What are the regulations regarding personalized medicines in the pharma supply chain?
The regulations concerning personalized medicines in the pharma supply chain remain unclear. Some regulatory bodies lump them in under the same category as other drugs, while others consider them an exception. But what are personalized medicines in the first place?
These refer to drugs that are custom-made for a single patient, with that patient’s individual DNA or other blood characteristics serving as a guide or actual source component. An intriguing example can be found in the novel chimeric antigen receptor T-cells (CAR-T). It causes a patient’s own T-cells to be converted into cells that can recognize the specific sort of cancer that the patient possesses and thus attack it in the same way that normal T-cells target viruses and bacteria. In a nutshell, it is a method of tricking a person’s immune system into attacking cancer cells that it would otherwise ignore.
When it works, the results can be spectacular. As a result, personalized, precision medicine, and pharmacogenetics have drastically improved medical alternatives for patients while opening up new revenue streams for the biotech sector. However, the question that has not been clearly answered yet is: due to the nature of these medicines as well as the method of their production, do they fall under the same standards of regulation as other medicines? This blog article will try to answer that question, alongside providing other important info regarding this very important type of drug.
How are personalized medicines in the pharma supply chain categorized?
Although they all fall under a single category, personalized medicines can be incredibly diverse both in their effects and manufacturing process. The only real thing they have in common is that they are made for a specific patient. Accordingly, we have decided to focus on a specific example, the one of CAR-T from the previous subsection.
CAR-T treatment is a type of medication that is based on autologous blood extraction, which means that blood is extracted and then re-infused into the same patient. In contrast, allogenic blood extraction is meant to be re-infused into other patients. Scientists are optimistic that CAR-T therapy using allogenic blood will be successful in the future. If or when this occurs, the ensuing medications may be off-the-shelf, as are the majority of existing cancer therapies. But, until that day arrives, CAR-T therapy is only available in autologous form.
Personalized medicines under the DSCSA
We have already written about the DSCSA and its meaning for upcoming serialization compliance requirements. Along with the EU FMD, it accounts for a huge percentage of global pharmaceutical manufacturers and transporters and as such heavily influences the global supply chain. Let us now take a look at what these two directives have to say about the status of personalized medicines in the pharma supply chain.
The DSCSA only applies to medication and biological products that are intended for commercialization. This means that the product would be sold to supply chain members before arriving at a dispenser, who would administer the product as is to the patient. CAR-T therapies, on the other hand, are “manufactured” especially for a specific patient from that patient’s own blood. Most importantly, because the manufacturer ships the product to an approved treatment centre for infusion into only the designated patient, the product is never the subject of a DSCSA “transaction”. Thus, it is not subject to the rules and regulations that other medicines being serialized and traced under the DSCSA are.
The complicating factor
This method of determining the status of personalized medicines in the pharma supply chain is based on the drug’s autologous nature. Any future allogenic CAR-T therapies might be designed to be less expensive and more “off-the-shelf.” In that situation, this argument may no longer be valid, because an allogenic-based CAR-T therapy would almost certainly be susceptible to DSCSA transactions in the supply chain. That is, they would most certainly be sold through specialized distributors.
Additionally, CAR-T therapy is made up of blood components that are injected into the patient. The DSCSA provides an exemption for the distribution of blood or blood components intended for transfusion. Transfusion and infusion have similar meanings. If the FDA believes that they are insignificant for the DSCSA, then this specific exemption would also apply to CAR-T therapy. Because this kind of analysis does not rely on the drug’s autologous nature, it may result in an exemption even if CAR-T therapies can someday be made with allogenic blood.
Personalized medicines under the EU FMD
Applying the EU’s Falsified Medicines Directive (FMD) to autologous personal medicines in the pharma supply chain is a little easier. Annex I of the Delegated Regulation (EUDR) contains a list of prescription-only medicinal items or product categories that do not have the safety features. That is, anything on that list is excluded from having both an anti-tamper device and a unique identification. One of the entries refers to “Advanced therapy medicinal products which contain or consist of tissues or cells.” CAR-T treatment pharmaceutical items appear to fit under this exemption category since they contain cells taken from the patient. This would apply not only to autologous therapies but also to allogenic therapies.
It appears that medications manufactured in the same manner as today’s CAR-T therapies should be exempt from both the DSCSA and the FMD. However, the main takeaway from this article ought to be that their status remains unclear at best. This has a lot to do with the fact that such therapies are quite new and have not been adequately discussed in this context, which is something to be remedied in the future.
Here at Nubinno, we offer a plethora of articles that discuss topics related to serialization, track & trace, as well as the pharmaceutical supply chain. Check out our other articles or have a perusal of our excellent services to help you meet any compliance requirements for your respective regulatory body.