More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports.

These findings highlight the need for a greater understanding of the U.S. Food and Drug Administration’s (FDA’s) emergency use authorization (EUA) process, as all current U.S. vaccines against this virus came via this pathway.

People with rare diseases are often at a higher risk of poorer outcomes with COVID-19, and generally advised to be vaccinated.

“As we move towards accelerated vaccination efforts in the US, it is important to understand the needs of the rare disease community,” the EveryLife Foundation for Rare Diseases states in its report, noting that approval is “only one consideration out of many important factors” in deciding for or against vaccination.

To understand these considerations, the foundation and the Community Congress COVID-19 Response Working Group held a survey into patient and caregiver perceptions and information needs regarding a future COVID-19 vaccine.  The COVID-19 working group, convened by the foundation, includes representatives from patient advocacy organizations, healthcare professionals, and members of the biopharmaceutical industry.

The survey — conducted from Oct. 20 to Nov. 4, 2020 — posed four questions:

  1. Does the FDA pathway for vaccine approval affect a patient or caregiver’s decision to get vaccinated?
  2. Do individuals in the rare disease community make this decision based on differing factors?
  3. Might a vaccination decision differ depending on the types of information sources available?
  4. Does being a caregiver to a rare disease patient affect a person’s decision to get a COVID vaccine?

Among the 1,479 people who responded to the survey, 62% had a rare disease, 34% were caregivers to children or adults, and 4% were both patients and caregivers.

Nearly a third of the respondents (31.93%) were people with autoimmune diseases, followed by those with neurologic disorders (19.73%), and neurodegenerative diseases (19.58%).

Most patients took measures to reduce the likelihood of being infected with SARS-CoV-2, the virus that causes COVID-19, including changing their behavior, restricting in-person contact with those outside their household, and canceling travel plans.

Despite these efforts, 2% of respondents had been diagnosed with COVD-19, and almost 4% reported having disease symptoms but were not tested to confirm infection.

Most survey participants (62%) reported usually following Centers for Disease Control and Prevention (CDC) recommendations regarding vaccinations, while for 22% compliance depended on the specific type of vaccine, and 12% preferred not to typically have recommended vaccinations.

FDA authorization of COVID-19 vaccines under the EUA route, however, was more troubling across respondents.

Patients were six times more likely to get vaccinated if approval came through the traditional and often years-long process, the survey found, rather  than under emergency use. As its name implies, EUA aims to shorten the testing and review given to a vaccine’s authorization — or that of a medicine or device — to allow an emergency, like the COVID-19 pandemic, to be addressed.

Emergency use authorization is based on a final or interim analysis of Phase 3 trial data in thousands of patients, the FDA states on a webpage, and includes safety data on “well over 3,000” fully vaccinated people followed for at least one month for “serious adverse events and adverse events of special interest.”

A near majority of the patients — 47.5% — were “very likely” to get a vaccine if it went through a regular approval, while 7.9% were equally likely with an EUA. Similar opinions were expressed by caregivers to children with rare diseases (47.9% very likely under traditional route, and about 10.3% under an EUA), and those caring for adults (55.1% traditional, about 10.3% EUA).

More than half of the patients responding — 55.1% — remained undecided on future vaccines arriving via an EUA, while 41.1% were similarly indecisive regarding vaccines following a traditional path to approval.

Among patients who also caregivers, a majority — 58.8% — were “very unlikely” to get a COVID-19 vaccine that came under an EUA. But they were also, by the largest majority voiced (about 55.6%), “very likely” to get a vaccine that went through a traditional route.

Little difference, in contrast, was seen in factors of importance to patients and caregivers in deciding to be vaccinated, with safety and efficacy in clinical trials and later, real-world use ranking at the top of their considerations. The number of required doses and out-of-pocket costs were consistently among factors of least importance.

For many, knowledge of a vaccine’s safety profile for their specific rare disease or age group was also an important factor.

Primary care physicians or specialists, along with the CDC and patient advocacy organizations were the major sources of “trusted information” used by patients in deciding to be vaccinated. Social media was the least favored source among respondents.

Being a caregiver also meant preferring to get a COVID vaccine. According to survey data, 52.4% of adult caregivers and 50% of those caring for children said they were “more likely to be vaccinated early” due to their status.

What’s needed now, the foundation said, is to use this information to help “the rare disease community navigate the [vaccination] process.” This especially means educating the community about vaccine safety and effectiveness — the two factors weighing most in vaccine decisions, and encouraging patient-doctor discussions about vaccinations, as a doctor’s opinion was of prime importance among those who were undecided.

Given the considerably hesitancy expressed with vaccines authorized through emergency use, “it is important to continue our efforts to understand and address the concerns that were articulated in the survey and in follow-up community engagements,” the foundation stated.

In February, the group sent a letter to the Biden Administration outlining the concerns of the rare disease community regarding COVID-19 vaccines, particularly regarding the need to know more about the EUA process and the safety and effectiveness of vaccines allowed through it, drafting clear templates for state and local health agencies to use in outreach efforts, and ensuring safe and accessible vaccination sites.

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