20 January 2021


First webinar on appraisal for rare disease treatments

WP10 is presenting a series of four webinars relating to appraisal of rare disease treatments. 


In this first webinar, Elena Nicod and Karen Facey presented international research with HTA bodies that has led to Recommendations to Enhance Appraisal Processes for Rare Disease Treatments.   


Cliff Goodman from the Lewin Group led the discussion about feasibility of proposals with HTA experts.  

The discussion and audience interactions will inform the research team's final development of the recommendations. Panellists included:  

  • Antonio Addis, Regional Health Service – Lazio Region (Italy)   

  • Einar Andreassen, NOMA (Norway)   

  • Peter Jackson, Chair HST NICE (England)   

  • Vinciane Pirard, Orchard Therapeutics (Industry)  


The slides presented are available as a PDF and recording of the webinar can be accessed HERE

15–16 May 2020


10th European Conference on Rare Diseases & Orphan Products (ECRD)

The 10th ECRD will take place in Stockholmsmässan, Stockholm, Sweden. For more information on the conference, please visit the ECRD website (here).

  • Poster abstracts – submissions now open (until 10 February)

  • Patient Advocate Fellowship Programme – applications now open (until 2 December)

  • New Linkedin ECRD page – please follow and share with your network.

This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 779312