14/15 December 2023
In recent years, the number of applications of cell and tissue products for the treatment of various diseases has increased considerably. Many of these applications and products come from medical development in hospitals, clinics and universities. Many of the developers or scientists involved either do not know or are not aware from which step or for what type of application they fall under the respective national or international requirements of the drug laws and/or licensing regulations.
The distinction between homologous and non-homologous applications, which means whether the administered preparation fulfils the same basic functions for donor and recipient or not, often does not seem to be correctly assessed (see News "FDA issues Warning Letter to Manufacturer of Human Cells, Tissue or Cell based Products").
At the end of May, the FDA published an "Untitled Letter" to R3 Stem Cell, LLC. On its website, the company describes a number of treatments with stem cell products in its clinics for the treatment of diseases such as rheumatoid arthritis, spinal canal stenosis and trigeminal neuralgia. In this letter, the FDA states that these therapies, as described there, are human cells, tissues or cellular or tissue-based products (HCT/Ps) as defined in 21 CFR 1271.3(d). If they do not meet the requirements of 21 CFR 1271.10(a) and no exception can be made in 21 CFR 1271.15, they will be classified as drugs, devices and/or biological products under the Federal Act on Food, Drugs and Cosmetics (FD&C Act) and/or the PHS Act. They are therefore subject to additional regulation, including a pre-approval review. On the basis of the information available, these products are not qualified for exemption under 21 CFR 1271.15 and are offered for non-homologous applications. Accordingly, a Biologics License pursuant to 42 U.S.C. 262(a) is required prior to marketing, but will be granted only after proof that the product is safe, pure and effective. Prior to this, i.e. during the development phase, such products may only be marketed for clinical application in humans if the sponsor has submitted an application for a new investigational product (IND) and this has entered into force.
The FDA also points out that there is an increased risk of unwanted side effects or contamination when used against serious diseases and administered by various routes (including intravenous) as described on the websites. Therefore, the authority makes it clear once again that both manufacturers and healthcare professionals who are uncertain about the regulatory status of their products should contact the FDA to obtain a recommendation or decision on the classification of an HCT/P. The FDA has also made it clear that the classification of a HCT/P should be based on a number of factors. Important information and basic guidelines can also be found on the FDA website.