NORD Hosts an Interactive Webinar Drawing More than 7,000 Registrants

While the COVID-19 pandemic has been socially and economically devastating for us all, the rare-disease community faces a unique set of challenges.

Seeking to address these challenges, the National Organization for Rare Disorders (NORD) leveraged Conduit – PlatformQ Health’s proprietary engagement platform – to host the webinar, COVID-19 Vaccines Update: FDA and CDC Leaders Address the Rare Disease Community

NORD, along with the ALS Association, Cystic Fibrosis Foundation, and Muscular Dystrophy Association, produced this special webinar featuring leaders from the US Food and Drug Administration (FDA) and the Centers for Disease Control and Prevention (CDC). The panel discussed the COVID-19 vaccines, as well as the recently granted Emergency Use Authorization (EUA) for the Pfizer and Moderna COVID-19 vaccines.

The live, interactive program was held on Friday, January 15 and featured: 

  • Stephen M. Hahn, MD, Commissioner of Food and Drugs, FDA
  • Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and Research, FDA
  • Amanda Cohn, MD, Chief Medical Officer, National Center for Immunization and Respiratory Diseases, Chief Medical Officer, COVID-19 Vaccine Task Force, CAPT, US Public Health Service, CDC
  • Peter Saltonstall, President and Chief Executive Officer, NORD
  • Rachel Sher, Vice President, Policy and Regulatory Affairs, NORD

Providing Guidance for the Rare-Disease Community

Dr. Hahn reassured the community that although it is difficult to study vaccine safety in one rare disease, because not enough people with those diseases have been vaccinated, there is important data that can potentially help answer many of the questions the community may have. He also expressed the importance of being transparent about the authorization process and, in doing so, he hopes that he can instill complete confidence.

 “We are working tirelessly and diligently to get vaccines in as many arms as possible, but also trying to prioritize vaccines to ensure that people most at risk from severe outcomes from COVID-19 get vaccinated early,” Dr. Hahn said. 

“[After older adults and health care professionals], the next largest group is individuals with underlying health conditions. I know that many of you are in that category. There is a CDC list of conditions that we have identified to put persons at increased risk, but I want to reassure you that that does not mean that we are leaving behind people that are not on that list.”

Answering the Community’s Top Questions

A large amount of time was given over to questions from the audience.

A. What information is there about these vaccines with respect to patients with rare diseases? What do we know about individuals with rare diseases and their abilities to get the vaccines?

Q. “The issue is we did not have enough people with rare diseases that were entered into these studies and studied in any systematic way,” said Dr. Marks. “And there were only some people that were immuno-compromised that were entered in the studies. Our recommendation at the FDA is that this is a choice people should make with their individual providers. We are not concerned particularly with these MRNA-based vaccines that there is going to be harm from giving it to someone who is immunocompromised or might have another condition. The question is will it be as effective and we simply do not know. In terms of practical advice, even if there is some decrement in efficacy, which we do not know there will be, the benefit risk here for COVID-19, particularly for people with respiratory illness or respiratory compromise or diabetes, etc., can be very severe. The benefit may outweigh the risk. We don’t feel as if there is a safety concern, but the questions are on its effectiveness.”

A. Do you have recommendations for which vaccine is best for the rare disease community?

Q. “Both! Whichever you can get a hold of,” said Dr. Marks. “For all intents and purposes, these are very similar vaccines. The schedule is very similar, 3 weeks vs 4 weeks. One can say we have seen allergic reactions to both of them and I cannot say one is better than the other. For right now, I can say whichever you can get in your arm the fastest is the best one.”

A. Some in the rare-disease community may be unable to take the vaccine in the injectable form, are there additional dosage forms or types of administration that are under development for these vaccines or any others in the future?

Q. “You would have to have a real conversation with your provider because giving these subcutaneously is something that has not been studied,” said Dr. Marks. “On the other hand, there are additional vaccines under development which can potentially be given subcutaneously. With a few months’ time there should be more information about giving these vaccines subcutaneously.”

The webinar is available on-demand on NORD’s website. 

An Ongoing Partnership

In 2019, PlatformQ Health and NORD established a partnership to provide online continuing medical education for clinicians, as well as tailored education for patients and caregivers. Together, NORD and PlatformQ Health deliver resources to health care professionals, patients, and families to facilitate timely diagnoses and improved care for people with rare disorders. In addition, PlatformQ Health has leveraged its proprietary learning-engagement platform, Conduit, to aid NORD in their virtual conferences, webinars, and digital programming.

The PlatformQ Health Virtual Engagement Solution

With 10 years of experience producing, hosting, and delivering virtual events to HCPs and patients, we are experts in driving online engagement and have developed a customizable platform for our partners to create meaningful educational experiences online.

  • Customizable platform leveraging the partner’s brand, content, and user-friendly layout
  • Product and tech support, including live help for attendees, gated access solutions, hosting services, and best-practice guidance
  • Capability to simulcast content across social-media platforms such as Facebook Live, Twitter, and YouTube Live·       
  • One-on-one and group training with production team for speakers and staff
  • Live engagement hooks, including live Q&A, polling, and chatting
  •  Integrated learning management system, allowing you to track the effectiveness of your programs and participant satisfaction  
  • Audience generation, marketing support, and syndication opportunities to increase attendance and improve the impact of education    
  • Sponsor solutions, including tiered listings and individual customizable pages

Have questions about this webinar or our platform? Email us at  

PlatformQ Health partners with leading health care experts and patient advocacy organizations to produce digital education – including tethered programming for both clinicians and patients – across a range of health topics.