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Michelle and her daughter Lilly's Story

Meet Michelle and her daughter Lilly

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Michelle, a native and resident of Winston-Salem, lives with her husband Tim and their two daughters Lilly and Logan. Michelle holds a doctorate in Pharmacy and spent several years managing care administration as a clinical pharmacist. Later in her career, she worked within the pharmaceutical industry in medical and regulatory affairs, which led her to a role and interest in the regulatory process of drug development, which turned out to be very insightful when faced with Lilly’s diagnosis of Dravet Syndrome. In 2008, she started a business called MWelborn Group that provides consulting services to companies developing drugs for rare diseases. Outside of work, Michelle loves to kayak, read, and enjoys time at the beach and lake with her family.

Tell us about your daughter, Lilly.

Lilly was diagnosed with Dravet Syndrome when she was about 5 months old. The syndrome is a very rare condition due to a random gene mutation that causes drug resistant epilepsy and other symptoms such as intellectual disability, ataxia, and problems with thermoregulation. In 2005, there were only 500 cases in the world. She was born a healthy, typical child, and then symptoms of the disease manifested when she was around 5 months old. Right before she was diagnosed, I had serendipitously met a pediatric neurologist during vacation who had just done a presentation on Dravet Syndrome. Overall, Lilly is a very happy child. All parents have the same end goal for their children and with Lilly we had to adjust and adapt what our end goal would be for her- from a future standpoint and a career standpoint. But for both of my children the end goal is happiness and what I can say about Lilly and her personality is that she is already there. She is intellectually disabled, so she functions at around a three or four-year-old level. She doesn’t worry, and she doesn’t have any regrets. She lives for the moment. She has really taught us this valuable lesson on how we should live and reminds me of it every day. Her favorite TV show is Barney and her favorite thing to do is to go to Victory Junction Camp. She also likes to go fishing and swimming. Overall, I think she is just about the happiest person I know.

What was your most recent accomplishment involving the FDA’s approval?

In the past 8 months, I have been a part of two new drug applications. One of these new applications is for a drug called Epidiolex, which was approved on June 26th for Dravet Syndrome and Lennox-Gastaut Syndrome. That has been a big win for me. I am so glad to have been a part of the team that made it happen. In 2012, I was asked to speak at a Dravet Syndrome meeting and present available data on the use of cannabidiol to treat epilepsy and I ended the presentation with this: “Desperate times call for desperate measures.” Later, I spoke to a physician who was researching whether or not the drug compound cannabidiol was safe for young children and said, “I think I can convince the FDA to do an expanded access program for an intermediate size population of 25-50 patients in a few positions. You should see what it does in all patients and in all kids that have drug resistant epilepsy, and you will be able to see if it works to define your development plan.” The plan worked and became the largest expanded access program in the history of the FDA. This program included 1,200 patients who were given access to the compound. The goal was to see if there is some type of signal and we rigorously collected the data even if it was patient reported. We saw some syndromes, Dravet being one, that it seemed to really help. I was involved in the regulatory aspect of the drug development for the first two years and served as the liaison between the FDA and the company. It has really been very rewarding, and I am glad that there is now a drug approved and proud to have been a part of it.

What is your hope for clinical trials in the future?

I live in the rare disease world. Lots of changes have taken place getting drugs through the approval process quicker for rare diseases. My hope would be the FDA would come up with another option for people with rare diseases because time is highly important when you are dealing with a catastrophic condition.

What would you change about clinical trials?

I wish the FDA’s stance on how much data needs to be collected for very rare conditions would change. In rare diseases especially, it takes far too much for the FDA to be convinced that a drug is advantageous for a condition that the person is going to die from eventually without treatment.

When you hear the mission of Greater Gift and their work to celebrate clinical trial heroes, while giving back, what comes to mind?

I think that all people involved with Greater Gift, including the individuals in the organization itself, the patients, and the partners, are a self-less community of people who practice social responsibility on many levels.

Describe clinical trials in 3 words.

Selfless. Commitment. Opportunity.

Are you ready to help?