Key takeaways

  • Changes to HTA methodologies and evidence generation aimed at capturing the full value of gene therapies should lead to more timely access.
  • Several countries are achieving some of the changes recommended in a previous OHE report, demonstrating examples of best practice.
  • However, there is considerable variability in the extent to which recommendations are being achieved between countries and within countries.
  • None of the countries assessed achieved all recommendations, demonstrating significant potential for improvement in the HTA of gene therapies.

Several challenges are preventing timely access to gene therapies. Based on a review of the literature, supplemented by the insights and discussions of an international panel of HTA and health economics experts, six overarching recommendations were determined and published in a previous OHE report. These recommendations highlight the changes to HTA methodologies and evidence generation activities that should be prioritised to enable the potential benefits of gene therapies to be realised. 

In this report, we develop country scorecards exploring the extent to which these recommendations are being achieved in eleven countries. The countries considered were: Australia, Canada, Denmark, England, France, Germany, Italy, the Netherlands, Spain, Sweden and Switzerland.  

Targeted literature searches, including searches of grey literature and official HTA agency documentation, inputs from participants of the roundtable informing the recommendations and insights from local country affiliates from Pfizer, were used to determine the extent to which the recommendation was achieved in each country. Three levels of achievement were considered:  

Category  Description 
Recommendation achieved  The recommendation has been considered, and relevant guidelines/agreements are routinely implemented. However, this does not preclude further improvements. 
Recommendation partly achieved  Steps have been taken to begin implementing the recommendation or implementation has begun, but uptake could be improved considerably.  
Recommendation not achieved  No steps have been taken to begin implementation of the recommendation.  


By identifying strengths across countries, we also highlight areas of best practice for each recommendation. 

The country scorecards reveal variations in the extent to which the recommendations are being achieved between countries and across recommendations within countries. 

Most countries in our sample recognise the lifetime benefits in HTAs and, therefore, achieve this recommendation. In contrast, considerable improvement is needed in the recommendation for operationalising additional value elements.  

Although progress is being made towards allowing the full value of gene therapies to be captured in HTA in many countries, there is still room for improvement. Further progress will facilitate patient access and enable the potential transformational benefits to patients of gene therapies, as well as accurately realise the benefits for health systems and society.  

This report “Are Recommendations for HTA of Gene Therapies Being Achieved?” and the previous report “Health Technology Assessment of Gene Therapies: Are Our Methods Fit for Purpose?” were commissioned and funded by Pfizer.