Thursday, February 5, 2015

NEJM Highlights January 2015: Dengue vaccine, Informed consent, Cancer drugs and Tiering for selection

A vaccine for dengue finally nears the market
Dengue is a mosquito transmitted viral infection that is often severe and occasionally fatal, and that has been identified as a growing public health threat, largely in the developing world but also with inroads in developed countries with hundreds of millions of cases yearly world-wide. At this time, there is no vaccine or treatment for dengue other than supportive care. In a placebo-controlled study, a dengue vaccine from Sanofi-Pasteur covering all 4 serotypes of dengue was found to be 60% efficacious in preventing disease, and 80% efficacious in preventing hospitalization from dengue raising the likelihood that in the coming years, a dengue vaccine will finally become available. Efficacy of a Tetravalent Dengue Vaccine in Children in Latin America (p. 113 – 2015 Jan 8 issue)

The regulation of clinical research within the standard of care: high stakes, strong disagreements 
Skirmishes continue about research regulation in studies when the research is conducted within the bounds of the standard of care (i.e. no novel intervention). On one side, the Office for Human Research Protections (OHRP) pushing for stringent guidelines whenever subjects are assigned to separate treatment groups, in particular with regard to informed consent, whereas researchers are pushing for rules that make subject enrollment easier given that they will be receiving a treatment that is commonly administered outside a research protocol.  The ORHP rules are open for comments – the outcome could have far-reaching consequences ranging from value proofs for mobile health to process of care experimentation within ACOs. Foreseeable Risks? Informed Consent for Studies within the Standard of Care (p. 306 –2015 Jan 22 issue)

On-going breakthroughs in oncology: the PD-1 story continues across cancer types
The latest in a recent burst of publications about PD-1 blockade – PD-1 is a T-cell receptor that down-regulates the immune response – empirically it has been seen that many tumors over-express PD-L1, an activator of PD-1 which is thought to help them evade immunity, something that can be reversed by blocking access to PD-1 with an antibody such as nivolumab (BMS) and lambrolizumab (Merck SD). Two studies show remarkably high response rates in aggressive, but very different cancers (refractory Hodgkin’s and melanoma). No wonder that at the time this is written, 41 studies are open for nivolumab ranging from ovarian cancer to gliobastoma. The potential for agents with an extremely broad range of application is clearly there. PD-1 Blockade with Nivolumab in Relapsed or Refractory Hodgkin's Lymphoma (p. 311 – 2015 Jan 22 issue); Nivolumab in Previously Untreated Melanoma without BRAF Mutation (p. 320 – 2015 Jan 22 issue)

Pharmacy tiering as a participant selection tactic for health plans
A perspective article that follows-up on a complaint that some health plans have been using tiering of pharmacy benefits to discourage enrollment of certain classes of participants. In this case the focus is on NRTIs which are a mainstay of HIV therapy and where a tiering difference can make a difference of many thousands of dollars a year in co-insurance payments (that is, if Pharma does not foot the bill through patient assistance programs). In a review of data across 12 states, the authors find a stark differentiation of plan coverage with 25% of the plans creating highly adverse tiering for HIV patients and point out that such patterns compromise the risk-sharing under-pinning of the ACA. Using Drugs to Discriminate — Adverse Selection in the Insurance Marketplace (p. 399 – 2015 Jan 29 issue)


The New England Journal of Medicine is a premier medical journal covering many issues 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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