Translate this page:
NORD Webinar: COVID-19 Vaccine Update: FDA and CDC Leaders Address the Rare Community
January 15, 2021
- Stephen M. Hahn, MD Commissioner of Food and Drugs, US Food and Drug Administration (FDA)
- Peter Marks, MD, PhD Director, Center for Biologics Evaluation and Research, US Food and Drug Administration (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 Centers for Disease Control and Prevention
NOTE: The text below is not a full transcript of the webinar. It includes highlights from the conversation that includes rare disease information that may not be as readily available on the CDC, NIH, etc. websites. The text comes from closed captioning, so there may be typos or incomplete text.
“… we will get asked and we often get asked and we will get asked, what about the vaccine with folks who have one disease versus another? Of course, it is really hard to study vaccine efficacy and safety in one rare disease, because we just don’t have enough people with those diseases to come to significant conclusions. But there are really important parts of the data that we review that can help us potentially answer some of the questions that you ask. It is why our thorough review of the data and why our conclusions we feel are so important and why we think that backs up the gold standard that FDA has come to be known around the world.” – Dr. Stephen Hahn, Commissioner of Food and Drugs, FDA
“Emergency use authorization is something that we can use for products that are meant to address biologic threats such as we are right now in a public health emergency. At the emergency use authorization allows us to take a product that normally would have to go through a pretty long approval process and get it out there usually with less evidence. In this case for vaccines, because vaccines are being given to so many people and because safety is so important, we in our guidance that we put out in June and October of this year made it clear that we were going to have a standard that was very similar in many ways to that which we would use for a vaccine that was approved through our normal pathway, the Biologics license application.
Where we are now is we have to vaccines that have met the characteristics that we set forth in our guidance that have come through and have received emergency use authorization. They have clear and compelling evidence that they are safe and effective. They were studied in large [trials], one in 44000 patients, the other in 30000 patients, approximately randomized trials. Both of these COVID-19 vaccinations had efficacy in the 94% to 95%. That is very good effectiveness. That kind of efficacy is not the usual. For instance, influenza vaccine at its best usually has an efficacy of 70%, and that’s usually against only one of the components in the vaccine. It’s not against all of them. That is a very good number. What is even more impressive with this vaccine is that it was — it had that 94, 95% effectiveness in various different subgroups, including different ethnic and minority groups and also had very similar efficacy in older individuals, people over the age of 65.
The vaccine was effective. The safety profile was quite reasonable. There were some local discomfort associated with the vaccine, particularly after the second dose. There is some short-term systemic effects that are reported by people, but reported frequently, including achiness of the joints or the muscles, a small number of people had fevers, but these things generally went away within a day or two. Probably the most notable thing that you may have heard about if there have been some severe allergic reactions, and those allergic reactions may be related to one of the components of the vaccine. We in conjunction with CDC, our colleagues are looking into this further. Right now it looks like those allergic reactions are happening on the order of one in 100,000 individuals or fewer. They have been treatable. We have actually required as part of the FDA labeling that anyplace that is giving the vaccine has to be able to treat such allergic reactions. We are pretty comfortable that the vaccine is safe and effective. ….
The good news is that there are other vaccines on the horizon. We expect in the next month or two to hear information about possibly a one dose vaccine and there are others in phase three trials as well.” – Dr. Peter Marks, Director, Center for Biologics Evaluation and Research, FDA
“It has been on my mind throughout this process that these groups that we think about with underlying medical conditions, it feels like persons with living with rare medical conditions do not see themselves in this group. There is a CDC list of conditions that we’ve identified to put persons at increased risk, but I want to reassure you that that does not mean that we are leaving behind people who are not on that list. In our guidance around who is considered at risk for COVID and should be vaccinated, we include both the CDC list where we have evidence to support that persons with these very common medical conditions may be at increased risk, but right below that we have language to say that we are unable to develop — we don’t have the evidence to support but we know that many rare conditions put individuals at increased risk for severe outcomes from COVID. We do want to make sure that all of you know that you are absolutely in that group, really any medical condition where a person is either medically fragile, has any diminished ability in terms of their lung function, in terms of neuromuscular function, to fight disease, particularly respiratory disease. All of those individuals should be considered for early vaccination.” – Dr. Amanda Cohn, Chief Medical Officer, Office of Vaccine Policy, Preparedness and Global Health, Office of the Director, NCIRD, CDC
Question: If I get the vaccine, can I still spread COVID to others?
“The real big question here is whether these vaccines will interrupt transmission from a symptomatically to individuals, and we don’t know that yet for sure. We know from animal studies that they appear to do that, but until we have more data from human studies in real-world evidence and for some other studies that are planned, we do not know that for sure. That’s the reason why we are telling people, even if you get vaccinated, you continue to wear your mask, social distance, and wash her hands. We do not know whether it will control the spread yet.” – Dr. Marks
Question: Are there any large patient groups or conditions where you are seeing that there are true contraindications for those patients who just should not get the vaccine at all?
