EMA officials discuss expedited approval pathways, scientific advice mechanisms

San Diego – Officials from the European Medicines Agency (EMA) discussed the different types of expedited approval pathways available to sponsors of innovative drugs and advanced therapy medicinal products (ATMPs), as well as the various mechanisms for sponsors seeking scientific and regulatory advice at the DIA 2024 Global Annual Meeting on 19 June.

The panel featured Anabela Marcal, an EMA liaison official to the US Food and Drug Administration (FDA), Evdokia Korakianiti, head of quality and the safety of medicine for EMA, and Sabine Haubenreisser principal scientific advisor at EMA’s stakeholders and communication division. The session was moderated by Steffen Thirstrup, EMA’s chief medical officer.

Marcal discussed the different pathways to get novel products approved through the centralized procedure. These pathways include accelerated assessments, which are similar to FDA’s priority review program. Under the program, sponsors have to show that products address an unmet medical need and products are reviewed within 150 days.

Another pathway is conditional marketing authorization, which is for orphan drugs and other products that treat life threatening or debilitating diseases, that also address an unmet medical need. Under this pathway, sponsors can seek authorization prior to obtaining comprehensive data showing a drug’s safety and efficacy. Conditional marketing authorizations are valid for one year and must be renewed to remain on the market.

The third route is approval under exceptional circumstances, which is granted when limited information on the drug’s safety and efficacy is available because of the rarity of the disease or the collection of full information is not possible or ethical. These authorizations are valid for five years.

Various meeting pathways for innovative dugs

Haubenreisser said that sponsors have different options to meet with the agency for questions related to product development.

These mechanisms include the EMA’s Innovation Task Force (ITF) which supports innovative drug development through a multidisciplinary approach to addressing new therapies and technologies. This forum is best for a brainstorming stue on innovation in areas without existing guidance and offers support for borderline product classifications and it also helps regulators know what technologies are on the horizon, and such meetings are free of change. The ITF team is comprised of a cross-functional team of representatives in the quality, safety, efficacy, and pharmacovigilance areas.

A complementary group to the ITF is the EU Innovation Network (EU-IN); the group “facilitates development of innovative medicine by addressing gaps in regulatory support and by providing a platform to share best practice and to strengthen engagement between regulators and innovators.”

The PRIME program is another mechanism to help sponsors develop products early on, product in this pathway must address an unmet medical need where the benefits of the product outweigh the risks.

Haubenreisser said that through this program, the rapporteur will work with the sponsors from development of the drug to its approval, this pathway is primarily used to support new medicines that have a major impact on public health.

The Scientific Advice Working Party (SAWP) is also available to answer any scientific questions during product development. The SAWP does not address regulatory issues and is chaired by Paolo Foggo of the Italian Medicines Agency and comprises 65 members from various EU Committees. Members have expertise in all major areas of drug development. These medicines often go through conditional approval.

For ATMPs, sponsors can get advice from the Committee for Advanced Therapies (CAT) provides advice on cell therapies. The CAT addresses the quality, safety and efficacy of ATMPs, and issues a draft opinion to the MAH. She said that “the CAT plays a vital role pertaining to all regulatory and scientific aspects of ATMPs.”

Haubenreisser added that “we have various platforms to help you support you in your development very early on … the earlier that you engage us the earlier you can get the marketing authorization.”

Advice for manufacturing methods

Manufacturers can also contact the EMA’s Quality Innovation Group (QIG) for advice related to quality issues associated with the use of advanced manufacturing methods for ATMPs, said Korakianiti.

The QIG works with manufacturers from the proof of concept to product development, approval, and throughout the product lifecycle.

Some areas that QIG is exploring with sponsors are decentralized manufacturing, process modeling, digitization and automation of manufacturing, and use of platform technologies.

The QIG holds public meetings twice a year where they discuss various case studies involving advanced manufacturing technologies which are then followed-up with closed meeting at the request of manufacturers seeking more tailored advice.

One recent case study is an applicant that has requested advice for a decentralized manufacturing strategy to produce fresh CAR-T cell products that are administered to patients in the proximity of a hospital. The QIG is working with the sponsors to resolve questions related to the use of platform technologies and comparability strategies to compare the current manufacturing process with a decentralized method.

DIA Global Annual Meeting

EMA officials discuss expedited approval pathways, scientific advice mechanisms

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