A recent tweet by a doctor I follow had me realizing we could all do with reviewing all hemophilia products… or at least, just knowing which product we use, and why. Currently, there are so many products and options. Do you know what your product’s name is, what “class” is it, and why you are using it?
First, know that all products approved by the US FDA for treatment are considered safe (from infectious disease) and efficacious (does its job). We always start by talking about products by first saying that there are two basic classes for standard factor: plasma-derived and recombinant.
The major difference between the two types is the origin of the factor, called the source material. Plasma-derived factor originates from human blood plasma. Recombinant factor originates from genetically engineered mammalian cells containing the human gene for factor. Most people with hemophilia in the US are now using recombinant products.
Recombinants can even be further classified according to their manufacturing process: first generation, second generation, and third generation. Several recombinant factor products also have a prolonged half-life, allowing you to infuse less frequently. The first of these new products was introduced in 2014.
First generation products contain human/animal proteins in culture medium; albumin added in final formulation
Second generation products contain human/animal proteins in culture medium; sugar added in final formulation, no albumin added
Third generation products contain no human/animal proteins added at any stage; formulated with sugar, not albumin
There is mention of a fourth generation–those products that are made not from mammalian cells but from human cells, so there is no risk of animal cells in the formulation, which may limit the formation of inhibitors.
And of course, there are nonfactor products, like Hemlibra.
So, know your treatment and why you use it! Ask:
Who selected your therapy—you, your doctor, or both? Could you explain to another patient or parent why you chose that product over the others Don’t leave the decision making to your doctor only, and absolutely not to your payer only. Remember that your payer may prefer to reimburse the least expensive product and not necessarily the best for you. Decide with your physician which product is best for your child. One day you may need to justify your product choice to your payer. It will help if you can learn to speak the language of therapies!
And use our chart, which we update frequently on our homepage. You can download this by clicking on the image on our homepage.