“Amanda can probably comment more on this, but from our perspective, really the only people that we would say definitely should not get the vaccine or people who have had a known allergic reaction to any of the vaccine components. Obviously, if somebody has had an allergic reaction to a previous vaccine but does not know they are allergic to any of these, they need to tell their healthcare provider. The CDC has put up guidance about how to watch people after they get the vaccine if they’ve had previous allergic reactions. You should tell your healthcare provider. Aside from being allergic to one of the actual components of the vaccine, there is no absolute contraindication to not getting the vaccine. Probably if you are sick with COVID-19, we would not recommend you get the vaccine until after you work better, but that’s pretty much the party line. Amanda probably can amplify on that if I have missed something.” – Dr. Marks
“The only thing I will add to what Dr. Marks has said is that I know the vaccine is being given in different places, so you are unlikely to be getting your vaccine right now from your regular doctor, but we have also put out clear and robust guidance about all of the administration sites, but in a very rare case you do have an allergic reaction, all of the administration sites have the capabilities to manage allergic reactions. At all of the administration sites, everyone has watched for a certain amount of time, if you have history of allergic reactions, severe allergic reactions, we recommend waiting 30 minutes after vaccination at the sites so that if you do have any symptoms it can be treated right away.” – Dr. Cohn
Question: 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 ability to safely get these vaccines?
“The issue is we did not have enough people with rare diseases that were entered into the studies and studied in any systematic way. There were only some people who had immunocompromised were entered into the studies. Also where we are and I think our feeling at FDA here is that this is not a choice that one needs to make with an individual provider. We are not concerned, particularly, with these vaccines that there is going to be harmed from giving it to somebody who is immunocompromised or who might have another condition for the question is, will it be as effective? We simply don’t know. That being said, I guess just in terms of practical advice, even if there is some decrement in efficacy, which we don’t know there will be the risk here in the setting of COVID-19, and particularly people with respiratory illness or any kind of respiratory compromise or diabetes can be very, very severe. The benefit may outweigh the risks. It is want to have with your individual provider to understand at least from the FDA point, we do not feel like there is a safety concern. It’s a matter of really understanding whether you understand that it may not be quite as effective. Again, we don’t know that. It could be, but we just don’t know, so it’s an individual discussion.” – Dr. Marks
Question: Do you have recommendations of one of the vaccines over the other for the rare disease community?
“Both. Whichever you can get a hold of. You know, for all intents and purposes, although, yes, the side effect profile might be slightly different, for all intents and purposes, these are very similar vaccines. The schedule is very similar. Three weeks versus four weeks. We have now seen allergic reactions with both of them, and I cannot say one is better than the other, so I think right now whatever you can get in your arm in a timely manner, that’s the vaccine that is the best.” – Dr. Marks
“We do recommend that you get the same product for the second dose once you’ve gotten the first dose, but you should also have, hopefully, when you get the first dose you will come away with them and an appointment for the second dose, so that should be taken care of.” – Dr. Cohn
Question: on the issue of the interaction of gene therapies with these COVID-19 vaccines. Many in the rare disease community are hoping to access and the not so distant future. Is there any concern that these mRNA vaccines may cause antibodies to develop that could interfere with future or current access to gene therapy is?
“Great question, and we have answered it before, so I can answer it here. These are mRNA package essentially in a soap bubble. The soap bubble is not like the virus is, and associated virus that is being used for directly administering gene therapy, nor is it like any of the other vectors that are being used. There is the likelihood of something happening here where one of these vaccines would preclude you from getting a gene therapy is pretty much nonexistent, so there should be no concern in terms of getting this vaccine and having it preclude you from getting a gene therapy. I’m just going to answer another question at the same time. If you are on a gene therapy trial, you should check with your provider, because these are considered unapproved products because they are under emergency use authorization, they are not investigational products. Many protocols will let one get vaccinated unless there is a specific reason why you shouldn’t. That could be I guess in the protocol, but there should be no reason why if you are on a gene therapy protocol you shouldn’t be able to get vaccinated, but just check with your provider in that regard.” – Dr. Marks
Question: As you all know, many rare disease patients are under 16. What do we know about the use of these vaccines in kids under 16? Are pediatric clinical trials underway, and when can those under 16 expect to have access?
“Thanks, great question for you we have a little data but the vaccine in 12 to 15-year-olds where the vaccine appeared to be safe, but it was not a very large data set. They are now going back and trials will be underway for both of the vaccines as well as for future vaccines in the pediatric age range. It will start with ages 12 through 15, once they make sure they are safe and those age range, they will de-escalate seven through 12-year-olds. We just want to make sure that each time — we are not so much worried about getting an effective the signal here, because we are going to look for antibody formations. So we will look at what are immune correlates of protection in children. Or at least antibody responses to start. What we need to make sure is that there is safety and that we don’t see any kind of inflammatory syndromes to be given the vaccine and vice versa. Some people said, it’s good enough for adults, why can’t you just half the dose and give it to children? We just can’t do that, because we have to find the right dose and we need to make sure it is safe. But it’s probably in a crystal ball, we are probably a couple months away from being able to have it administered in 12 to 15-year-olds and probably I hope maybe by summertime we will have data so that it can be administered to younger children, although that is still — I have to say that will have a lot of wiggle room, depending on how things go.” – Dr. Marks
Question: How can an individual, physician, advocate for a member of the rare disease community, whether it be a patient or caregiver to get access in these earlier groups in those states.
“So I think there has been a lot of guidance over the last week. We are continuing to reiterate from the CDC that we don’t want these recommendations to be so rigid that, a, you are not getting vaccines that are observed or, the not vaccinating people who, clearly, should be vaccinated early. I think this messaging will help. That’s new messaging this week as we started to realize there was a real difference in flexibility that states were allowing. Honestly, I am really also hopeful that in the next couple of weeks there will be this rush with the older age groups, this demand that has been created by opening up to older age groups and it will even out a little bit in that, you know, if somebody can’t get access to it this week, I would recommend that they circle back around next week in the week after. I do think that things will shift over time.” – Dr. Cohn