Patient preferences to Assess Value IN Gene therapies: the PAVING study as an IMI PREFER case study

  • Date: –15:00
  • Location: Online
  • Lecturer: Isabelle Huys (KU Leuven), Brett Hauber (Pfizer), Eline van Overbeeke (Pfizer)
  • Contact person: Carl Steinbeisser
  • Seminarium

Decisions about whether to approve new and emerging gene therapies are impeded by the associated uncertainties, e.g. long-term outcomes. In this complex context, the PAVING study investigated haemophilia patients’ preferences for gene therapies. Learn about how the preference study was designed, what the results were, and what the impact might be in this webinar.

Gene therapies are innovative therapies that are increasingly being developed. However, health technology assessment (HTA) and payer decision-making on these therapies is impeded by uncertainties, especially regarding long-term outcomes. Through measuring patient preferences regarding gene therapies, the importance of unique elements that go beyond health gain can be quantified and inform value assessments. Therefore, the Patient preferences to Assess Value IN Gene therapies (PAVING) study was conducted to investigate treatment attributes important to haemophilia patients, and the trade-offs they are willing to make when asked to choose between a standard of care and gene therapy. During this webinar, insights will be shared on how a patient preference study was designed in the complex context of gene therapies, as well as the results and their potential impact. 

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