Today we are pleased to offer a review of an article from which just about anyone working in the specialty pharmacy segment might learn something…. or even a lot.
The topic is cell and gene therapy (CGT)….. which is getting increasingly greater attention given recent approvals and a jaw dropping pipeline.
Can you define cell therapy vs. gene therapy?
Even now, many would struggle to clearly differentiate between them.
Here’s what the article says……
- Gene therapy is defined as the repair, replacement, or deactivation of dysfunctional genes to reestablish normal function.
- Cell therapy is defined as the transfer of autologous (from oneself) or allogeneic (donor) cellular material into a patient for medical purposes.
- To devlop a CGT product a target gene or specific type of cell is identified to determine whether that gene or cell can be modulated directly within a patient’s own body (known as in vivo treatment), or if genetic or cellular material must be extracted from the body, modified, grown externally, and then transferred back into the patient (known as ex vivo treatment).
- Over the past decade+ the FDA has approved more than 30 cell, cord blood, and gene therapy products (see list in the article).
- Most investigational CGT products will continue to target oncologic conditions and rare diseases.
- More than 2,000 CGT products globally are in various stages of clinical development. and…. as we have all surely noticed by now,
- CGT therapies are among the most expensive medications in the world…….5 of the 10 most expensive drugs of 2023 were gene or cell therapies….. keeping health insurance companies awake at night worrying how to pay for them.
A reading of the full article will fill in much more detail of the current and, most importantly, give insight into the CGT ‘cures’ that one day are likely to reshape the legacy SP market.
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