Importance of Regulatory Oversight
Companies developing and manufacturing an advanced therapy also need to deal with an emerging regulatory regime. Duncan Mackay, director of regulatory affairs at Genzyme, explained that “the absence of a regulatory framework hinders the development of the cell-therapy sector.” He added that it should always be remembered that “the priority of the regulators is to protect the patient,” so the level of regulation should be proportional to risk. Companies, therefore, need to have a risk-management plan in place.
Mackay went on to summarize recent developments in the regulatory area. The EU introduced the Advanced Therapies Directive in December 2007, which means that the European Medicines Agency (EMEA) has control of cell-therapy products and that they are treated mainly as medicines (rather than devices).
In the U.K., the government has named the Human Tissue Agency as the competent authority for the Human Tissue Act, and firms now need licenses to develop cell and tissue therapies. It means dealing with a network of different agencies, all of which have different interpretations of what is required.
Although the U.K. remains a liberal environment for Phase I, once licensed, some products move quickly from the limited clinical environment of 500 patients, for example, to a situation where half a million could be receiving a product. Regulators, therefore, have to be conservative.