The MD-CARE Act was signed into law in 2001 and reauthorized in 2008. What’s that got to do with a clinic survey you might ask? The MD-CARE Act was a pivotal moment. In a certain way, it was nearly as important as the identification of the dystrophin gene. The hearing was the first time Duchenne was said out loud in Congress. Duchenne was on the NIH radar, as well as, CDC. What you might not know about are all the meetings leading up to the passage of that legislation.
One meeting was with the CDC. It was pretty clear we had NO natural history of Duchenne, other than a study from many years earlier when steroids were not recommended. We had NO information about clinics, only rumors of clinics that had expertise in Duchenne and others less or none. We had NO data and without data, clinical trials are likely to fail (comparing apples to oranges because nothing was standardized) and industry trials only a wish.
We walked into that meeting with the Cystic Fibrosis data in hand: rigorous data published annually. Looking at the data we understood optimal care for CF, we understood what the ‘optimal’ clinics looked like in terms of available resources, numbers of patients, and interventions. We knew about the lives of the CF patients, the quality, life span, education, and employment. We knew we needed data to learn about individuals with Duchenne, where care was provided, what care looked like, and what resources were available. And in response to that need, DuchenneConnect was developed. Today, we have more than 2,000 patients registered internationally. We are working globally with TREAT-NMD and closely with Industry.
The clinic survey is structured to have a clear view about care, about available resources, and to understand your view about what is needed and wanted to improve efficiency. Simple as that. And this will help all of us – patients and families, physicians, healthcare professionals, biotech, and pharma improve healthcare delivery, identify sites for trials, understand your needs, improve interventions, develop clinical studies and trials, and reach our goal…to end Duchenne.
Participation helps all of us.