FDA/EMA blood 'cluster:' Pharma could benefit from streamlined clinical trial requirements

The FDA and EMA's most recently formed discussion group addresses blood-related drug products, and pharma companies involved in the space said they hope the agencies can agree on study designs. The result could be the avoidance of unnecessary testing replication or unnecessary diverse testing methodologies, sources said.

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The FDA and EMA's most recently formed discussion group addresses blood-related drug products, and pharma companies involved in the space said they hope the agencies can agree on study designs. The result could be the avoidance of unnecessary testing replication or unnecessary diverse testing methodologies, sources said.

The next blood "cluster" meeting has been scheduled for 27 October.

Clusters are information-sharing agreements under which EMA and FDA engage in periodic teleconferences to discuss common regulatory concerns. The purpose of the blood cluster, like the other clusters, is to discuss issues such as policy, guidance, product development, and potential product safety signals, an FDA spokesperson said.

Brendan Cuddy, scientific administrator, Manufacturing and Quality Compliance, at the EMA, said the agencies can exchange product information including scientific assessments. He said the companies may look at pending product applications to the FDA and/or the EMA.

The blood cluster will address concerns related to plasma derived products such as albumin, immune globulin, and clotting factors, the spokesperson said.

"There 'may' be agreement by both agencies on study design and particulars," she said. "When it's possible to harmonize requirements, then yes, that's the streamlined approach to global product development."

It would be a boon to CSL Behring if Hizentra, an immune globulin subcutaneous 20% liquid for treating patients diagnosed with primary immunodeficiency (PI), is discussed, said Val Romberg, the company's senior vice president of global research and development. The product, approved in the US, is undergoing EMA review. He said his hope is that the cluster could lead to streamlined clinical trial requirements for blood-related products.

For Hizentra, the company had to conduct one Phase III trial for the US but an additional trial for European authorities, he said. "Ideally in the future, it would be nice if regulatory authorities could arrive at a much more aligned view for clinical development," he said.

Pfizer's (NYSE: PFE) Ed Mascioli, vice president, BioTherapeutics R&D, orphan and genetic diseases, agreed that the cluster could lead to streamlined trial requirements. Mascioli, speaking to this news services at a recent orphan drug conference in St. Louis, added that the company has pipeline hemophilia products.

Baxter (NYSE:BAX) and Talecris Biotherapeutics (NASDAQ:TLCR), which are also involved in the hemophilia space, could also see benefit, a healthcare consultant said. Both companies declined to comment.

The net effect of the cluster is likely a positive for pharma, especially when it could mean FDA acceptance of international trial data that is often a sticking point in applications, a healthcare consultant noted. A consolidated regulatory viewpoint would make the most sense but the devil is in the details. A combined FDA/EMA perspective in clinical trial development could also mean additional regulatory complexity, he said.

Romberg noted that the FDA reviews applications more from a science standpoint, such as whether there is statistical significance of a primary hypothesis and whether the mechanism of action is plausible. The EMA tends to take a "medical view" where it considers more often in applications whether the physician understands how dosing works and how it would be modified, he said. The EMA is also more open to accepting opinions of KOLs, he said.