Regional Comparisons

The median approval time for new drugs in Switzerland lengthened in 2022, mainly because of applications filed during the peak of the COVID-19 pandemic, says the country’s regulator.

A newly-translated guidance document from Japan’s drug and device regulator outlines the points that clinical trial sponsors should consider when using electronic methods to collect informed consent from participants.

Rhythm is working with country-level authorities and centers of excellence in Europe to gain reimbursement and access for its obesity treatment Imcivree.

This second segment of a two-part article on a regional comparison of EU and US approaches to decentralized clinical trials highlights the differences between the two regions relating to the more practical aspects of setting up and running such studies.

The investigator's ability to delegate trial-related activities to local HCPs could create operational and reimbursement challenges, as well as potentially boost patient recruitment, stakeholders said. The guidance does not address digital divide and how to deal with a massive influx of data.

In this regional comparison, regulatory experts at a US-based decentralized clinical trials (DCT) firm discuss their views on how the FDA’s draft DCT guideline compares with the pan-EU recommendation paper on this topic. This first segment of a two-part article dwells on the thinking behind why US and EU regulators tend to go down different paths. 

The EMA offers more flexibility than the US FDA when it comes to missed primary endpoints in certain circumstances, meaning a potentially quicker route to authorization in the EU.

Drugs for Alzheimer’s disease, amyotrophic lateral sclerosis and other high unmet medical needs are among the new products that will now be eligible for assessment under the OPEN framework, the European Medicines Agency’s initiative for collaboratively assessing marketing applications with non-EU regulators.

More than seven months after the Oncologic Drugs Advisory Committee voted that Pepaxto and Copiktra had negative benefit-risk profiles in their labeled indications, the drugs' approvals remain intact. Oncopeptides is still considering whether to heed the FDA's request to withdraw the Pepaxto NDA, even though the drug is not currently marketed. Secura Bio says Copiktra remains on the market and available for prescribing.

The two regulators were an OCEAN apart on their interpretation of the overall survival and progression-fee survival data from the Phase III confirmatory trial. European regulators saw fit to recommend Pepaxti’s approval, even after the drug was voluntarily discontinued in the US market due to FDA concerns.

Guidance on regulatory and data protection considerations for decentralized clinical trials varies widely between the EU and the US. Trial sponsors need to pay particular attention to issues such as missing data, informed consent and data collection, say lawyers from Sidley Austin.

European Medicines Agency insisted on a stricter statistical analysis than the US FDA for demonstrating equivalence on the primary endpoint for both Coherus/BioEq’s Cimerli and Samsung Bioepis’ Byooviz; there also were differences in the regulators' preferred primary analysis population and primary endpoint interval.

Janssen’s ponesimod for relapsing multiple sclerosis had a slightly faster review in the US.

In approving ponesimod for relapsing multiple sclerosis, neither the FDA nor EMA included in labeling data from a novel patient-reported outcomes fatigue instrument; FDA said the PRO was not shown to be capable of measuring within-patient change, while EMA said the difference relative to Sanofi’s Aubagio fell short of the threshold for a clinically meaningful benefit.

Janssen went against EMA’s advice on the choice of secondary endpoints for its multiple sclerosis drug Ponvory (ponesimod), but still enjoyed a relatively brisk review. The hierarchical order of endpoints did not seem to ruffle any feathers among US FDA reviewers.

Stemline Therapeutics’ Elzonris was approved for a rare cancer in Europe in January, following a long and arduous two-year journey through the EU regulatory process that included an initial rejection and a subsequent re-examination by the EMA. Its fortunes were very different in the US, where the review took just six months from filing to approval.