Health Preference Research in the Context of Gene Therapy in Rare Diseases

September 27, 2021

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Open to all ISPOR Members and Non-members


Moderated by:

  • Janine van Til, PhD

Presented by:

  • Holly Peay, PhD, MS
  • Lizheng Shi, PhD 
  • Eline van Overbeeke, PhD

Monday, September 27, 2021
10:00AM EDT | 3:00PM BST | 4:00PM CEST 

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Gene therapy is increasingly recognized as a potential treatment approach for a broad range of conditions. The promise of therapeutic and/or curative benefits through the modification of genetic material has offered hope to many patient groups. At the same time, the known and potential risks of gene therapy, the history of gene therapy in the US, and public attitudes fueled by strong media attention associated with this kind of treatment have resulted in public discussions about the desirability of gene therapy. The acceptable trade-off between potential benefits and harms is pertinent to the value of gene therapy for future patients.

During this webinar, the speakers will present their respective studies on gene therapy in Duchenne muscular dystrophy, spinal muscular atrophy (SMA) and hemophilia.  They will discuss the importance of the disease context, the qualitative research that influenced attribute selection, the selection of health preference method and design of the health preferences’ tasks, and how the study findings relate to the potential value of gene therapy.

The second half of this webinar will be reserved for a moderated discussion on the specific value of health preference research in this context, and the interpretation and implications on the outcomes and Q&A from the audience.
The audience will learn about:

1.    The types of specific knowledge gaps/research questions pertinent to gene therapies that can be answered using preference methods
2.    How gene therapy characteristics are reflected in attributes selection and operationalization
3.    The interpretation of study results, i.e., what can be concluded about factors that impact the value of gene therapy to patients and their treatment preferences.

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Brought to you by: Health Preference Research Special Interest Group


Janine van Til, PhD, Assistant Professor, Faculty of Behavioural, Management and Social Sciences, University of Twente, Enschede, Netherlands


Holly Peay, PhD, MS, Senior Research Scientist, RTI International, Research Triangle Park, NC, USA

Lizheng Shi, PhD,
Professor, Tulane University School of Public Health and Tropical Medicine, New Orleans, LA, USA

Eline van Overbeeke, PhD, Health Economics and Outcomes Research Manager, Pfizer, Inc., Brussels, Belgium

Please note: On the day of the scheduled webinar, the first 1000 registered participants will be accepted into the webinar. For those who are unable to attend, or would like to review the webinar at a later date, the full-length webinar recording will be made available at the ISPOR Educational Webinar Series webpage approximately 2 days after the scheduled Webinar.

Reservations are on a first-come, first-served basis.

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