EMA: Drug repurposing efforts saw limited success during pilot

The European Medicines Agency (EMA) said researchers working to repurpose out-of-patent drugs face significant challenges, such as interpreting the available data to support a regulatory application for a new use. They also noted that during clinical trials, they faced challenges developing the right inclusion/ exclusion criteria, isolating the drug's effects in a trial that used combination therapy, and choosing the right primary endpoint.

In a report on the agency’s pilot project to repurpose drugs, EMA summarized how researchers have been working with regulators to find new uses for drugs that have expired patents and offered details on the challenges they faced.

The pilot project was created as part of a framework developed by the European Commission’s STAMP Expert Group and other stakeholders in 2019 to help not-for-profit organizations and academia to repurpose drugs that were viewed as not commercially viable by the pharmaceutical industry. The framework allowed researchers to get free scientific advice (SA) from national- and federal-level regulators to get marketing authorization for new indications.

"The overall aim of the pilot was to assess whether the proposed framework could facilitate an application (variation/extension or marketing authorization application) for a new indication for an off-patent medicinal product," said EMA. "In that regard, it aimed to identify suitable candidates, to characterize repurposing development programs, to understand impact of the SA, to measure industry engagement as well as to explore the feasibility of using the EMA real-world evidence (RWE) initiatives to strengthen support to the champions."

Over the past few years, nine projects addressing important public health and unmet needs from not-for-profit organizations and academia were selected for the pilot. The projects received tailored support from EMA and the NCAs, such as introductory meetings, review help with briefing document preparation, and debriefing meetings.

“Despite the limited number of projects and although each project had its own specificities linked to the disease characteristics and the robustness of the existing data, certain scientific issues were frequently encountered such as issues with inclusion/exclusion criteria of the patient population in studies, with the isolation of the effect of the investigational medicinal product when used in combination therapy or when investigated in a trial without an internal control, with the choice of the primary endpoint and with the amount of evidence for the proposed dosing regimen,” said EMA.

The report also stated that the researchers had difficulty "appropriately describing the available data for the purpose of supporting a regulatory application and outlining the main data to establish the benefit-risk balance."

Regulators were able to provide a "safe harbor" when giving scientific advice to the pilot projects while reducing their administrative burdens and allowing flexible timelines. Despite these benefits, EMA noted that the process was resource-intensive for both regulators and researchers. They also noted that researchers have been facing hurdles in engaging with marketing authorization holders (MAH), generic drugmakers, and industry associations.

“To improve uptake by applicants and MAHs, Industry Associations are encouraged to facilitate connections between not-for-profit organizations and their member companies,” said EMA. “EMA and NCAs can also explore multi-stakeholder interactions to support joint discussions between champions, MAHs/applicants, and regulators.”

EMA said that so far, the pilot has had limited success. Only one of the selected projects received approval for a new indication by Spain's Agencia Española de Medicamentos y Productos Sanitarios (AEMPS). EMA also said it is considering a marketing authorization for one of the projects that it has approved.

Despite the limited success, EMA noted that seven of the nine projects are still under development and are still generating data.

EMA report

EMA: Drug repurposing efforts saw limited success during pilot

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