Struggling: An opinionated take on NEJM highlights for May 2018

One more on the chin for Alzheimer’s

A report of a large phase 3 study of the highly potent oral BACE inhibitor verubecestat (Merck) with yet again a lack of therapeutic effect, despite a dramatic reduction of the cerebrospinal fluid content of beta amyloid in various forms.  With a string of prior failures, this may be the near final blow for amyloid as a treatment target for Alzheimer’s disease (and Biogen’s adacanumab would be the end of the line if it also comes up tails). But note that the study population, although described as “mild-to-moderate”, clearly has sustained substantial brain damage by the time an attempt is made to address the disease process.  If amyloid is truly a root cause of Alzheimer, then the winning therapeutic approach is perhaps prevention rather than treatment. Unfortunately, proving (or disproving) this is hard as it would require following large cohorts over long timelines (5-10 years?).  Randomized Trial of Verubecestat for Mild-to-Moderate Alzheimer’s Disease (subscriber access)


The supply and price of old drugs

Monopolistic pricing of new drugs protected by patent is supposed to drive innovation – and it does, I see it every day from my office in Kendall square. Conversely, monopolistic pricing of old drugs that have lost exclusivity is supposed to draw additional manufacturers thus lowering prices – but unfortunately, there have been multiple well-publicized instances where this has not worked well. Typically this occurs either for drugs that address very small populations where a cost plus business model is not an attractive proposition, or for IV drugs where the time and effort to get manufacturing set-up and regulatory approval is significant. Enter Project Rx, an effort mostly driven by providers to set-up a non-profit manufacturer to counter these issues. From a provider economics perspective, this can make sense especially under alternative payment models where a system may take risk on total cost of care including hospital drugs. The authors mostly argue for the feasibility of such a scheme but one element they do not consider is the potential consequence of success. Market failures occur when players are deterred to enter a market and that could easily happen if they see a competitor with deep pockets and no profit motive. Project Rx will have to strike a careful balance between filling gaps in the supply of drugs vs. scaring off generic manufacturers and morphing a competitive market into a plannified economy.  Addressing Generic-Drug Market Failures — The Case for Establishing a Nonprofit Manufacturer (free access)


The worst medical care in the world

Healthcare delivery is an approximate and unfortunately error-prone affair, but it is usually self-correcting at the individual level because patients that continue to be unwell or are getting worse will instinctively continue to knock on doors until their issues are addressed. An exception is mental illness, where an increase in severity of disease often leads to less seeking of care, and when care is given, a reduced level of credibility given to what the patient says. Read this article by a distraught brother and learn how a mentally ill patient can be in the system for months without anyone noticing that somewhere along the line, he became paralyzed from the neck down.  In these situations, there are two ways to receive good medical care – one is to have one physician in charge who feels truly committed to the well-being of the patient, the other is to have a fully developed standardized approach to quality care. Unfortunately, the first way is not scalable, and we are not there with the second.  The Best Medical Care in the World (free access)


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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