Transferability of Real World Evidence to Support HTA Recommendations in Lower Income European Countries

Area of Science:

• Health economics and outcomes research
• Rare disease treatment
• Health technology assessment (HTA)

Background:

• Lower income European countries (LIECs) face financial constraints for high-cost rare disease treatments compared to higher income countries (HIECs).
• Myelodysplastic syndrome (MDS) serves as a case study for exploring evidence transferability.
• The HTx project investigated integrating target trial emulation (TTE) into routine Health Technology Assessment (HTA) work.

Purpose of the Study:

• To explore the transferability of real-world evidence (RWE) generated by TTE from HIECs to support treatment recommendations in LIECs for rare diseases.
• To identify challenges and propose solutions for transferring TTE-generated RWE to LIECs.
• To enhance HTA capacities in LIECs for rare disease treatment decisions.

Main Methods:

• Utilized target trial emulation (TTE) to generate real-world evidence (RWE) in higher income European countries (HIECs).
• Conducted workshops with HTA consortium partners and external experts to identify challenges in transferring TTE results to lower income European countries (LIECs).
• Focused on the myelodysplastic syndrome (MDS) case study.

Main Results:

• Lack of local real-world data, differing patient pathways, comparator technologies, and limited expertise/resources are key barriers for TTE in LIECs.
• Transferring RWE via TTE offers an improvement over standard HTA methods, especially with joint clinical assessments for orphan medicines.
• TTE enables LIECs to evaluate high-cost technologies for specific patient subgroups, but requires training in advanced methodologies.

Conclusions:

• This study is the first to examine RWE transfer using TTE for rare diseases in countries with limited HTA capacity.
• Transferring TTE-generated RWE is feasible and beneficial for LIECs, despite identified challenges.
• Addressing challenges requires training HTA professionals and potentially collaborative approaches like joint clinical assessments.