As the super-contagious Delta variant of COVID-19 sweeps across the globe, driving up infection rates as well as deaths, top US health officials are urging everyone 12 and older — including the country’s 30 million or so rare disease patients — to get vaccinated quickly.
Some 185 million people have been infected, and nearly 4 million have died worldwide since the pandemic began in February 2020. About 57% of American adults have been fully vaccinated and do not need to wear masks, according to the Centers for Disease Control and Prevention (CDC).
But for those who haven’t yet gotten their shots, the risks are great, experts say. That’s why the National Organization for Rare Diseases (NORD) partnered with CDC and the US Food & Drug Administration (FDA) to host a June 16 webinar on COVID-19 that covered everything from vaccine effectiveness among immunocompromised people to the future need for booster shots.
Edward Neiland, MD, PhD, NORD’s chief medical and scientific officer, moderated the hour-long event, which was cosponsored by 3 patient advocacy groups: the ALS Association, the Cystic Fibrosis Foundation, and the Muscular Dystrophy Association.
“I recognize that it’s been a difficult path for those living with a rare disease, as they may be among the more vulnerable to COVID-19,” FDA Acting Commissioner Janet Woodcock, MD, said. “We’ve been working since the earliest days to advance a far-reaching response, putting enormous resources and tools to work in our search for solutions.”
‘An Exhaustive Review’
Dr. Woodcock assured the audience that the FDA conducted an exhaustive review before granting Emergency Use Authorization for the Pfizer-BioNTech and Moderna vaccines, both of which use mRNA technology.
“We looked at manufacturing and quality-control data as well as safety and effectiveness data,” she said. “This confidence is particularly important as we continue on a national vaccination campaign to get more protection. Each of you working with or living with rare diseases can play an important role in this regard. Your understanding of our process — which we have had for some time — can go a long way to helping boost confidence in the FDA and the products we authorize or approve.”
Joining Dr. Woodcock on the virtual panel were Peter Marks, MD, PhD, director of FDA’s Center for Biologics Evaluation and Research; Dr. Anne Schuchat, MD, the CDC’s principal deputy director; and Sara Oliver, MD, MSPH, who co-chairs the COVID-19 work group at the CDC’s Advisory Committee on Immunization Practices.
“No corners were cut in the development of these vaccines,” said Marks, explaining that the FDA merely eliminated the time normally spent between clinical trials; it also let companies scale up manufacturing before trials had finished, in order to get a jump-start on distribution.
“While it’s true that these mRNA vaccines were all developed in under a year, they were studied in clinical trials the size of those we normally use for approving vaccines,” he said. “Granted, we don’t have the normal 6-12 months of safety follow-up for these vaccines, but we do have the efficacy data and very good ongoing surveillance of those who participated in the trial, plus safety surveillance conducted by the CDC and by the FDA on those who have received the vaccines and now number in the hundreds of millions.”
On July 12, 2021, Israel became the first country in the world to offer a third vaccine dose against COVID-19, when its Ministry of Health announced that, effective immediately, health providers may now administer booster shots to all immunocompromised adults. The decision comes as the Delta variant spreads throughout Israel, even though more than 56% of the population is now fully vaccinated.
Questions on Comorbidities
Although the FDA doesn’t yet have data from studies done on vaccinated patients with diabetes and other comorbidities, said Marks, that shouldn’t prevent people from getting vaccinated.
“Studies are ongoing in immunocompromised populations, and some data on people undergoing chemotherapy for cancer,” he said. “There is some indication that there can be a decrease in the immune response [among such patients]. But while we wait for additional trials to be conducted, it’s still better to get vaccinated, because some protection is better than no protection.”
Dr. Schuchat said such individuals may take a longer time to recover from COVID-19 after being exposed to the virus.
“It may be that they don’t make as good an immune response. But we think vaccines are beneficial and safe for them, and that when others are vaccinated, the less likely they are to spread the virus to each of us,” the CDC official said, adding that “until we reach a better stage of reduced transmission, the idea of being exposed to lots of other people in crowds with poor ventilation might be risky.”
Dr. Schuchat also said it’s unclear when or if the US will reach herd immunity, thereby providing indirect protection to rare disease patients who fear the vaccines might not be effective for them.
Until then, she said, “the more people that get vaccinated, the less spread and risk of variants emerging. But it’s really a race to get as many vaccinated as possible. It’s incredible to me how highly effective these mRNA vaccines are in real-world use.”
Efficacy and Side Effects
Following their presentations, the experts fielded audience questions, including one on how patients will know whether or not their vaccinations actually worked.
“Some tests can say yes you have antibodies or no you don’t, but they’re not going to tell you accurately what that level of antibody is,” Dr. Marks said, urging people to just get vaccinated and take extra precautions until more is known. “The last thing we want to do is give you a false sense of security. It can be very confusing.”
Meanwhile, Dr. Woodcock said, the FDA does not recommend using any of the commercial tests now on the market to see if the vaccine worked or not.
The panel was also asked if patients with clotting disorders such as hemophilia are at greater risk of side effects after getting the vaccine. This follows reports of a rare adverse event called thrombosis with thrombocytopenia syndrome (TTS) in adult women younger than 50 who took the vaccine developed by Johnson & Johnson’s Janssen subsidiary.
“Right now, there’s no evidence to suggest you should not get vaccinated. In fact, all the evidence is quite to the contrary,” Dr. Marks said, explaining that 1 in 6 people who come down with COVID-19 develop some kind of blood clot — a much greater risk than the vaccine itself.
Another question involved potential injection site injuries, and whether patients with rare skin or muscle diseases might be able to receive the COVID-19 injection orally or through some other alternative method.
The short answer, said Marks, is no.
“Right now, these vaccines are being given with a very skinny intramuscular needle. People with bleeding disorders should let their vaccinators know, and hemophiliacs are told to take their factor replacement the day before the shot,” he said. “But needles should be perfectly fine even without factor replacement. There’s not a lot to be concerned about.”
He added: “Other methods are being looked at, but for this particular part of the pandemic, we’re not going to see those put into use. This is not the time to wait for a needle-free vaccine. Maybe in the future, but not in the near horizon. So for now, we’re stuck with needles.”