Posted by on in NEJM Highlights

Taking stock: two decades of progress in heart failure: Here comes a clever study using existing clinical trial data to assess progress in standard of care over time for heart failure. For each trial, the authors assessed the rate of sudden cardiac death during the early part of the study (excluding patients with ICDs), and it appears that between 1995 and 2014, it decreased by nearly half.  As always, in observational retrospective studies, one has to worry about systematic biases around the population that are included (i.e. are they really the same over time?), but there is a likely a… Read More

Posted by on in NEJM Highlights

A hammer finds new nails (which happen to be eyeballs) The insulin growth factor receptor 1 (IGF-1R) was once upon a time a popular cancer target pursued by multiple biopharmas each with their own humanized antibody, and each without much success. In 2013, River Vision licensed the Roche compound teprotumumab, to treat Graves’ ophthalmopathy, a condition in which hyperactivity of the thyroid gland causes (among many other issues) bulging eyeballs with esthetic, comfort, and sometimes severe visual implications for which treatment options are limited. Nobody quite knows why the ocular issues occur, but hyperactivity of fibroblasts which are cells that… Read More

Posted by on in NEJM Highlights

Calendaring care The length of our sidereal year is an accident – we happen to be circling a G2 star from which the habitable zone where free surface liquid water can exist lies at around 150,000,000 km; by Newton’s laws this in turn corresponds to an orbital period that is our year.  Even if it is not particularly strongly connected to the underlying human biology, a lot of healthcare cycles are aligned to the Earth Year for convenience but is it effective and efficient? Individuals with diabetes are at risk for diabetic retinopathy which can cause blindness and for which… Read More

Posted by on in Biopharma, Payers, Population Health

Summary Drug companies are naturally incentivized to price their drugs under assumptions of optimal clinical value, i.e. as high as possible.  Payers react to this by setting stringent conditions for patient eligibility for coverage of those therapies. As a consequence, patients who do not meet these conditions do not receive those drugs even though they could derive benefit, albeit not of a magnitude that would justify the cost.  Here we lay out a population health based scheme by which payers and drug companies can design a system that ensures access to a drug to a larger group who could benefit… Read More

Posted by on in NEJM Highlights

Gene therapy for sickle cell disease Typical diseases targeted by gene therapy are those for which there is a defect that prevents the production of a functional protein needed for normal life; remediation is achieved by inserting functioning copies of the gene, and fortunately, it is usually the case that expression at a low level is sufficient to greatly improve outcomes. The situation is different in sickle cell where the defective hemoglobin is actually harmful, and where success of gene therapy requires not only production normal hemoglobin, but replacement of the defective hemoglobin. In an N-of-1 study, a French group… Read More

Posted by on in NEJM Highlights

Surgical volume and referral for surgery: The impact of surgical volume on outcomes has been well documented, but is it top of mind with physicians referring patients to surgery? Readers of the Journal were polled on a hypothetical scenario whereby a community physician would be referring a patient in need of a major surgical procedure to either a nearby community hospital with a well-respected general surgeon doing approximately 5 of these cases a year versus a tertiary medical center 40 miles away. The great majority of readers chose the option to refer to the tertiary medical center. This does not… Read More

Posted by on in NEJM Highlights

Systems biology finally gets real: an unexpected use for a diabetes drug Chronic Myelogenous Leukemia (CML) has been the poster child first for a disease with a precise genetic cause (the Philadelphia chromosome), and then for targeted drug design (with imatinib – Gleevec). Unfortunately, few patients achieve a complete response to therapy which means that they have to stay on drug indefinitely. This commentary highlights recent research which shows that pioglitazone (Actos), an approved diabetes drug that activates a specific cellular pathway (STAT5) can synergistically enhance treatment with Gleevec to achieve a potential cure.  While this particular work affects only… Read More

Posted by on in NEJM Highlights

Interventionalist treatment for stroke: In the 80s and 90s, treatment of myocardial infraction was greatly advanced by the introduction of systemic clot busting drugs (t-PA and others); further advance occurred in the 90s when it was shown that immediate cardiac catheterization produced even better results. Acute embolic stroke has followed the same path – in the 90s, it was shown that t-PA treatment within 3 hours of onset of symptoms was beneficial, and ever since there has been a move toward treatment modalities where an interventional radiologist acts on the clot directly. Two randomized controlled studies now show unambiguously that… Read More

Posted by on in NEJM Highlights

The rise, fall, and rebirth of the Chinese healthcare system A fascinating account of the evolution of the Chinese healthcare system which almost seems to be an upside-down picture of the rest of the country’s development. Tremendous public health improvements occurred in the 50s, 60s, and 70s but the transition to a free market model of healthcare in the 80s seems to have been a disaster only mitigated by the general increase in wealth of the population. Seeing this as a major threat to social stability, the Chinese government has been trying to pick up the pieces with reforms in 2003… Read More

Posted by on in NEJM Highlights

A promising agent for Crohn’s Disease, a miserable illness Crohn’s is an inflammatory bowel disease that is notoriously unpredictable; flares can affect any part of the digestive tract and lead to grave complications. In this double-blind phase 2 study, patients were dosed with mongersen (licensed by Celgene) an anti-sense oligonucleotide that down-regulates the expression of a protein implicated in the inflammatory cascade. In general these classes of medications have to be given parenterally but in this case the target is the gut so it can be taken orally. At two weeks of treatment, the response rate was about 60% in the… Read More