RE: JPM18 Jan 2023 18:40
vid - thanks - interesting stuff !!!
1. Cell and gene therapies – big opportunities, big challenges
This is the area where I think this tension is the most obvious. Everyone agrees that cell and gene therapies are poised for an explosion, with far more drugs in the pipeline than are currently approved, and the FDA predicts it will clear 10 to 20 per year by 2025.
“I’m going to make a statement that could come back and bite me, but I think T-Cell therapies are going to be bigger than monoclonal antibodies,” AdaptImmune CEO Adrian Rawcliffe said at one session.
These therapies have incredible potential to fight cancer and other diseases using the patient’s own immune system. This could mean avoiding the side effects associated with radiation therapies, even targeted ones. The treatments also have the potential for long-term remission. As such, big and small pharma alike are investing in this area and the hype around it at JPM was strong.
But the challenges are also severe. Whether autologous or allogeneic, manufacturing a cell therapy is much more complicated than manufacturing a small molecule. There’s so much precision required, and so much possibility for introducing error at this stage, that even small companies are investing in internal manufacturing operations. And they have to do it early because regulators are just as interested in their chemistry, manufacturing, and control (CMC) as their efficacy data.
And there are big questions about how these therapies will be paid for, particularly those that turn out to be essentially curative in a payment system that’s set up to treat cancer as a chronic disease.
Ultimately, we are going to see these therapies proliferate and the industry figure out how to execute them efficiently. But because of the cost and risk associated with them, they’re likely to be the strongest in areas where no other, more traditional, alternatives exist.
5. Massive disruption to clinical trials
Expect more on this topic from me in a larger piece soon, but suffice it to say that clinical trials are being disrupted from all sides, and most seem to agree we haven’t even seen their final form.
First there’s decentralisation, a process innovation that was kicking along in very low gear until COVID-19 came along and launched it into the stratosphere. Unlike telehealth, which has experienced somewhat of a reversion to the mean, decentralised clinical trials (DCTs) are here to stay. The big difference? While telehealth’s effect on the bottom line is questionable at best and dependent on reimbursement changes, DCTs are showing clear savings and efficiency gains (though they are not without their growing pains).
Second, we’re in the middle stages of an industry-wide reckoning with the bigoted history of clinical trials and there’s a massive corrective effort underway to make sure that clinical trials reflect the disease populations they’re studying.
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