smart medication Gene
Collaboration platform and electronic patient record for gene therapy
Digital treatment process
Guidance through the entire gene therapy treatment process (manufacturer-independent for hemophilia A/B)
Electronic patient record
Structured documentation of the entire gene therapy and follow-up care
Cross-center collaboration
Collaboration based on the hub-and-spoke model
smart medication Gene is a collaboration platform with an electronic patient record for gene therapy in hemophilia A and B. It implements the hub-and-spoke model of EAHAD and EHC in software: hub and spoke centers run the entire treatment process in a structured, manufacturer-independent way, share findings, contracts and consent forms in encrypted form and document follow-up care in a shared record.
At a glance
- Target group
- Hemophilia treatment centers as hub (dosing) and spoke (home center, follow-up care), hemostaseologists
- Indication
- Gene therapy for hemophilia A and B, manufacturer-independent; usable for other gene therapies
- Model
- Hub & spoke according to the joint statement of EAHAD and EHC – overview: documentation and collaboration between centers
- Functions
- Guidance through the gene therapy process in sections, electronic patient record, checklists for eligibility criteria and tasks, laboratory and follow-up data, secure in-app communication, interface to electronic diaries
- Requirements covered
- Implementation of the requirements of G-BA, EMA, GTH and clinical registries; basis for the mandated long-term post-authorization studies
- Data transfer
- Encrypted between the connected centers
- Support
- Sponsored by BioMarin and CSL Behring; developed and operated by smart medication
Gene therapy for hemophilia
Gene therapy aims to actively treat a genetic defect in body cells. A single infusion replaces regular factor administration while still enabling long-term expression of the missing clotting factor. Even a small increase in factor activity leads to a marked improvement in symptoms – and thus in quality of life – for many patients.
The gene therapy from BioMarin for hemophilia A, medicine information from the EMA, opens in a new tab has held conditional approval in Europe since August 2022 (announcement by the Paul-Ehrlich-Institut, in German, opens in a new tab), but the manufacturer has been withdrawing it from the market, BioMarin statement, opens in a new tab since February 2026. The gene therapy from CSL Behring for hemophilia B, medicine information from the EMA, opens in a new tab has been approved and available since February 2023; in the U.S., the FDA had already approved it in November 2022, FDA press release, opens in a new tab. What this means for centers and documentation is explained on the knowledge page Gene therapy for hemophilia: documentation and collaboration between centers.
The hub-and-spoke model
Because the new gene therapies for hemophilia bring challenges for the treatment process, EAHAD and EHC introduced the so-called hub-and-spoke model, joint statement by EAHAD and EHC, opens in a new tab. The concept aims at effective communication and collaboration between the hemophilia centers involved in a patient’s gene therapy, to support shared treatment decisions for long-term benefit.
Large hemophilia centers that hold a high level of specialist knowledge about gene therapy and offer gene therapy to patients are called “hubs”. The hub center is responsible for the practical delivery of the gene therapy and thus for all tasks related to the infusion.
The “spokes” are the patients’ home centers, where they receive regular care. The spokes are responsible for advising patients as part of an informed decision-making process. Follow-up care after the infusion, including regular check-ups, is meant to take place locally at the home center.
Implementation of the hub-and-spoke model in the software
All content of the diagram as text
Collaboration tool to support shared treatment decisions for long-term benefit
- Guidance through the entire gene therapy process
- Encrypted transfer of patient data between networked centers
- Structured documentation of the entire gene therapy
- Cross-center collaboration to optimize the GT over time
- Implementation of the requirements of the G-BA (Federal Joint Committee), EMA, GTH (Society of Thrombosis and Haemostasis Research), and clinical registries
- Interface to electronic diaries for the course of treatment
- Secure in-app communication between centers
- Documentation of laboratory results / follow-up data in the patient record
- Checklists for eligibility criteria and tasks in the GT process
- Provision of relevant information such as brochures, checklists, or videos
So the focus can be entirely on the patient.
With Gene, we have developed a collaboration platform that supports physicians throughout the entire gene therapy treatment process for hemophilia.
Contracts with partner hospitals, consent forms, physician letters, and laboratory reports are shared within Gene and filed in a structured way in center and patient records. Fax machines and paper binders are a thing of the past.
Documentation made easy.
Together with hemostaseologists, pharmaceutical manufacturers, and pharmacists, a standardized treatment process was developed in sections, covering all legal and medical requirements of gene therapy.
Thanks to its structured, straightforward data capture, the patient record is particularly well suited to long-term observation in the sense of a clinical registry and provides the basis for the long-term observational studies required by legislators.
Everything about gene therapy in one place
smart medication Gene makes documentation around gene therapy for hemophilia A/B simple and intuitive. Focus entirely on your patient and connect with other specialized treatment centers.
Frequently asked questions
What is the hub-and-spoke model in gene therapy?
Is smart medication Gene tied to a manufacturer?
What is documented in Gene?
Is Gene suitable for long-term observation and registries?
How do hub and spoke centers communicate with each other?
News and publications about smart medication Gene
smart medication Gene is the digital solution for the hub-and-spoke model in gene therapy. Beyond hemophilia, Gene can also be used for other gene therapies.
For further information, feel free to contact us.
