BioWorld International Correspondent

BRUSSELS, Belgium - A flurry of new guidelines on biotechnology product development greeted European researchers on their return from the end-of-year break - notably on the manufacture of gene therapy and somatic cell-based products.

"Within a few years, several gene therapy products and somatic cell-based medicinal products may be designated and submitted for EU marketing authorization," said a new European Agency for the Evaluation of Medicinal Products (EMEA) concept paper on good manufacturing practice for those products, adopted at the end of December. "The increased use of these novel therapeutic techniques demands that their quality and safety be ensured in order to safeguard public health and to prevent the transmission of diseases and of infectious genetically modified organisms."

The EMEA focus is partly safety related: products intended for gene therapy and cell therapy and based on human or animal cells now are legally considered as medicines, and consequently have to meet the full range of European Union rules in that area. But there also is another dimension: harmonization between the EU member states is needed to allow products to move freely around the 15-country (and soon to be 25-country) area. The new guidance is intended to create a "unified framework," and "to ensure that [manufacturing or clinical trial] sites and organizations in member states take all necessary measures to ensure these products are manipulated, processed, stored and distributed under controlled conditions."

Manufacturers should comply with basic good manufacturing practice requirements for EU medicines, the new guideline said. But the EMEA already is planning to produce further separate and more detailed guidance for gene therapy and somatic cell therapy products. For gene therapy products, supplementary regulation is envisaged with specific elements on gene delivery systems and potential biohazards, to minimize the potential adverse effects of the deliberate release into the environment of genetically modified organisms. And for somatic cell therapy products, "specific measures should be taken to ensure safety, quality of tissues, and cells of human or animal origin." Guidance should be established, the EMEA believes, not only to minimize the risk of infectious disease transmission, but also to ensure the traceability of somatic cells involved in the final cell therapy product.

At present, some member states have put in place national regulations for the products, and the EMEA has sent all member states a questionnaire to provide an accurate overview of the current situation on controls in place. The next step will be to create a group to draft new, specific rules. The EMEA said it wants to move fast, since increased use of those products "demands guidance should be developed in a timely manner to ensure public health."

At the same time, the EMEA has produced additional guidance on how to check other aspects of biotechnology-derived medicines. A new note was agreed at the end of December on the comparability of medicines based on biotechnology-derived proteins - currently a sensitive topic, since the comparability of biotechnology generics (or "biosimilars") was one of the most contentious discussions on the package of new EU pharmaceutical rules. The guidance suggests that it might not be necessary for a generic copier to repeat all safety and efficacy tests, but clinical trials will usually be necessary, with their scope and duration determined on a case-by-case basis (depending on the type of product, therapeutic area, and experience).