NEJM Highlights August 2015: cancer and joints

The emergence of a new approach to drug development in cancer

Cancers are classified by the organ or tissue from which they arise, but as our molecular understanding increases, another level of categorization is emerging based on the molecular characteristics of the tumor. In a novel but sure to be growing approach, Roche/Genentech tested their drug vemurafenib (Zelboraf, currently approved for melanomas with the BRAF V600 mutation) in a study population that was largely agnostic to tumor provenance as long as it was BRAF V600 positive. 122 patients were enrolled with more than half a dozen cancers represented – the overall response rate was 43%.  One can envision a future where a development and approval program would be centered on a biochemical signature with an end point that instead of being e.g. “approval for use in pancreatic cancer” becomes “approval for use in tumors with biochemical signature X”.  Vemurafenib in Multiple Nonmelanoma Cancers with BRAF V600 Mutations; Let’s Not Put All Our Eggs in One Basket (subscriber access)


Progress in multiple myeloma

About 25,000 patients are diagnosed with multiple myeloma yearly in the US and the disease, despite being initially treatable usually eventually leads to death. In this phase 1-2 study, a monoclonal antibody against a marker of myeloma cells (daratumumab, Janssen) was given as monotherapy to 42 (for the max dose arm) patients who had essentially reached the end of their therapeutic options. The response rate was 36%, impressive considering the population, and possibly greatly improvable with patients earlier in their disease and with multidrug therapy.  An antibody from Sanofi with the same target is also in development although it is 1-2 years behind.  Targeting CD38 with Daratumumab Monotherapy in Multiple Myeloma; Monoclonal Antibodies in Multiple Myeloma Come of Age (open access)


Bundling the whole course of care for joint replacement

After some experimentation under the ACA, Medicare is now proposing to define all interventions associated with total knee or hip replacements as a single pre-agreed payment for a 90-day episode of care, including all post-acute care. This considerably extends the old DRG model that was limited to the hospital phase (which comprised 50% of costs) and fits into a drive toward an accountable integrated continuum of care by increasing the risk of downside (and upside subject to quality metrics) to providers. If this approach goes forward, any hospital that provides joint replacements will need to have very well defined relationships with post-acute providers (if only to decide how the monies will divvied up), and likely these relationships would seed broad ranging collaborations well beyond joint replacement. A main concern that has been raised is that given that the median number of joint replacement procedures is 90, hospitals with low volumes will be highly vulnerable to outliers who can easily end up costing 4 times as much as a usual patient. A variety of special dispositions to deal with low volume hospitals are being contemplated – but really, as discussed here, it is not in the best interest of patients to go to a low volume hospital anyway and trends will continue to drive consolidation if only for quality purposes. Mandatory Medicare Bundled Payment — Is It Ready for Prime Time? (open 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 a brief overview of highlights that might be of interest to our clients and others.

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