Objectives: Making innovative treatments available across the European Union is a complex process due to differing evidence requirements, assessment methods and value judgements by HTA agencies. HARMONY, the hematological malignancy (HM) specific big data for better outcomes Innovative Medicines Initiative (IMI) project has over 50 partner organisations with varying expertise in evidence development strategies to support new treatments and indications. We aimed to produce a guide for the evidence required for market access and coverage/reimbursement ("access evidence") focussing on challenges facing innovative HM drugs and the jurisdictional differences across Europe.

Results: We developed a multilevel framework based on methodology guidelines, interviews and previous submission reports and documentation from key HTA agencies (n=13), regulators (n=6) and payers (n=2). Level 1 incorporates a signposting tool to raise awareness of the regulatory/HTA and payer landscape and terminology, highlighting HM specific outcome requirements where available. Level 2 is a series of easy access dashboards incorporating domains and topics from the HTA Core Model® including safety, clinical effectiveness, cost and economic evaluation and ethical and organisational aspects. Populated from data extracted from 77 previous reimbursement decisions on 12 HM products across 9 HTAs, the framework highlights key differences/requirements and evidence use by both HTA and disease indication, and how these may impact on recommendations for reimbursement.

Practical implications: The freely available access evidence framework is anticipated to have several benefits for HARMONY and HM researchers. Developed as a unique resource for this clinical area it provides a concise point of reference with key learnings and requirements for HM evidence generation across the EU. It allows a direct comparison across differing agencies on the acceptability and use of evidence for key markets and could potentially increase the efficiency in generating and preparing relevant access evidence but also of predictability of HTA outcomes.