realize. That means that there may need to be incentives for companies to produce these.

I don't know the answers, but I know the problems. I know the questions. And next week I will be sitting with a United State Congressman who does have power to be part of these answers. I will tell him my story. I will tell him the problems. I will ask the questions. I will ask for him to be part of the answer. And I will continue to hold my politicians accountable. We vote for our leaders. We pay their salary. We need to work with them.