RARE is not a dirty word

rare

Editor’s Note: No one in my childhood cancer community, including me, likes the word “rare” when it used to describe the disease. I met Rob and three others from the childhood cancer community at a three day RDLA event in Washington, DC. One of the speakers I listened to at the conference was a reporter from the Wall Street Journal and she spoke of a really, really rare cancer her mother had. She said only 7,000 people, yes that’s right, only 7,000 people per year were affected by the type of cancer from which her mother eventually died. At that moment, I realized my grandson’s neuroblastoma which affects 600 kids a year was in fact rare. My experience at the conference mirrored Rob’s which he very eloquently describes below. By  the way, at the conference we met Danielle Leach  from Inspire.Com and she suggested the title for this article.    Team Captain

rdd-logoThis was my second consecutive year at the Rare Disease Legislative Advocacy Days and the experience did not disappoint me. RDLA has the same challenges with rare populations as we do with childhood cancer, some of them being FDA reform, low or nonexistent funding and lack of drug development. The atmosphere at RDLA is very comfortable for the childhood cancer advocate. We are welcome  as part of the team and it seems that they recognize that our challenges are much in line with theirs. Statistically, the childhood cancer community fits within the scope of rare disease with the guideline being less than 200,000 diagnoses per year and includes 7000 other diseases. There is a lot that this group does well but there are a few in particular that the childhood cancer community can embrace and aspire to. The 2 things that I wish to highlight from the experience are education and access.

The schedule surrounding the RDLA week was a busy one. At some times choices were offered of topics at different venues to fit your interest. We attended as many as we could and the education that was offered through the sampanel discussions and presentations were unprecedented in my 6 years of advocacy experience. The opening night was a documentary on “progeria” that educated us about this disease that had no treatment. The documentary is a case study of how a single family rallied around this disease and pushed a drug through to market that is actually adding about 5 years to these kids life expectancy through drugs that improve their vascular strength.

After the documentary, the panel discussion opened with introductions, panelists included Dr. Francis Collins, Director, NIH. I am proud to announce that the 5 attendees from the childhood cancer cause in the audience lead the Q & A session with Dr. Collins and no one was holding anything back but we were all respectful at the same time.  I actually had a short conversation with him after the event and he was very receptive to our challenges and assured me that our advocacy efforts are necessary to get the changes we need for our patient population, for our kids.

rdla logoIn conclusion, I can attest that the first year I was a bit star-struck. This year I knew what to expect and was ready to engage. The experience that was led by RDLA is one that all serious advocates should attend to shape their advocacy tools and experience how another well organized cause does things. I encourage everyone to get involved with RDLA and they will open doors for our childhood cancer community.

Author: Rob Whan

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This entry was posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized and tagged , , , , . Bookmark the permalink.

One Response to RARE is not a dirty word

  1. Pingback: Rare Disease Week Update | Four-Square Clobbers Cancer

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