A computer-based model to assess costs associated with the use of factor VIII and factor IX one-stage and chromogenic activity assays.J Thromb Haemost 2016; 14: 757-64. EssentialsChromogenic factor VIII and factor IX assays have not been widely adopted partly due to perceived cost. Chromogenic assays may be needed for monitoring therapy with some extended half-life concentrates. Efficient use of reagents makes the costs of chromogenic and one-stage factor IX assays similar. A similar approach for factor VIII delivers lower costs for chromogenic compared with one-stage assays.Summary. Background: Measurement of coagulation factor factor VIII (FVIII) and factor IX (FIX) activity can be associated with a high level of variability using onestage assays based on activated partial thromboplastin time (APTT). Chromogenic assays show less variability, but are less commonly used in clinical laboratories. In addition, one-stage assay accuracy using certain reagent and instrument combinations is compromised by some modified recombinant factor concentrates. Reluctance among some in the hematology laboratory community to adopt the use of chromogenic assays may be partly attributable to lack of familiarity and perceived higher associated costs. Objectives: To identify and characterize key cost parameters associated with one-stage APTT and chromogenic assays for FVIII and FIX activity using a computer-based cost analysis model. Methods: A cost model for FVIII and FIX chromogenic assays relative to APTT assays was generated using assumptions derived from interviews with hematologists and laboratory scientists, common clinical laboratory practise, manufacturer list prices and assay kit configurations. Results: Key factors that contribute to costs are factor-deficient plasma and kit reagents for one-stage and chromogenic assays, respectively. The stability of chromogenic assay kit reagents also limits the cost efficiency compared with APTT testing. Costs for chromogenic assays might be reduced by 50-75% using batch testing, aliquoting and freezing of kit reagents. Conclusions: Both batch testing and aliquoting of chromogenic kit reagents might improve cost efficiency for FVIII and FIX chromogenic assays, but would require validation. Laboratory validation and regulatory approval as well as education and training in the use of chromogenic assays might facilitate wider adoption by clinical laboratories.

This Industry Update covers the period 1–31 March 2014 and is based on information sourced from company press releases, scientific literature, patents and various news websites. This month saw some significant commercial activity with Baxter (IL, USA) announcing a split, Corium an initial public offering and Vectura a major acquisition, which both expands its development pipeline and also gives it access to another inhalation device platform. A number of technologies are under development that enable the transfer of drugs across the blood–brain barrier and MedImmune (MD, USA) continued its interest in the area by announcing a new deal with BiOasis (Vancouver, Canada). In another drug-delivery deal, Caisson (TX, USA) announced an expansion of its HEPtune collaboration with Novo Nordisk (Bagsværd, Denmark). Clinical progress was reported by Arrowhead Research Corporation for its RNAi therapeutic for the treatment of chronic hepatitis, which began a Phase IIa study, and Ocular Therapeutix (MA, USA) embarked on its Phase III clinical program to evaluate the safety and efficacy of its dexamethasone formulation. In the regulatory arena, the US FDA approved oral treatment from Celegene (NJ, USA) for psoriatic arthritis and Avanir (CA, USA) announced the FDA has accepted its new drug application for a nasally delivered sumatriptan for the treatment of migraine. A paper in Nature described an 'on-skin' system that can monitor patients, deliver drugs and communicate data. A paper published by a group form University of Illinois (IL, USA) described a biodegradable battery that could be used to power implanted devices used to deliver drugs.

This Industry Update covers the period from 1 January through 31 January, 2015, and is based on information sourced from company press releases, scientific literature, patents and various news websites. This month saw drugs from Shire and Janssen win fast track approval status from the US FDA which reduces the target time for the Agency to take action from 10 to 6 months. Sanofi and Regeneron also heard that they have won fast track approval for alirocumab a PCSK9 inhibitor for cholesterol lowering following their purchase of a ‘Priority Review Voucher’ from BioMarin. This is significant in their race to approval with Amgen. In the devices area, Unilife announced a new deal for their electronic autoinjector and Pari got approval for the use of their electronic eRapid to deliver Genentech's pulmozyme which is used in the treatment of cystic fibrosis. Following safety and tolerability studies, 3M announced that its Hollow Microstructured Transdermal System is now available for partnering. Medtronic announced the roll out of its new MiniMed insulin delivery system taking it a step closer to being able to offer a complete artificial pancreas. GSK expanded to four, the number of respiratory drugs that it provides in its new Ellipta inhaler in the US market. The University of Warwick announced a new means of targeting the delivery of drugs using nanoparticles which combine at the target site and release their drug payload. Adamis received a patent on its dry powder inhaler technology, acquired from 3M and TiGenix a patent in Europe for its tempo cell technology.

This Industry Update covers the period from 1–31 December 2013 and is based on information sourced from company press releases, scientific literature, patents and various news websites. In the parenteral area there was news about the development of pump technology for noninsulin applications, which provides evidence for the growth in demand for large volume subcutaneous drug delivery, a need that cannot be met using conventional injection technology. There was also considerable activity in the respiratory area with announcements on genetic competition to GlaxoSmithKline (London, UK) Advair/Seretide, a global top five selling drug as well as some news on the development and launch new respiratory drugs. The largest mergers and acquisitions deal was the much anticipated buyout of Bristol-Myers Squibb (NY, USA) by AstraZeneca (London, UK) from their diabetes joint venture and the second US $1 billion plus deal for AstraZeneca in 2013 as they strive to rebuild a late stage pipeline to offset the generic threat to their revenues over the next few years.