Company insight
Bridging innovation and access: A CDx collaboration journey in haemophilia care
Companion diagnostics (CDx) are transforming personalised medicine, especially for rare diseases like haemophilia. Randox has successfully developed and commercialised the ConcizuTrace ELISA, a CDx supporting concizumab therapy for haemophilia A and B.
aemophilia, an inherited bleeding disorder caused by a deficiency of clotting factors VIII (haemophilia A) or IX (haemophilia B), leading to prolonged or spontaneous bleeding, affects over 800,000 people worldwide, with more than 200,000 having severe forms. Despite advances, only 20–30% of patients in low and middle-income countries receive proper preventive treatment.
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From bioanalytical manual ELISA to regulatory-ready CDx Randox, in partnership with Novo Nordisk, developed the ConcizuTrace ELISA to quantitatively measure Alhemo (concizumab-mtci) levels in human plasma, aiding dose adjustments per the drug label. Randox’s journey began with the technology transfer of the bioanalytical ELISA clinical trial assay (CTA). Randox developed this into an IVD CDx assay, developed under ISO 13485:2016 design control.
usable and applicable in a typical end-user setting representative of the intended use of the device. Intended use validation also included a multi-site study that was conducted across three laboratories (two in the US, one in the UK) to demonstrate reproducibility of the device. A critical requirement for regulatory approval of the ConcizuTrace ELISA was demonstrating the analytical and clinical performance in line with the CTA that was used throughout the drug clinical trials. A bridging study was conducted to assess the concordance between the ConcizuTrace ELISA and the CTA when contemporaneously measuring the same set of native patient samples to determine if the two assay methods provide concordant results within the preset analytical quality specifications.
Global regulatory strategy The ConcizuTrace assay was registered in
“The ConcizuTrace case shows how innovative diagnostics can improve rare disease care.”
Assay optimisation ensured regulatory standards for IVD approval while maintaining alignment with the original CTA for consistent performance. Following assay design lock, design verification was completed in accordance with CLSI guidelines (e.g., EP05-A3 for reproducibility, EP25-A for stability). These studies demonstrated the analytical performance of the ConcizuTrace ELISA in the final kit format.
End-user validation studies were conducted, also in line with CLSI guidance, to ensure the ConcizuTrace ELISA was
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more than 40 countries with tailored regulatory strategies. Its classification varied by country, as either a companion diagnostic or monitoring tool. Close collaboration between Randox and Novo Nordisk teams helped navigate diverse requirements and speed approvals. Randox’s co-commercialisation aligned the CDx launch with the drug rollout, ensuring coordinated global execution and support for treatment access in this rare disease: ■
from blood collection through to sample reporting directly to the physician.
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Sample collection kit: To support sample collection and ensure standardisation, Randox developed specific barcoded kits tailored to country-specific regulations.
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Laboratory network: Qualification of laboratory partners performing the ConcizuTrace ELISA within a global, closed laboratory network ensuring standardised assay execution and controlled data reporting.
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Customer support: Based on the global availability of the therapeutic, Randox has ensured global support across seven key languages, as well as multilingual resources and training videos hosted on country-specific resource hubs.
Lessons learned
Key insights from the journey include: ■
early engagement with regulatory authorities is critical
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end-user validation in intended settings enhances adoption of the solution into market
in-house manufacturing capabilities ensure long-term supply stability
strategic partnerships mitigate commercialisation risks and accelerate market access
The ConcizuTrace case shows how innovative diagnostics, combined with collaboration and global commercialisation, can improve rare disease care. This model offers a blueprint for integrating diagnostics and therapeutics to provide personalised, equitable healthcare worldwide. ●
Web portal: A dedicated platform was designed that enables sample traceability
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