Telemedicine, social media, mobile medical Apps, stem cells……. just a few examples of innovations that are quickly becoming an integral part of modern (rare disease) health care. At the same time these innovations in medicine seriously challenge our current regulatory framework.
On 30 April 2012, the European Medicines Agency (EMA) released a reflection paper on the classification of advanced therapy medicinal products (ATMPs) for public consultation. In principle, gene therapy, cell therapy, and tissue-engineered medicines should qualify as ATMPs. However, what became clear to me after having read the reflection paper is that in reality classification of ATMPs is complicated.
In particular classification of stem cell therapies may be the subject of considerable discussion. For those not fully familiar with the concept of stem cells, I can highly recommend a wonderful introductory video by the Irish Stem Cell Foundation, a non-profit organization. In brief, there are three types: embryonic, adult, pluripotent. Each type has a unique set of pros and cons.
In the EMA reflection paper it is stated that “Products consisting of cells or tissues may scientifically be at the border between Tissues and Cells directive (Directive 2004/23/EC) and the advanced therapy Medicinal Products (ATMP) regulation (Regulation (EC) No 1394/2007). Whether stem cells will classify as an ATMP depends on the level of manipulation during preparation of the product, because this entails a risk of change in the product’s biological characteristics. A second important question is whether the product is “intended to be used for the same essential function in the recipients”.
Where does this leave adult autologous stem cells ? In the US, 21 CFR 1271 has recently been changed, and consequently many adult autologous stem cell therapies will (have to) be considered as drug products. However, in a recent issue of the Journal of Translational Medicine Freeman & Fuerst argue that this type of therapies should be considered as practice of medicine, rather than a drug product. Of course, protecting the interest of patients should always come first and the authors do not question the important role of the FDA in safe-guarding the health of the US. However, Freeman & Fuerst express a serious concern that implemented regulatory changes hold little societal benefit and are in fact stifling medical innovation in the area of adult autologous stem cell therapy.
What it all seems to boil down to is striking the right balance between ensuring patient safety and allowing timely access to innovations in medicine with a real added-value.
Unfortunately, like beauty, the right balance is in the eye of the beholder.
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