Saturday, January 25, 2025

Is the Implementation of Gene Therapy HTA Recommendations Taking Place?

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A comprehensive analysis of the challenges affecting timely access to gene therapies has led to the development of six key recommendations, which were outlined in a prior report by the OHE. These recommendations emphasize changes in Health Technology Assessment (HTA) methodologies and evidence-generation practices necessary to fully realize the potential benefits of gene therapies.

In this latest report, country scorecards were created to assess the extent to which these recommendations have been implemented in eleven countries: Australia, Canada, Denmark, England, France, Germany, Italy, the Netherlands, Spain, Sweden, and Switzerland. The evaluation was based on targeted literature searches, insights from HTA experts, and input from local country affiliates at Pfizer. Three levels of achievement were considered: achieved, partly achieved, and not achieved.

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The country scorecards revealed variations in the degree to which recommendations were being implemented among countries and across different recommendations within countries. While most countries in the sample recognized the lifetime benefits of HTAs and achieved this recommendation, significant improvements were needed in operationalizing additional value elements.

Progress is being made in many countries to better capture the full value of gene therapies in HTAs, but there is still room for enhancement. Further advancements in this area will enhance patient access and unlock the transformative potential of gene therapies, benefiting both individuals and healthcare systems.


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