I had the privilege of attending the National Organization for Rare Disorders (NORD) Gala on Tuesday evening. I say “privilege” for a number of reasons. First, I was invited as a guest of our client, Lundbeck, and had the opportunity to meet patients and advocates from the Huntington’s Disease Society of America and the Tuberous Sclerosis Alliance. Second, the event celebrated the 30th anniversary of the passage of the Orphan Drug Act and the founding of NORD. Abbey Meyers, who led both of these efforts, was honored, as well as the policymakers William Corr, Senator Nancy Kassebaum and Representative Henry Waxman.
The Orphan Drug Act has successfully stimulated interest in rare disease therapies. In the decade before 1983, only 10 new products for rare diseases had been developed by the pharmaceutical industry. Since then, there have been more than 400 orphan therapies and hundreds more are in the pipeline, according to NORD. Aegerion, NPS Pharmaceuticals, Onyx Pharmaceuticals and Sigma-Tau Pharmaceuticals were celebrated for their innovative therapies.
Advocates in training
The highlight of the evening was seeing “patient advocates in training” give awards to industry leaders. Megan Barron is a Duke University senior and a former White House intern. She also lives with dystrophic epidermolysis bullosa, which causes the skin to be so fragile that even the slightest friction can cause severe blistering—both inside and outside the body. There is currently no cure for dystrophic epidermolysis bullosa. Megan has been interviewed by ABC News, and she also wrote a blog for them. Megan and her family are active supporters of DebRA of America.
Brooke Foster is 11 years old and lives with mastocytosis, which is caused by too many of a certain type of cell known as “mast” cells in the body. Brooke has presented to the New Jersey state legislators and with her family is active in the Mastocytosis Society. Seven-year-old Ian Brown calls himself a “histio-warrior.” Ian has Langerhans cell histiocytosis, which is caused by overproduction of white blood cells known as histiocytes. Ian and his parents have spoken at Capitol Hill and are active fundraisers for the Histiocytosis Association of America. These three young advocates brought the crowd to its feet with their inspiring words.
Stephen Groft, PharmD, Marlene Haffner, MD, MPH, Lars-Uno Larsson and Jess Thoene, MD were all honored, and Frank Sasinowski was presented a lifetime achievement award by his son in a moving speech. Rare disease advocate Jack Klugman was also featured. It was an amazing evening honoring people who have made a true difference in the world. I’ll close with a quote from Abbey Meyers: “The Orphan Drug Act is truly a blessing for humanity.”
- 20 May 2013 at 11:05am
- Eileen O'Brien (@EileenOBrien)
NORD Gala highlights http://t.co/4z5j04q0z8 w/ @HDSA @tsalliance @DebRAofAmerica @tmsforacure @Histiocytosis @lundbeckUS ...