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Gene and Cell Therapies….. Oh MY!

The hottest topic in the media these days is Artificial Intelligence (AI). It seems like everyone is talking about it. But that may be short sighted if you are neck deep into specialty pharmacy.

We’ve sent several reports recently on gene therapies because it is rapidly becoming ‘the next big thing’ in medicine. Analysts say that by the end of 2024 about 20 cell therapy launches and as many as 30 gene therapy launches are expected.  Oh MY!

Within the past few days, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee voted to back accelerated approval for SRP-9001 for patients with Duchenne muscular dystrophy with a decision due before month’s end. Such approvals are remarkable as these new ‘wunderkind’ therapies started to sprout a only a short time ago. 

So, what is the impact of these ‘one and done cures’? Our conclusion is that the specialty pharmacy model doesn’t coexist well at all. To date, SPs have had not role in dispensing or even distributing gene therapies given the hospital-based process to harvest patient samples for the brewing of the ultimate patient/genetically specific elixir.  Oh MY! Oh MY!

If you are interested in a basic primer around gene therapy CLICK HERE

If you are curious about how many gene therapies have been approved as of 4/2023 CLICK HERE

If you would like to see what others are saying about commercializing specialty drug cell andgene therapies CLICK HERE


Critical Success Factors for Commercializing Specialty Drugs, Cell and Gene Therapies

Although payers have become somewhat accustomed to the high cost of specialty drugs, the price tags for gene therapies are causing “sticker shock.”

Apr 17, 2023 — Specialty product innovation is increasingly viewed as a journey that begins during pre-commercialization and continues throughout the product life cycle. Pharmaceutical and biotechnology manufacturers now recognize the need to develop, implement and refine a process-driven strategy that is built upon solid structural dynamics and a proactive approach for every phase of commercialization, spanning ideation, clinical research and development (R&D), clinical trials, product launch, market access, distribution and beyond. This strategic foundation gives life sciences companies and their people a clear pathway and focus to ensure that tasks and projects are aligned across teams and departments.

With the development of a potential new specialty drug, cell or gene therapy (CGT) typically requiring a decade or longer, there are critical success factors along every iterative step of the product life cycle that all point toward the same goal: achieving market dominance and capturing substantial market share. .

…………..CLICK ABOVE to access the full article


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