Rare Disease Week Update


2016 Rare Disease Legislative Advocacy Update

RDLADuring the first week of March Every Life Foundation hosted the 2016 Rare Disease Week on Capitol Hill. The week was packed with events that began with Rare Disease Day at the NIH followed by a documentary viewing of “Dusty’s Trail” and panel discussion. Day 2 was an extremely informative Legislative Conference and Advocacy Training. Day 3 the group was on Capitol Hill with scheduled meetings with our respective reps and senators. Day 4 was the Rare Disease Congressional Caucus and a Rare Artist Reception. There were 7 childhood cancer advocates that participated in these events. The most powerful takeaway from this trip is that it motivated us all to share how the Rare Disease efforts are very much in-line with the goals of the childhood cancer community and how they embrace our participation. We share all the same challenges and by definition all childhood cancer is designated as “Rare.” Further, some organizations object to straying off their talking points and legislative requests. RDLA has always encouraged groups with disease specific requests to include in their presentations. Our group was excited to include the STAR act in all our meetings.

NIHThe week began on Monday with Rare Disease Day at the NIH; this is always informative and offers unprecedented access to researchers, doctors and NIH/NCI staff. (the agenda can be found here) In the early evening there was a cocktail reception and a documentary screening. The documentary “Dusty’s Trail-Summit of Borneo” was a dustys-trail-key-artcompelling story about Duchenne Muscular Dystrophy which is a fatal and debilitating muscle wasting condition that affects one in 3,500 boys worldwide. The story tells about people coming together from around the world to climb a mountain in Borneo for charity to raise awareness for Duchenne. The panel was complete with the filmmaker as well as Dusty’s mom, Catherine. She is the driving force for the climb and the founder of Coalition Duchenne.

Day 2: The legislative conference was a full day of information, speakers, advocacy updates and training. This truly has to be experienced to achieve an understanding of how impactful this is. Considering the leaders of the breakout sessions there were 33 speakers throughout the day from a variety of different experiences and expertise. The speakers consisted of consultants, Capitol Hill staff, FDA staff, NIH Director (of the Office of Rare Disease Research), patient and parent advocates as well as a 16 year old social media guru.

Day 3: The requests for our day on Capitol Hill were all relevant to the childhood cancer community.

The Senate request advocates asked for co-sponsorship of the following bills:

*S.1421: Orphan Product Extensions Now (OPEN), Accelerating Cures and Treatments

*S.1597: The Patient-Focused Impact Assessment Act

The House request advocates asked for co-sponsorship of the following bills:

*H.R. 971: Orphan Product Extensions Now (OPEN), Accelerating Cures and Treatments

*H.R. 3731: Rare Disease Fund Act (RaD Fund)

*Advocates also urged the Senate and House Reps to join the bipartisan and bicameral Rare Disease Congressional Caucus.

StarActWhatisitSMALL_edited-2*The Childhood Cancer advocates were all on board with presenting H.R. 3381/S. 1883 the Childhood Cancer Survivorship, Treatment, Access and Research, STAR Act in both the House and Senate meetings as well as participating in some “drop offs” for targeted offices in their respective states.

Thursday: Rare Disease Caucus Briefing and Rare Artist Reception.

In closing, I would like everyone to understand that the Rare Disease community is an ally in our fight. Contrary to our communities’ beliefs statistically and by definition of the NIH all of childhood cancers are RARE.

Rare Disease as Defined by the NIH: Rare diseases, by definition, are diseases that affect fewer than 200,000 people in the United States. Yet, they present a significant health care concern since there are almost 7,000 rare diseases, affecting more than 25 million Americans and their families.

Rare diseases may involve chronic illness, disability, and often, premature death.

Rare diseases are complex, and often with inadequate or no treatment, thereby representing a disproportionate share of health care spending.

Patients with rare diseases are frequently misdiagnosed or are undiagnosed.

Few drug companies conduct research into rare diseases since it is difficult to recover the costs of developing treatments for small, geographically dispersed populations.

To advance medical research on rare diseases, a research network facilitates collaboration, enrollment in studies and trials, and sharing of data.

KEY FACT: Prevalence of ALL Leukemia: In 2012, there were an estimated 75,176 people (including adults) living with acute lymphocytic leukemia in the United States.

Let’s continue to engage and be a part of this Rare Disease community.

Author: Rob Whan

Related Articles:


“Rare is not a dirty word”





Dusty’s Trail


Sick Chicks


Open Act


Patient-Focus Act https://drive.google.com/file/d/0B-mzhw_KfGVmYlJqQ3MyeXdzb0E/view?usp=sharing

RaD Fund https://drive.google.com/file/d/0B-mzhw_KfGVmSXhLTUlzQXNpaVk/view?usp=sharing



Closing facts:




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