NORD 2022 – Washington DC

On October 17 and 18, 2022, the US National Organization for Rare Disorders (NORD®) hosted the Conference on Rare Diseases and Orphan Drugs. More than 830 enthusiastic attendees from around the world gathered to address and take action on the opportunities and challenges facing the rare disease community.
Items on the agenda included accelerated approval, newborn screening and fair access to care.

A special session was organized for the Patient Leaders on Sunday afternoon. We were divided into groups and a specific topic was discussed per table. Lex took part in the discussion about ‘good governance and board management’.

The conference on Monday and Tuesday mainly consisted of panel discussions. The panels consisted of a wide variety of participants from different backgrounds such as patient organizations, the US Food & Drug Administration (FDA), pharmaceutical companies, National Institute of Health (NIH) and NORD.

The photo album below shows a number of slides from presentations. The FDA gave a presentation on, among other things, the medication alpelisib in patients with PROS (PIK3CA Related Overgrowth Spectrum).

Highlights (link to NORD website).

During this congress we also had the opportunity to set up a booth. We have made full use of this!
Our booth was in the room where not only breakfast was served, but also the coffee break and lunch. As a result, we had a lot of visitors, also because our booth stood out because of the many colors.

In addition to the usual stroopwafels on the table, we also brought a series of clog slippers. This concept turned out to work well.

Given our previous experiences, especially with the parent support guide (which was often ‘sold out’ in a day’s time), we had a sheet of paper with, among other things, a link to a special page on our website where all leaflets and booklets can be ordered directly. This turned out to work fine.