Haemophilia is a rare bleeding disorder that affects roughly 1 in 5,000-25,000 men world wide. It is characterised by a deficiency or defect of specific blood clotting factors causing spontaneous bleeding episodes, compounded by the development of joint damage in later life. Fortunately, the introduction of recombinant factor concentrates has greatly improved the quality-of-live for this patient group, although the treatment often remains very expensive (in the order of 500,000 to 1,000,000 euros per patient per year).

The Netherlands has long been at the forefront for the push for treatment optimisation to bring down costs and improve the allocation of treatment to patients who need it the most. The SYMPHONY consortium was conceptualised in 2019 and aims to improve diagnostics and treatment personalisation by birthing national collaboration between the five largest haemophilia treatment centres in The Netherlands. In 2020, I joined this research consortium as a PhD-student to develop AI-based methodology to improve our models and to develop an online web-application to deliver these models to physicians in The Netherlands (and currently operating world-wide).

I personally designed, developed, and maintain the OPTI-CLOT web portal (opticlot.com): an online web-application that allows physicians and nurses to request tailored dosing advice for their patients suffering from rare bleeding disorders. The application allows users to securely share sensitive medical information of their patients to our centralised system at the AmsterdamUMC while a team of expert pharmacometricians provide personalised dosing advice based on the needs of the patient. Currently, over 200 patients have received dosing advice through the system (which accounts for roughly 14% of all patients with Haemophilia in The Netherlands).

During the development process, I have had extensive discussions with legal staff, privacy officers, and IT security personnel to ensure the application meets the rigorous standards set for medical devices. I also set up the server that hosts the application as well as maintain the data base services. I am currently in the process of transferring these responsibilities to our IT staff at the pharmacy of the AmsterdamUMC.

The web application is also used in several clinical trials that focus on evaluating the benefits of personalised treatment strategies versus traditional dosing strategies. In The Netherlands, we support the DosEmi clinical trial by providing dosing advice as well as supporting the clinical with clinical implementation of the trial. The latter for example includes leading discussions surrounding optimising the measurement assays to improve accuracy. This trial has already achieved relevant costs reductions (in the order of millions of euros) while so far pointing to similar efficacy. Internationally, a clinical trial investigating the benefits of personalised treatment using a novel drug to treat patients with von Willebrand disease will also be run through the web portal.

The web portal is currently expanding to provide personalised advice for patients world-wide.