Regulatory Focus™ > News Articles > 2020 > 11 > EMA sets 50% efficacy goal – with flexibility – for COVID vaccines

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Posted 19 November 2020 | By Kari Oakes 

EMA sets 50% efficacy goal – with flexibility – for COVID vaccines

3035 The European Medicines Agency (EMA) has released its planned framework for issuing marketing authorizations for vaccines against COVID-19, as the agency continues its rolling reviews of candidate vaccines. Rather than setting hard-and-fast efficacy standards, the guidance lays out EMA’s expectations, while allowing room for a balanced evaluation for conditional marketing authorization of a vaccine during the pandemic.
In terms of efficacy, EMA is expecting to see at least one “well-designed large-scale phase 3 efficacy trial” to support marketing authorization of a COVID-19 vaccine. For pivotal trials, laboratory-confirmed COVID-19 disease of any severity is the primary endpoint, with efficacy analysis limited to those participants who were seronegative at the time of their baseline assessment “as it is important to show that the vaccine protects subjects not likely to have been exposed to the virus before.”
Trials should be powered to provide “a convincing demonstration of efficacy,” said EMA. In specific terms, sponsors should look for a point estimate of vaccine efficacy of at least 50%, with the lower bound of a 95% confidence set above 20%, “and preferably above 30%.” Trials of vaccines with favorable safety profiles that hit these numbers “would support a regulatory decision,” said the agency.
However, recognizing that that the actual lower bound of the confidence interval for an effective vaccine may fall between zero and 20%, EMA said that its evaluation will balance the actual point estimate of vaccine efficacy, the point estimate’s precision and the safety profile of the vaccine. “Therefore,” said the agency, “if the actual results show that the vaccine is superior to the control group, i.e. the vaccine is efficacious, vaccine developers are encouraged to discuss the data with the EMA.” (RELATED: Marks sheds more light on COVID-19 vaccine EUA guidance, Regulatory Focus 08 October 2020)
Secondary endpoints for efficacy should measure symptomatic disease for all study participants, yielding an estimate of protection against contracting a symptomatic case of COVID-19. EMA also recommends that “trials attempt to assess vaccine efficacy against severe disease,” if prevalence and accrual permit these data to be gathered.
For the safety evaluation of COVID-19 vaccines, EMA points first to its standard suite of guidance documents for vaccine trials. Since most vaccine-related adverse events occur within the first month to 6 weeks after vaccination, the agency states that “In principle, conditional marketing authorization for a COVID-19 vaccine could be based on review of at least 6 weeks post-vaccination safety data.”
After approval, EMA is looking for participants to be followed for at least one year post-vaccination to track post-approval safety and efficacy. “These longer-term data are important to document any late adverse reactions and to assess whether there is waning of protection against SARS-Cov-2 disease over time,” said the agency.
Earlier this month, EMA issued a comprehensive risk management plan that includes monthly post-approval reporting by sponsors, “so that EMA can act as fast as possible when a signal is detected.” (RELATED: EMA rolls out COVID vaccine monitoring plan, Regulatory Focus 13 November 2020)
EMA is also planning a public meeting for 11 December 2020 to discuss its regulatory processes in advance of the approval of vaccines against COVID-19.
“The public meeting will inform citizens about EMA’s role in the pandemic and of EU regulatory procedure,” said the agency in a press release. “It will also give the opportunity to the public and stakeholder groups to speak and share their needs, expectations and any concerns, that will be considered by EMA and the European medicines regulatory network in the decision making process.”


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Tags: coronavirus, EMA

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