ISPOR white paper recommends coordinated evidence generation across European healthcare decision-making

ISPOR’s latest white paper calls for closer coordination between regulators, HTA bodies and payers to reduce evidence fragmentation, address uncertainty and support more consistent patient access decisions across Europe.

The Baseline

• ISPOR has published a white paper calling for closer alignment between regulators, health technology assessment (HTA) bodies, and payers to improve healthcare decision-making across Europe.
• The report recommends coordinated life-cycle evidence planning, broader early scientific consultations, and greater use of real-world evidence (RWE) to reduce uncertainty and support more consistent patient access decisions.
• It also proposes stronger governance, common methodological standards, and enhanced collaboration to improve evidence generation and decision-making across the EU and European Economic Area.

ISPOR—The Professional Society for Health Economics and Outcomes Research—has published a new white paper examining how healthcare decision making could be better aligned across the EU and European Economic Area (EEA). Based on discussions from the ISPOR Strategic Dialogues: European Union Regulator-HTA-Payer Forum held in October 2025, the report, Achieving Systemic Coherence for Healthcare Decision Making in Europe, outlines recommendations intended to improve evidence generation and strengthen collaboration throughout the life cycle of health technologies.

The paper argues that patient access across Europe continues to be hindered by fragmentation between regulatory authorities, HTA bodies, and national payers. It states that patient access pathways are "impeded by structural fragmentation" and that the absence of "a shared evidence roadmap" contributes to "misaligned evidence, persistent uncertainty, and systemic delays."

According to the report, these challenges stem partly from the distinct but interconnected roles performed by regulators, HTA bodies, and payers. Regulators assess the quality, safety, and efficacy of medicines and other health technologies, HTA bodies evaluate their comparative clinical and economic value, and payers determine pricing, reimbursement, and access within national healthcare systems. Although these processes are closely linked, they largely operate sequentially and often require different types of evidence, resulting in repeated evidence requests and contributing to ongoing uncertainty.

To improve alignment, the white paper recommends expanding early scientific consultations beyond regulators and HTA bodies to include payers and patients. The authors argue that broader engagement before market entry could help establish shared life-cycle evidence plans, clarify pre- and post-launch evidence requirements, and identify strategies for addressing uncertainty from the outset.

The report also recommends adapting assessment approaches to better reflect the level of innovation and uncertainty associated with different technologies. Among the recommendations are distinguishing transformational from incremental innovation, introducing living HTA for products supported by evolving evidence, and making greater use of adaptive pathways and regulatory sandboxes to support evidence generation after initial authorization.

A further priority is strengthening the use of RWE. The paper recommends that HTA bodies and payers play a greater role in shaping the European Health Data Space (EHDS) by defining the data elements, comparators, endpoints, and economic variables needed for healthcare decision-making. It also advocates common European methodological standards for RWE and greater use of cross-border data networks to address evidence gaps, particularly for rare diseases and other small patient populations.

The report also argues that achieving better alignment will require stronger governance and communication across the healthcare decision-making landscape. It recommends establishing formal mechanisms for collaboration between regulators, HTA bodies, and payers while preserving institutional independence, alongside sustainable support for patient participation and greater transparency around uncertainty, value judgements, and budgetary considerations underpinning access decisions.

The paper identifies several priority actions for the next 12–36 months. These include broadening Joint Scientific Consultations to incorporate payer and patient perspectives, increasing HTA and payer involvement in EHDS governance, developing EU-wide life-cycle evidence frameworks, harmonizing RWE methodologies, and institutionalizing periodic HTA re-evaluation linked to post-launch evidence generation.

Commenting on the publication, ISPOR Chief Executive Officer Rob Abbott said:

“ISPOR's unique strength lies in our ability to bring together regulators, HTA bodies, payers, patients, industry, and researchers around a shared evidence agenda. As a neutral convener at the critical interface between scientific evidence and health policy, ISPOR is committed to driving the structured, multistakeholder dialogue Europe needs to move from fragmented evidence generation to a truly integrated, patient-centered system.”