You are a unique snowflake.
You like your coffee with Soy milk, in a thick glass, with no sugar, and extra foam.
And it must be boiling hot, otherwise, it doesn't kick in just right.
Right?
So why on earth do you accept the same pill for headaches everybody else is taking?
Did you know there are thousands of possible reasons for this headache?
(Don't Google it!)
Our understanding of medicine is growing exponentially, and so is our ability to prescribe personalized drugs.
It starts with a better understanding of the genetic makeup of a patient or their specific tumor which allows Oncologists to prescribe the most effective therapy available to them, but it doesn’t end there.
Some medicines are 100% personalized in the sense that the actual medicine is custom-made to a single patient.
The most famous are CAR-T cell therapies, where the patient’s own immune cells are engineered to kill a specific tumor, and there are quite a few other notable mentions in the cell therapy space.
Antisense oligonucleotides are another class, pioneered by Milasen which was specifically tailored to Mila, a girl who suffered from a genetic mutation unique to her and got this cure just for her.
Developing the manufacturing process of these therapies is, by definition, developing a platform technology, and like anything in life, they differ in complexity.
Cell therapy woes go a long way- from poorly defined starting materials (i.e.- patient cells), tiny batch sizes that are hard to quality control, shelf lives sometimes measured in the hour’s scale, and insanely complex logistics.
On the other hand- Oligonucleotides are much easier to tweak from batch to batch while keeping the platform largely unaffected (with some caveats). So adjusting the order of nucleotides for the next patient can be done relatively easily, and this will be reflected in the cost of these therapies.
So the more complex the platform at hand, the more reliance we will see on advanced technologies- and indeed, we see that the Cell therapies are at the forefront of the industry’s early adopters, simply because they can’t rely on the traditional methods.
Economies of scale play a key role here and will influence the overall abundance of each kind of these therapies. We will also encounter new ethical questions and public health concerns with regard to reimbursement and accessibility of these treatments.
All in all - these are challenges that the industry is creatively tackling, and as we progress down this road we will surely see better personalized medicines that are safer, cheaper, and more effective.
The main driver will remain the platform complexity, and how advanced technologies make it easier to manufacture.
Exciting times ahead for CMC geeks!