Debating the risks of advanced therapies

Do advanced therapies represent medical miracles or risky business? This is the question that is debated by an Informa analyst in an Executive Briefing for Scrip.

New EU rules concerning the development, evaluation and approval of advanced therapies came into effect almost a year ago. More recently, however, the EC has issued a new directive that, among other things, states that a risk-based approach will be taken into consideration when determining approval for all Advanced Therapy Medical Products (ATMPs).

"Work on the new EU advanced therapies framework began back in May 2005," Ian Schofield, author of the report and Principal Analyst at Informa, explained in a press statement. "In the words of the commission, gene therapy, somatic cell therapy and tissue engineered products are expected to have a major impact on public health by improving patient's quality of life and by changing medical practice significantly."

Schofield continued: "The three also share a number of scientific and ethical features associated with the use of human tissues in R&D and medical practice. Of course, because this is new territory, there are also unknown risks and the new regulations aim to keep this process as safe as possible for patients and their families."

When submitted via the EU's centralized procedure, ATMPs, as with all new pharmaceuticals, must be accompanied by a risk management plan. According to Schofield, this is especially important for ATMPs because their novelty, complexity, and technical specificity may bring unknown risks.

In particular there could be risks involved in the administration procedure, which may elicit unwanted immune responses, as well as risks relating to the quality of the cells used, and the storage and distribution of the product. There may also be risks posed to living donors.

Although there are several concerns surrounding ATMPs, the potential benefits are enormous — possible cures for cancer, paralysis, Parkinson's, Alzheimer's and more. The press statement explains: "Research into stem cells could lead to treatments for neurodegenerative diseases, and gene therapy could be of use in treating inherited disorders, certain types of cancer and viral infections."