Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study.
Eline van OverbeekeSissel MichelsenBrett HauberKathelijne PeerlinckCedric HermansCatherine Marie LambertMichel GoldmanSteven SimoensIsabelle HuysPublished in: Haemophilia : the official journal of the World Federation of Hemophilia (2020)
This qualitative study showed that most PWH have a positive attitude towards gene therapy and that besides efficacy, safety and the related uncertainties, also impact on daily life is important to patients. The identified patient-relevant attributes may be used by regulators, health technology assessment bodies and payers in their evaluation of gene therapies for haemophilia. Moreover, they may inform clinical trial design, pay-for-performance schemes and real-world evidence studies.
Keyphrases
- gene therapy
- clinical trial
- end stage renal disease
- case report
- healthcare
- newly diagnosed
- ejection fraction
- public health
- chronic kidney disease
- prognostic factors
- peritoneal dialysis
- physical activity
- randomized controlled trial
- study protocol
- copy number
- dna methylation
- gene expression
- climate change
- phase ii
- phase iii
- case control
- drug induced
- clinical evaluation
- health promotion
- patient reported
- placebo controlled