Paving the way: First PREFER PhD defence

2020-10-09

Eline van Overbeeke has investigated whether patient preference studies could help bridge the gap between new treatments and well-established decision-making structures. And bring gene therapies to market and patients sooner. She defends her thesis on 9 October 2020. 

Eline van Overbeeke
Eline van Overbeeke

Gene therapy is treatment by genes. They have the potential to create permanent effects in patients. With the rise of precision medicine and advanced therapy medicinal products, decision makers will have to deal with more uncertainty in long-term outcomes.

When Eline van Overbeeke started her PhD project, it was unknown how to design and conduct preference studies in the context of rare diseases, gene therapies and health technology assessment (HTA), and what method to use. In addition, the current system of evaluating medical products does not necessarily work for new and emerging medical products. And it does not necessarily cover all aspects of gene therapies that are relevant to patients.

Performing patient preference studies may help inform decision making by providing insight on whether patients accept the uncertainties associated with new treatments. To support the decision-making process, the patient preferences collected need to be fit for purpose, which means stakeholder and patient engagement is key.

 “PREFER has set the collaborative multi-stakeholder context for the PhD project. Thanks to the PREFER network I was able to conduct studies with key health agencies as the FDA, EMA and national HTA bodies,” says Eline van Overbeeke.

Eline van Overbeeke's dissertation coverPatient preference studies can help provide structured patient input into decision-making processes. Within this project, we have investigated how patient preferences can be elicited and used in HTA and payer decision making regarding gene therapies for rare diseases.

“Through PREFER I also had the opportunity to learn from methodological experts. The knowledge gained throughout the years contributed to my ability to conduct the PAVING study [on patient preferences regarding gene therapies in hemophilia] that became part of the PREFER portfolio, and this with the support Brett Hauber and a study-specific advisory board that included multiple PREFER members,” Eline van Overbeeke continues.

She advises that in the context of patient preference studies for HTA on gene therapies in rare diseases, education on gene therapies should be provided and preference heterogeneity should be explored. This would allow HTA representatives patient preferences as supportive evidence, something they have claimed to want in the interviews she conducted.

Eline van Overbeeke’s thesis also issues 5 key recommendations, listed below. The dissertation is currently embargoed, but anyone who is interested is invited to contact Eline van Overbeeke to request a copy. 

Recommendations

  • Ensure developers consider regulatory and payer needs in gene therapy development plans
  • Invest in rare monogenic disease preference research and orphan drug access
  • Increase methodological guidance for patient preference studies
  • Enhance stakeholder involvement in patient preference studies
  • Provide patient education on gene therapies beyond patient preference studies

Eline van Overbeeke’s PhD defense

Thesis title

PAVING the way: Patient preferences to Assess Value in Gene therapies

Date of defense

9 October 2020

Supervisors

  • Prof. Dr. Isabelle Huys (KU Leuven)
  • Prof. Dr. Steven Simoens (KU Leuven)
  • Prof. Dr. Michel Goldman (Université Libre de Bruxelles)

Chair

  • Prof. Dr. Pieter Annaert (KU Leuven)

Jury members

  • Prof. Dr. Minne Casteels (KU Leuven)
  • Prof. Dr. Martina Vandebroek (KU Leuven)
  • Prof. Dr. Sofie Bekaert (UGent)
  • Prof. Dr. Ardine de Wit (UMC Utrecht)