Busy summer: An opinionated take on NEJM highlights for July/August 2019

Is the lack of new (non-viral) antibiotics a market failure?

A lament about the lack of success in getting the biopharma industry to invest sustainably in the development of new antibiotics against highly-resistant organisms, and a recommendation “it is time to seriously consider the establishment of nonprofit organizations for developing these lifesavings drugs”.  Let’s be clear; the reason economic viability of this therapeutic area is problematic is due to three confluent factors: (1) treatment is curative – i.e. does not provide recurrent revenues (2) the targeted population is small (3) unmet need is unpredictable. On its face, the risk-reward ratio is not attractive for return conscious investors, and given the political reluctance to use public monies to subsidize biopharma shareholders, that leaves the option of publically funding non-profits. One wonders though, about what happens if all programs fail – do we keep throwing money at the non-profit, and conversely, if there is an amazing windfall from a successful product, do we force the non-profit to spend the money on more antibiotic programs regardless of relative need? Sustainable Discovery and Development of Antibiotics — Is a Nonprofit Approach the Future? (subscriber access)


Two new drug classes in cancer

Whereas we’d be lucky to get two new antibiotic drug classes on the market in a decade, we get two new cancer drug classes in the journal in two months. First a fibroblast growth factor receptor (FGFR) specific tyrosine kinase inhibitor (class lead: erdafitinib – Balversa, Janssen) showing effectiveness in advanced urothelial carcinoma with FGFR3 mutations. Second a blocker of exportin 1 (XPO1), a protein that shuttles cargo from the nucleus to cytoplasm (class lead: selinexor – Xpovio, Karyopharm) showing effectiveness in patients with very advanced refractory multiple myeloma.  Both studies were small (~100), single arm (no placebo), monotherapy (though selinexor was accompanied with dexamethasone) in patients with few options. In keeping with a new, less risk averse calculus at the FDA, both studies have led to accelerated FDA approvals, conditional to confirmatory studies.   Such a pace in the development of new targets will likely lead to more than 100 distinct drug classes in the treatment of cancers in the next decade. At some point, the old model of oncology education, training, and practice is no longer going to be appropriate. We should be thinking about what will replace it now. Erdafitinib in Locally Advanced or Metastatic Urothelial Carcinoma; Oral Selinexor–Dexamethasone for Triple-Class Refractory Multiple Myeloma (subscriber access)


Large genomic-EHR cohorts in the US

I had never heard of the “All of Us” program which aims to aggregate 1M participants across the US with an enhanced focus on populations that are typically under-represented in biomedical research.  Interestingly, Fitbit and Walgreens clinics are partners (among others).  This joins the Geisinger/Regeneron collaboration as well the Million Veteran Program as one of the large studies in this quickly ripening area. More details here (participation) and here (data). The “All of Us” Research Program (open access)


Dredging up memories with electricity

A fascinating letter to the editor relating an incidental finding testing deep brain stimulation in Alzheimer’s (it does not work). In some patients, this induced vivid memory flashbacks where the strength of the applied voltage would change the level of detail of the flashback.  The human brain is a very strange organ. Fornix-Region Deep Brain Stimulation–Induced Memory Flashbacks in Alzheimer’s Disease (subscriber access)


A strong marketing effort

The back covers of my issues of the NEJM for July and August:



The New England Journal of Medicine is a premier weekly medical journal covering many topics of interest to the health sector. In this monthly series we offer an opinionated perspective on selected highlights that might be of interest to our clients and others.

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