February News 2021: ED
Message from the Executive Director
Dear NNPDF Friends and Family,
Rare Disease Day is approaching. On February 28th, families from around the world join together as one voice. Throughout the month, Niemann-Pick families across the US sent photos in support of our “I Care About Rare” program and helped us amplify the Niemann-Pick voice.
Niemann-Pick is one of nearly 7,000 rare diseases. The path to diagnosis for a rare disease can be long, winding, and complicated with few specialists and supports. At the same time, the path to approval of new medications for rare diseases is complicated. We have less patients for clinical trials than a “typical” disease and traditional clinical trial designs may not fit the unique patient populations of rare diseases like Niemann-Pick.
NNPDF is advocating on behalf NPD families, to treat rare diseases differently from mainstream diseases with larger population. We are sharing our thoughts and concerns with the FDA in order to discuss challenges of clinical trials with small patient populations, to find solutions that may make approved treatments possible in the near future, and to increase the patient voice in decision making.
To help support our positions, we now have a vast array of data from our “Challenges of Access” Survey conducted in 2020. Preliminary findings were presented in a poster at WORLD Symposium and will also be shared in a summary report shortly. Thank you again to all of the families who participated in this survey. Your responses are helping us to better understand the health insurance needs and challenges families face in accessing care and services. The data we gathered will provide the foundation needed to discuss these challenges with key opinion leaders, like legislators and regulatory officials, and to provide a platform for our fight to ensure coverage for treatment options.
Last summer our Family Support & Medical Conference featured a keynote address by Dr. Kathleen, Acting Director, Division of Rare Diseases and Medical Genetics (DRDMG), Office of New Drugs, US Food & Drug Administration. This summer, our 2021 Family Conference will again take place as a virtual event. Our Scientific Advisory Board recommend a best way to safely gather our Niemann-Pick families for our annual conference is to hold a virtual conference this year. Our Board of Directors and staff take this recommendation, and the health of the Niemann-Pick community, seriously and support this guidance.
Our program will still be held July 29th – August 1st – starting with Family Advisory Working Groups on the morning of July 29th, and our conference beginning on the evening of July 29th. We are fortunate to host the INPDA Global conference in conjunction with ours and are excited to be joined by our international friends and families. Given the success of last year’s virtual conference, we know this year will be another interactive and engaging weekend filled with clinical & research updates and discussions, our unique family break-out sessions, and plenty of time to connect on our virtual conference platform throughout the weekend.
Registration will be available shortly. Watch for the announcement and link soon! In the meantime, don’t forget to download the NNPDF app for connecting, updates, info, contests, and more!
Joslyn Crowe, MSW, MA
NNPDF Executive Director
Supporting One Another. Supporting Our Community.