PREFER PAVING: GENE THERAPY IN HAEMOPHILIA

An online learning to inform patients and assess patient preferences.

PREFER is a 5-year, IMI-funded, project that aims to investigate how and when patient preferences regarding the benefit/risk of drugs can be used in the medical product lifecycle. As part of the project, researchers are evaluating various research questions in clinical patient preference case studies.

One of the case studies being evaluated in PREFER is in the area of haemophilia. The PAVING case study is led by KU Leuven. To support this case study, MindBytes developed interactive educational tools to inform patients on several aspects of the study:

The study objective
The concept, benefits and risks of gene therapy

PREFER PAVING Gene therapy in haemophilia_on demand treatment_person

PREFER PAVING Gene therapy in haemophilia_treatment explanation

PREFER PAVING Gene therapy in haemophilia_treatment explanation graph

PREFER PAVING Gene therapy in haemophilia_treatment explanation vector

Rare disease

Haemophilia is a rare genetic bleeding disorder occurring in 1 of 10 000 births. There are two types, A and B, caused by different gene defects. These genetic errors cause patients to bleed longer. These bleeds can cause a variable range of other medical issues.

Lower burden of treatment

Current therapies are based on increasing coagulation factor concentrations through factor replacement. Invasiveness of this therapy (intravenous injection) and high administration frequency causes a high burden of treatment. Recently, gene therapy for the treatment of haemophilia has been developed. This seems very promising and may be able to cure patients, without such a high burden of treatment. However, it is currently still unknown what long-term side effects may occur and whether the therapeutic effect of gene therapy will be maintained throughout the full lifespan of patients.

Choice between SoC & gene therapy

In this Patient preferences to Assess Value IN Gene therapies (PAVING) case study, the team evaluates how features of the standard therapy (SoC) and gene therapy influence patients’ choices between both.