Our first Rare Disease Week in DC


At the 2016 MLD Family Conference a conversation with Dean led to a decision to attend the Rare Disease week.
It was a first visit for wife Nancy and I to Rare Disease week and a pleasure for us to assist daughter Michelle in attending this year.

Last year she had planned to attend but her daughter Emily had been admitted to Boston Children’s Hospital so Kim Brown had to pave the way and the great job she did encouraged us . Having visited DC all the way back to 1968 and many times since, I can conclude that big government is definitely in vogue.
Our first challenge was finding the right hotel and transportation and as always Dean came to the rescue. We stayed in Arlington Hilton Garden Inn based on one caveat, they gave us a reservation that could be cancelled at the last minute, no charge. Michelle was a suspect attendee based on her stroke and cancer recovery status but she was able to recover sufficient to make the trip. We used the metro to transfer from the hotel to meetings and soon were comfortable with travel inside the beltway. My recommendation would be to use Uber (what a great innovation in free and fair trade) to avoid any walking and searching for buildings in the rain.

We attended the RDLA Legislative Conference on Tuesday, which was a series of presentations related to rare disease issues apart from one political speech in support of the ACA which is an oxymoron. Most of the presentation related to current issues of interest to the advocacy community. The room was packed with attendees and the presentations were helpful in preparing us for the next day.

We met and heard some people from NC who had significant experience in advocacy and the group that we participated in helped navigate us through the halls of the senate and congress during our representative visits on Wednesday. We were prepared that we would not get to visit face to face with our representatives and that senior staffers were equally important.

I did see a number of senators in the basement as they departed the subway to the capitol building including Elizabeth Warren who smiled at Michelle as she was being pushed by me but had an MA Rare Disease badge on. My disappointment at not seeing any senator or congressman in our office visits was more than countered by the magnificent scene on Tuesday’s TV appearance of Megan Crowley* in POTUS’ message to a joint session of Congress and the first time ever Presidential recognition of Rare Disease Day!

In summary, it was a great experience and one we will repeat again next year, Lord willing. For many years I have lamented my lack of involvement in the MLD lobby. Dean and Teryn have carried the baton alone too long. This year was a manifold increase from Kim Brown’s sole visit last year. I hope that next year we will have an MLD hotel block rate to encourage participation.

Maybe Trump will attend 😊

 

Farewell, Adios, Ciao, Au Revoir, Sayonara and Zàijiàn,

Tony Hodgson for Michelle Hodgson Pierce and Nancy Hodgson

* Megan Crowley, her father John and the rest of her family were featured in the 2010 film, Extraordinary Measures.

openNHS Manifesto – Meeting Report

A meeting of researchers, clinicians, industry and academia was convened by the MLD Foundation on June 24th in Washington, DC to discuss the openNHS Manifesto we wrote about in this blog post.

Since a NHS is not a therapy, NHS participants have historically be giving time, energy, and effort, not to mention exposing their MLD loved ones to occasional invasive and potentially painful testing with limited feedback from the NHS study teams.

The openNHS Manifesto

  • recognizes the importance of NHS to better understand the disease and as a baseline to determine efficacy and obtain regulatory approval of new therapies.
  • calls for the NHS study team to be well-informed about MLD and to give back to the participants ideas and insight into improving the participants quality of life and ongoing clinical care.
  • calls for study sponsors to collaborate pre-clinically up front with other researchers and industry to design a study that meets the sponsor’s needs as well as  reasonably anticipated future needs
  • calls for study data to be open and accessible as raw data (in its entirety) to future researchers.  The Manifesto recognizes that some limited time protection may be necessary to honor publishing and IP rights.

At the DC meeting there was extensive discussion and sharing of perspectives and concerns about openNHS from many points of view.

We are pleased to report the meeting was a success on all fronts!  MLD Foundation, on behalf of those affected with MLD and the ongoing research community, was able to facilitate full support of the Manifesto and will be working with MLD collaborators in general, as well as Shire as sponsor of the current US late infantile NHS, to implement the Manifesto on current and future MLD Natural History Studies.

We look forward to sharing more specifics about what this means to MLD families and NHS study participants in the near future.

We will also be sharing our success with other advocacy groups with the hope that they too can call for openNHS in their communities.