What we observed this past year might be the early signs of a tsunami for a new class of biopharmaceutical products: living viruses and cells. These signs include recent regulatory approvals of a genetically engineered virus to treat patients with vision loss, genetically engineered T-cells to treat patients with acute lymphoblastic leukemia and large B-cell lymphoma and a genetically engineered virus to treat patients with melanoma. Additionally, in the United States, the 21st Century Cures Act accelerates FDA review of regenerative medicine advanced therapies. In Europe, EMA has revealed that, out of the 30 medicines that have received their PRIme MEdicines (PRIME) designation, one- third are advanced therapy biologics.

Regulatory authorities recognize the importance of moving these products through clinical development, but they also recognize that the transfer of these advanced therapy manufacturing processes from universities or hospitals into appropriate GMP and quality system compliance for human clinical studies is a major challenge.

Patients using these products need to be protected, and these new living biopharmaceutical products have unique challenges (Table 1).

Table 1 Unique Challenges for Protein-based and Virus/Cell-based Biopharmaceuticals

For the protein-based biopharmaceuticals, GMP and quality systems are well developed and, when implemented correctly, have served and protected patients during clinical development very well. But because of the unique challenges presented by the living virus- and cell-based biopharmaceuticals, these systems have to be adapted in a practical, common-sense way.

At the end of the PDA Europe conference, Advanced Therapy Medicinal Products, in June, the following course will be offered, “Practical Application of Phase- Appropriate GMP & Quality to Clinical Development of ATMPs.” This course will discuss common sense and regulatory- compliant CMC approaches to appropriately and adequately protect patients both during the transfer from a university/ hospital into a patient, and during the clinical development of these advanced therapy medicines. For more information and to register, visit the course website.