[New, more expensive - Not necessarily better]. / Nouveau et (beaucoup) plus cher ­ Meilleur ? - Chroniques génomiques.

The prices of new oncology drugs are frequently above 100,000 US dollars, and this does not generally correlate with significantly improved clinical efficacy. In the absence of effective regulation and of real competition, companies tend to charge « what the market can bear ¼. Regulatory intervention is required, notably at the EU level.

[A long-awaited - but prohibitively expensive - therapy]. / Une thérapie très attendue et un prix exorbitant ! - Chroniques génomiques.

After many years of development, a gene therapy for haemophilia B has demonstrated effectiveness and durability, at least over a time span of three years. It has just been approved (as Hemgenix®) by the FDA in the USA and by the EMA in the EU. However, the multimillion price tag of this treatment is a serious problem for healthcare systems and for patients, and raises a number of thorny issues about marketing practices of the pharmaceutical industry.

[Cost/benefit analysis of cancer drugs]. / Bénéfice clinique et coût des traitements anticancéreux.

A detailed analysis of the clinical benefit for 47 approved cancer drugs, using two internationally recognized assessment systems, shows essentially no correlation between clinical benefit and weekly treatment costs. This is true both in the USA and in four European countries, although prices are dramatically lower in Europe.

[An update on DNA sequencing]. / Actualités du séquençage d'ADN - Chroniques génomiques.

The planned acquisition of Pacific Biosciences by Illumina could signal a big change in the DNA sequencing landscape, and provides the incentive for an update on a field that still has a number of players and could be disrupted by innovative technologies.

[From HGP-write to « Ultra-safe Cells ¼]. / De HGP-write aux « super-cellules ¼ - Chroniques génomiques.

The HGP-write project, announced in 2016 but not really implemented yet, comes back as a project aimed at constructing an "ultra-safe" human cell line fully resistant to virus infection and with other desirable characteristics. This involves introducing 400,000 changes in the genome and raises a number of technical and financial issues, but may become realistic in mid-term.

[A pragmatic approach to managing expensive therapies]. / Une approche pragmatique pour gérer les traitements onéreux - Chroniques génomiques.

Inflated drug prices necessarily raise the issue of rational allocation of health care resources. The system operated by the NICE agency in the UK attempts to do this by calculating the cost per quality-adjusted life year gained (QALY) and recommending funding only for drugs whose cost per QALY falls under a certain threshold. The whole process is documented in detail and easily accessible, and often results in significant discounts on drug prices. Given that some kind of rationing of health care is inevitable, the rational and transparent process followed by NICE has a number of positive features.

[Nanopore DNA sequencing becomes a reality]. / Séquençage d'ADN : l'offensive des nanopores - Chroniques génomiques.

A practical DNA sequencer based on nanopores has now demonstrated interesting performance and may be on the way to capturing a significant slice of the sequencing market while opening up new application fields thanks to its portability and low capital cost.

[Synthesizing a human genome?] / HGP-write : après la lecture, l'écriture ? - Chroniques génomiques.

The recently proposed « HGP-write ¼ project aims to synthetize a full human genome and to introduce it into cells. This ambitious endeavour is fraught with financial and technical uncertainties and, if successful, would make « synthetic humans ¼ a definite possibility even though this is not part of its announced goals. Accordingly, it has not been received with enthusiasm.

[Does a cancer moonshot make sense?]. / Cancer :faut-il viser la lune ? - Chroniques génomiques.

The recently announced "Cancer moonshot" may be greeted with scepticism: the previous "War on cancer" (1971) was not very successful. However the current plan seems well targeted, with emphasis not only on research investment but also on a strong effort to "break down silos" and ensure wide dissemination and use of clinical and molecular data. One big uncertainty, however, is what will happen to the programme after the 2016 presidential election… ‡.

[23andMe and motion sickness]. / 23andMe et le mal des transports - Chroniques génomiques.

A Genome Wide Association Study on propensity to motion sickness published by 23andMe gives interesting results, shows validity for self-reported phenotypic information and underlines the value of the model developed by the company for customer participation in genetic studies.

[CRISPR-Cas9, a new chance for somatic gene therapy]. / CRISPR-Cas9, une nouvelle donne pour la thérapie génique - Chroniques génomiques.

Targeted modification of genes ("gene editing") is made much easier by the recently developed CRISPR-Cas9 system. This has raised alarm about possible uses of this technology for germline modification of the human genome; however this technology has less controversial applications, notably for somatic gene therapy with already some striking demonstrations in animal systems. Because of its precision and relative ease of use, CRISPR can be expected to drive a revolution in gene therapy and to turn it into a more mainstream approach.

[Sequencing babies?]. / Séquencer les bébés ? - Chroniques génomiques.

An extension of newborn screening to genome sequencing is now feasible but raises a number of scientific, organisational and ethical issues. This is being explored in discussions and in several funded trials, in order to maximize benefits and avoid some identified risks. As some companies are already offering such a service, this is quite an urgent matter.

[Desperately seeking targets]. / Recherche cibles, désespérément - Chroniques génomiques.

In clinical oncology, obtaining sequence data from tumour samples has become practical in terms of logistics, turnover time and costs. The major issue now is the interpretation of this sequence to define actionable targets, i.e. genome changes that are involved in tumorogenesis and that predict the efficacy of existing and available targeted treatments. This is the focus of major efforts from public clinical centres and from companies that see it as a promising commercial activity.

[Monshot projects in biology]. / Course à la lune en biologie - chroniques génomiques.

Three recent and very large projects aim to integrate several "omics" approaches in order to promote pro-active medicine and to create a "Wellness industry".

[A surprising commercial success]. / Chroniques génomiques - Un triomphe commercial surprenant.

Direct-to-consumer (DTC) genetic tests have become quite popular in the USA. This article explores the uses to which they are put and the motivations of the customers.

[Eating my hat?]. / Un chapeau pour mon repas ?

After clearing many hurdles, gene therapy is now reaching the commercialization stage and can be expected to make a modest but real contribution to treatment in the near future.