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CAR-T: What’s next? Part 2

In my previous blog post, I suggested that the market size for gene-modified T-cell therapies would be roughly $9 Bn in 10 years, with a range of around $5 - $20 Bn. In this post I will provide some structure for this wide range by breaking out five key factors that will help guide you in interpreting what happens over the next few years. Three of these factors are clinical and scientific: the products and their indications (this one is the most obvious); performance against solid tumors and a broader set of hematological malignancies (i.e., will they work beyond the B-cell malignancies where they’ve performed best so far); and the success of allogeneic ‘off-the-shelf’ treatments (versus the

CAR-T: What’s next? Part 1

Over the last two months, several news events have brought CAR-T cells to widespread public attention, the most prominent of which is the FDA approval of the first gene-modified cell therapy, for children with a type of leukemia who have failed other options. The new therapy, Novartis’ Kymriah (tisagenleucleucel; CTL019) is the first in a novel class of treatments known as CAR-Ts, or Chimeric Antigen Receptor T-cells. These are T-cells that are isolated from a patient’s blood, genetically modified to target the cancer cells, then infused back into the patient. Clinical trials of CAR-T therapies have shown spectacular success, with complete response rates as high as 93% in some early trials (

Is Digital Health ever going to become mainstream?

I’ve been involved with Digital Health since 2012. At that time, the industry was espousing how Digital Health was ready to explode – applications were going to be prescribed by physicians; men, women, and children would readily adopt these solutions as they had Facebook and texting; physicians would be “led” through artificial intelligence to make better-informed diagnoses and treatments. While there have been some interesting pilots and plenty of new entrants into the market, one would be hard-pressed to point to more than a few dozen products or services that have gained substantial traction over the last 5 years. This despite approximately $4.5 billion of venture capital invested in 20

Pharmacy Benefit Managers (PBMs): Bending the cost curve in the wrong direction

In the NY Times article, “Take the Generic, Patients Are Told. Until They Are Not.”, Pharmacy Benefit Managers (PBMs) were called out for pushing branded products over generics to patients. PBMs are primarily responsible for developing and maintaining the formulary, contracting with pharmacies, negotiating discounts and rebates with drug manufacturers, and processing and paying prescription drug claims. They predominately work with insurers, self-insured companies and government programs striving to maintain or reduce the pharmacy expenditures of the plan while concurrently trying to improve healthcare outcomes.[1] In The New York Times article, UnitedHealthcare was called out for putting Ad

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