Case Study

Multi‐functional support for EU access strategy and NICE submission for a novel haematological therapy


Our client required support to develop an access strategy to meet EU payer requirements, critical appraisal, evidence generation, value proposition development and HTA submission, for their new product.

Product X was a novel disease modifying therapy for a severe haematological condition with high unmet need, historical underfunding and lack of innovation.

A number of challenges influenced an evolving strategy; identification of an appropriate target population, use of surrogate markers, combined RWE and RCT evidence base to support the value proposition, limited data on disease population, few HTA precedents.


A multi‐functional HEOR team was established to interact with the client across several jurisdictions.

The objective was to contribute and shape strategy, provide guidance and support HTA assessment.

Multiple interconnected workstreams were conducted in four main areas:

  • Access strategy: EU strategy workshop, pricing analysis
  • Evidence review and generation: Critical appraisal, SLRs
  • Value proposition: Value message development and testing, GVD
  • NICE/SMC submission and advisory board


Outputs from each of the workstreams contributed to the evolving strategy for product X and provision of data to support payer assessment.

An optimal strategy for meeting the needs of EU payers was developed along with key information to support HTA assessment.

HEOR managed the NICE submission; decision is expected Q4 2022.