NORD 2013 – Washington DC

From October 6 to October 9, 2013, the U.S. organization for rare diseases, NORD, in collaboration with the American organization DIA, organized a conference in Washington DC.

Becky Gallis from the CMTC-OVM US organization and Lex van der Heijden from the global CMTC-OVM organization both took part in this conference.

During this period the U.S. Government was ‘shutdown’ (locked) which caused various speakers from the Food & Drug Administration (FDA) and National Institute of Health (NIH) to not be permitted to participate in the conference. This created a major impact on the conference due to approximately one-third of the presentations had to be reassigned to different speakers. Fortunately, those organizing the conference were able to absorb this impact and cover the vacant positions.

This was a special conference for NORD as they celebrate their 30th anniversary.

What struck us was the presence of relatively large pharmaceutical companies and that they seek cooperation with patient organizations. This move was also visible during the Eurordis conference in Brussels in 2012, and actually appears to be a continuation.

Becky Gallis, president of CMTC-OVM US, has been invited to participate in the annual DIA meeting in 2014 as they celebrate their 50th anniversary in San Diego, CA. We are very grateful for this invitation and also see this as a great opportunity for CMTC-OVM to create a better placement on the map of advocate organizations.

Lisa Phelps (NORD) had the honor of opening the pre-conference kickoff. Then it was the turn of Jennifer Webb-Adree from DIA to welcome us. Tai Spargo of NORD presented the work to the Rare Disease Day 2014, and notified us that social media (Twitter, Facebook, …) is the medium NORD uses to make business. Lex and Tai agreed that our social media specialist will be working under the Rare Disease Day with her. NORD has a ‘media coverage and press kit available on the NORD website for reuse by other patient organizations.
Jeff Lindeman, lawyer, gave a presentation on the U.S. patent law and intellectual property. This is a complex matter and in other countries it may have a different design. However, the message is in any case that we need to be extra alert to such things as an organization, and when there is something we plan to do it’s imperative we seek the advice of the correct lawyers within the country in which we conduct business.

The morning session’s main topic was the U.S. healthcare system and in particular the implementation of health insurance. The U.S. government has mandated that everyone is insured and if you’re not insured you will be fined.
The plenary morning session was devoted to the investment environment of rare drugs and devices. The pharmaceutical companies want to work closely with patient organizations in the field of drug development. After the plenary session, there were two parallel sessions. Becky and Lex chose to attend the session titled, “Listening to the Voice of the Patient.” Pharmaceutical companies need patients when they wish to try drugs. This saves the company a lot of time (and therefore money) and they help the patient in turn, for example, the declaration of expenses by their insurance.
The Genzyme company organizes meetings between patients and employees.

The morning began with a plenary session on “Natural History Studies”, this is to describe the natural course of a disease from the time immediately prior to its inception, progressing through its presymptomatic phase and different clinical stages, to the point where it has ended and the patient is either cured, chronically disabled or deceased without external intervention. This information is being used for drug development.

After this session we had the opportunity to visit the poster sessions. A poster session is basically a big vertical board on which posters can be hung from patient organizations and someone from the organization explains the specific disorder.

The afternoon sessions consisted of two parallel sessions which Becky and Lex selected the session on the collaboration between patients and industry for research funding. This was a series of presentations which most deeply emerged in medical or genetic backgrounds. Our expectation was that this session would have been focused on patient/industry communication and how it could be obtained. We expected more practical information.

The first session was a series of presentations on the health systems of the future. The second session was a panel discussion on cooperation between patients and industry in the field of rare diseases. The session was led by Pfizer.
It also brought forward that not every project results in success and how they learn from these situations. The intentions of each study is to ensure no one runs a financial risk. In contrast, that if nothing is done, the patient is at risk.
Delay before the start of ‘clinical trials’ takes an incredible amount of money and it is therefore of great importance that the patient organization concerned has people wishing to participate in the testing of the developed drugs available.

During the conference we monitored our own Twitter account and noted the number of “followers” had clearly increased. This is believed to be a direct link between the ‘follower’ and our presence at this conference.