Posted by on in Biopharma

The public debate on drug pricing has sharpened markedly over the past year. We are seeing more political scrutiny and media coverage, including the blowback on Mylan’s EpiPen pricing, tweets from now President Trump, as well as an unsuccessful California ballot initiative to force lower drug prices. This is all on top of a backdrop of seemingly ever-increasing coverage of the high costs of new medicines and double-digit price increases. Now market forces may be gearing up: in the past four months, Anthem — one of the “Big 3” payers covering 40 million lives or 1 in 8 Americans– has… Read More

Posted by on in NEJM Highlights

Successful use of CAR-T therapy in a solid tumor Chimeric Antigen Reception T-cells (CAR-T) are immune cells molecularly engineered to seek out and destroy cancer cells; the push to develop them into a scalable generally usable treatment is likely the most exciting challenge in cancer right now.  Successful CAR-T use has so far been generally confined to hematological tumors.  In a brief report, a group from City of Hope reports on the use of CAR-T in a patient who was dying from an advanced, aggressive form of brain cancer, which led to a regression of the tumors for several months. Read More

Posted by on in NEJM Highlights

“My name is T-Cell…, James T-Cell” Immune T-cells are licensed to kill other cells through a quick molecular kiss of death, and as such are potentially powerful allies in controlling a tumor. For obvious reasons this killing power is under strict regulatory control and in particular T-cells display PD-1 proteins on their surface, which when engaged by the ligand PD-L1 on another cell, protects that cell from being killed. Tumors often display high levels of PD-L1 so that disrupting the interaction between PD-L1 and PD-1 can enhance the effectiveness of immune killing of tumor cells. Amplifying results from last year… Read More

Posted by on in NEJM Highlights, Providers

I am not sure how many docs continue to do this, but I still read the actual hard copy of my NEJM, and that means I flip past ad pages with smiling grandfathers playing with grandchildren thanks to supercalifragilistic products on my way to scholarly papers with tables and figures.  But this time, I stopped in puzzlement when I came across exhibit 1; Intermountain is a health system based in Utah, very highly respected for its sound approach to quality and cost control[1], but not broadly well known for cancer care in the way of centers like Dana Farber… Read More

Posted by on in NEJM Highlights

Continued progress in multiple myeloma About 25,000 patients are diagnosed with multiple myeloma yearly in the US. Despite being initially treatable, typically this disease is ultimately lethal. Following a highly successful phase 1-2 study a monoclonal antibody against a marker of myeloma cells (daratumumab, Janssen) underwent phase 3 studies in combinations with established mainstays of therapy (the proteasome inhibitor bortezomib and the immune modulator lenalinomide) in a patient population several years out from their initial diagnosis.  Results were stellar, with the inclusion of daratumumab decreasing the disease progression rate by half at 1 year.  Daratumumab, Lenalidomide, and Dexamethasone… Read More

Posted by on in NEJM Highlights

Taking a page from HIV to build a response to opioid abuse A couple of perspectives on the challenges of treating individuals who suffer from opioid dependence. The first highlights the importance of integrating medication assisted treatment (e.g. methadone or buprenorphine) into hospital and post-hospital care – plausibly an ED visit or a hospital stay for an event triggered by opioid abuse (such as an overdose) is a significant opportunity to go beyond treating the acute issue and starting patients on long term treatments. In the second, the author recalls the tremendous progress in treating HIV/AIDS in the early 90s… Read More

Posted by on in NEJM Highlights

There is such a thing as too much hygiene The prevalence of asthma in children has increased dramatically over the last few decades. Observational studies have shown that children in “dirty” environments such as farms seem to be relatively protected from asthma.  A theory is that the lack of exposure to microbes leads to higher sensitivity to allergens, but this causality has been hard to show. Amish and Hutterite farm communities are genetically similar, but Amish rely on animals instead of machinery, and Amish children have much lower incidence of asthma. In a fascinating study comparing Amish and Hutterite, it… Read More

Posted by on in Consumer Health, Digital Health, Providers

Summary Cheap home devices are starting to generate a flood of high frequency, low latency biometric data, much of it of uncertain clinical value This uncertainty makes designing the service model difficult: high value use cases may get bundled with broader, low value, more speculative ones (e.g. behavior change), reducing overall ROI and uptake Given the patient-generated nature of the data and uncertain accuracy / calibration of the devices, use cases will need specific targeting or depend on subsequent clinical grade investigation to sort signal from noise High value use cases are likely going to require tightly designed delivery models… Read More

Posted by on in NEJM Highlights

Adaptive clinical trials slowly coming of age In an adaptive clinical trial, the protocol of the trial is allowed to change in a pre-specified manner during the study based on on-going study events.  In this issue of the NEJM, two research papers, one perspective, and one editorial are devoted to the I-SPY 2 trials which dynamically changed randomization procedures for neo-adjuvant (pre-surgery) chemotherapy for stage II and III breast cancer and allowed accelerated identification of subgroups that benefit from a novel tyrosine kinase inhibitor (neratinib – Puma Biotechnology) and a PARP inhibitor (veliparib – Abbvie).  Adaptive trials are described in… Read More

Posted by on in NEJM Highlights, Population Health

Disappointing interim results from two ACA experiments Two papers reporting results from ACA experiments – the Comprehensive Primary Care (CPC) Initiative in which primary practices were incentivized with fairly generous payments to strengthen care management activities such as management of chronic conditions, or coordination of care – and the ACO initiatives (2012 cohort) described elsewhere in many reviews. Both papers provide a view on the early impact of these initiatives (2 years out) on costs and outcomes by using well controlled no-intervention comparison groups. The upshot is that so far, the CPC and ACO (v. 2012) initiatives do not show… Read More