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December 21, 2016 by Robert Ostrea

The Fight Never Ends for Rare Disease Advocates

By Robert Ostrea

As an involuntary member of the rare disease community (my youngest daughter, Hannah, was diagnosed with a rare, genetic disease shortly after birth), I have been fairly active in participating in events and organizations geared towards raising awareness of as well as educating people in positions of power (elected officials) about the importance of funding for rare disease research. The recent passing of the 21st Century Cures Act (Cures) was a huge milestone for those of us advocating for increased funding for the National Institutes of Health (NIH). This is the agency where a lot of programs geared towards finding treatments and cures for all diseases, rare and common, are taking place. Each year during the last week of February, for Rare Disease Week, a large group of rare disease advocates comprised of patients, parents and supporters, descend upon Capitol Hill to meet with members of Congress to share our individual stories and encourage continued support for legislation, such as Cures. I’ve participated the last three years and it was satisfying to see this significant piece of legislation pass the House, then the Senate to finally have the President sign the bill into law.

While I was happy that the bill passed, I was disappointed that there was not one single mention of how important this bill was to the rare disease community. The presidential signing ceremony began with an introduction of someone whose daughter was affected by an opioid addiction, then the President began his remarks to discuss Vice President Biden’s son’s untimely death due to cancer and how this legislation would help people affected by these conditions. While I can certainly appreciate that the opioid problem and the fight against cancer are extremely important issues which require attention, this bill also provides an important source of hope for those who are suffering from a rare disease. It would have been nice to have this mentioned during the ceremony but we will certainly revel in the signing of the bill!

The increased funding this bill designates for NIH research will benefit both rare disease patients and those suffering from more common diseases and conditions. My daughter’s condition, Gaucher Type 2/3, is a lysosomal storage disease with a strong link to Parkinson’s. Similarly, a related lysosomal disease called Niemann-Pick Type C is closely related to Alzheimer’s Disease. Current research on these rare diseases have yielded a tremendous amount of information which could lead to better treatments and perhaps a cure for Parkinson’s and Alzheimer’s. The same is true for many of the over 7,000 rare diseases known today.

The next step for rare disease advocates is to encourage Congress to consider passing the OPEN Act, which will promote the repurposing of current major market drugs to treat rare conditions. This will be the primary bill I introduce to my legislators this coming February when our rare advocacy groups gather again in Washington, D.C. for Rare Disease Week.  To learn more about the events and activities surrounding Rare Disease Week in Washington, D.C., visit RDLA’s website http://rareadvocates.org/rdw/.

Robert Ostrea is the President of the Little Miss Hannah Foundation, a 501(c)3, non-profit organization which was created to honor his 3-year-old daughter, Hannah, who lost her battle to Gaucher Type 2/3, a rare metabolic genetic disorder.

Filed Under: Blog, LMHF News Tagged With: 21st Century Cures, Advocacy, Legislation, OPEN Act, World Rare Disease Day

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