EMA’s annual report highlights regulatory activities in 2022

The European Medicines Agency (EMA) on Monday released its annual report for 2022, detailing the agency’s activities for a year marked by adjusting to the COVID-19 pandemic and major regulatory transitions as the agency implemented the EU Clinical Trials Regulation (CTR) and took on an expanded mandate.

The report and its two dozen annexes span nearly 250 pages and review EMA’s key achievements and metrics in 2022, highlighting the agency’s work to evaluate and monitor medicines, its response to overlapping public health emergencies, the agency’s newly expanded mandate and its efforts to improve the EU clinical research environment. The report also provides figures for the agency’s human and veterinary medicines programs, rapporteurships across its various committees and inspections and compliance activities.

“Although we saw some return to more routine activities, largely thanks to the speed of scientific progress and the roll-out of effective vaccination campaigns across the EU, the COVID-19 virus continued to force us to pivot, innovate and adapt,” said EMA’s Executive Director Emer Cooke.

Medicines and regulatory activities

In 2022, EMA recommended the authorization of 89 medicines, including 41 that contained a new active substance (NAS). Of those, five were recommended after receiving an accelerated assessment, nine medicines received a conditional marketing authorization and five medicines were authorized under exceptional circumstances. EMA also recommended eight medicines with PRIority MEdicines (PRIME) designation and six advanced therapies, including CSL Behring’s hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec).

EMA also noted that it recommended 10 veterinary medicines in 2022 and oversaw the application of the Veterinary Medicinal Products Regulation, which took effect in January 2022.

As part of its COVID-19 response, EMA recommended two COVID-19 vaccines and two therapeutics for authorization in 2022: Sanofi and GKS’s VidPrevtyn Beta, Valneva’s COVID-19 inactivated adjuvanted vaccine, Pfizer’s Paxlovid (nirmatrelvir/ritonavir) and AstraZeneca’s monoclonal antibody Evusheld (tixagevimab and cilgavimab).

EMA noted that it recommended 21 orphan medicines in 2022 that had their rare disease status confirmed by the end of the year, and three medicines that previously had orphan designation lost that status by the time they were authorized.

Additionally, EMA said it recommended 90 extensions of therapeutic indication for already authorized drugs in 2022, more than one-third of which were for pediatric use. The agency also recommended three medicines for use outside the EU under the EU Medicines for all (EU-M4All) procedure, including Takeda’s Dengue Tetravalent Vaccine (live, attenuated) and two drugs to treat diabetes mellitus.

Three medicines received negative opinions, and EMA said that 98% of its opinions were unanimous with “no divergent opinions” among its committee members.

The agency also pointed out that more than half (52%) of submissions that received a positive opinion were given scientific advice or protocol assistance from the agency at some point during product development. When looking at NASs, that number rises to more than three-quarters (78%) of submissions.

In 2022, EMA said it received 833 requests for scientific and protocol advice, a slight decrease from 2021. While the agency saw more requests for scientific advice and follow-up requests, it saw fewer requests for scientific advice related to COVID-19 and fewer requests for protocol assistance.

The agency granted 13 PRIME designations and rejected 27 requests for the designation in 2022, a drop from the previous four years.

EMA said it received more requests for orphan designation in 2022 than it did in any prior year going back to 2018. Among the requests for orphan designation, there were more requests for the designation for medicines for both adults and children, while requests for designation for conditions only affecting children declined. The most frequent requests were for products intended to treat congenital, familial and genetic disorders (62); nervous system disorders (29); and blood and lymphatic system disorders (22).

The length of time it took for EMA to issue a positive opinion on a drug increased to 461 days, up from 394 days in 2021. The increase was largely driven by longer company clock-stops, EMA said, noting the average length of company clock-stop time was 205 days, up from 154 days in 2021.

When looking at the types of reviews, the length of assessment varied from an average of 222 days for accelerated assessments, 386 days for NASs, 402 days for submissions that received scientific advice and 511 days for small- and medium-sized enterprises (SMEs).

Inspections

While EMA does not conduct inspections itself, it plays a role in coordinating them across the EU regulatory network.

In 2022, the number of good manufacturing practice (GMP) inspections carried out by European Economic Area (EEA) regulators dropped to 180, down from 369 the previous year. EMA noted that the decrease was in line with the number of inspections that were carried out in 2020, which was also down significantly from years prior. EMA said that six statements of non-compliance were issued in 2022.

More good clinical practice (GCP) inspections were conducted in 2022 (75) compared to 2021 (36) and 2020 (59), though the number of inspections was markedly down from the two years prior to the pandemic.

The agency also said that fewer pharmacovigilance inspections were conducted in 2022 than in most of the last five years.

New mandate, CTIS, and more

In 2022, EMA’s mandate was extended to reinforce its role in crisis coordination, preparedness and response.

“This mandate gives us added responsibilities in the area of crisis co-ordination and response. As well as formalising some of our ad hoc procedures, it gives us new tools to respond effectively to future public health crises and tackle medicine shortages,” Cooke said, adding that the global mpox outbreak put those tools to the test.

As part of its expanded mandate, EMA stood up a new Executive Steering Group on Shortages and Safety of Medicinal Products (MSSG), which developed lists of “critical medicines” for both COVID-19 and mpox. The agency also formalized its Emergency Task Force (ETF), which was originally formed to steer the agency’s COVID-19 response.

The agency oversaw the development of the Clinical Trials Information System (CTIS) as part of the CTR, which entered into force on 31 January 2022. While use of the CTIS was voluntary in 2022, EMA said it authorized more than 200 initial clinical trial applications and had another 200 applications under review at the end of 2022. Use of the CTIS became mandatory on 31 January 2023.

Additionally, EMA played a role in establishing the Data Analysis and Real World Interrogation Network (DARWIN EU), which Cooke said could usher in “a sea change for medicines regulation, feeding into a future European health data space.

Report, annexes, statement

EMA’s annual report highlights regulatory activities in 2022

Related topics

Recommended content

Welcome to the new RAPS Digital Experience