FDA Draws Up Guidelines For Biologic Products

The US Food and Drug Administration has issued guidelines on its requirements for the registration of biological products, which include cell-based therapies such as Genzyme Tissue Repair's Carticel (autologous cartilage cell culturing service) for knee joint repair.

An important distinguishing characteristic of these guidelines is a flexible approach to the FDA's requirements for evidence of effectiveness; the agency will not require prospective, randomized trials if effectiveness can be reasonably demonstrated by other means. For example, the FDA said that it will accept data from short-term studies as adequate to support approval if a favorable risk-benefit profile has been established and long-term safety concerns are low. In those cases, post-marketing studies or registry data may be used to confirm longer-term outcomes.

Instead of formal controlled studies, the agency will also allow comparative evaluations with historical databases to demonstrate effectiveness in those cases where compelling differences can be shown.