Being diagnosed with a rare disease like myelodysplastic syndromes (MDS) is scary, especially if you’ve never even heard of the disease. Sharing your experience with others who’ve been in a similar situation can be empowering. MDS patient Ray Malles, who’s lived with MDS for nearly a decade, shares his thoughts on why it’s important for patients to swap stories.
How long ago were you diagnosed with MDS?
I was diagnosed November 2006, when I was 76. We spent our winters in Florida, and my wife and I were told by our doctor to see a hematologist after some tests. When we approached the building, we saw that it said “Florida Cancer Institute” and just looked at each other. When the hematologist told me that I had MDS, I said, “What’s that?”
How did you react?
I’m the kind of person who doesn’t just do what others tell me. I wanted to know everything about my disease. I rolled up my sleeves, read articles and contacted the MDS Foundation. Somewhere around 2011, my doctors told me that my numbers were dropping and recommended a blood transfusion. I had learned enough about the complications associated with blood transfusions and told them that I didn’t want it. So we explored other treatment options that have been working just fine for me so far.
I don’t think that patients should feel like they are hostages. They shouldn’t put their heads in the sand and never question anything.
It seems like being an active participant in your treatment is important to you. Do you see that in other MDS patients as well?
There are all kinds of people that make up this world of ours. I don’t think that patients should feel like they are hostages. They shouldn’t put their heads in the sand and never question anything. I want to know what my disease is and what the side effects of my treatment are.
Are there other ways that you have made your voice heard?
I’ve produced a series of YouTube videos to educate people on MDS and have attended several MDS patient forums. I’ve become a very big patient advocate of those forums. After my diagnosis, my daughter discovered that one was taking place in Philadelphia, so all three of us—my daughter, my wife and I—participated. I’ve learned a lot from talking with other patients and have given presentations in my community. Patient-to-patient communication is very important for MDS patients, or any patient with a rare disease.
When you talk with other patients, what do you hope they take away?
Doctors sometimes throw these big words around, and that can be intimidating. Hopefully, I’m encouraging them to be an active participant in their treatment. Let me give you an example. My daughter works for a medical practice in North Carolina and has been in contact with two other people over the past 8 years with MDS. She reached out to them and asked if they would like to speak with me. I answered their questions from my perspective, and they were very appreciative.
Since the Orphan Drug Act of 1983, the U.S. Food and Drug Administration has approved over 500 treatments for rare diseases. What advice would you give to the FDA with regard to new treatments for rare diseases?
I once attended a session about the number of steps involved in the clinical trial and drug approval process. I know that the objective is to protect patient health and make sure the therapies are safe for patients. But 10 years to move a treatment from concept to market? There are a lot of patients who can benefit during that time. The FDA should do everything that they can to remove any unnecessary barriers to getting therapies approved as quickly as possible.
This article was originally published February 29, 2016.