Key takeaways
- In recent years, the European Medicines Agency (EMA) has introduced a number of accelerated regulatory pathways to support timely access to promising therapies in areas of high unmet medical need. On the other hand, Health Technology Assessment (HTA) and pricing and reimbursement (P&R) processes across Europe have not consistently evolved in parallel, resulting in uneven access to these treatments between countries.
- OHE developed the Accelerated Patient Access to Cancer Care in Europe (APACE) framework through a structured, multidisciplinary stakeholder engagement process. APACE focuses on oncology treatments for conditions with significant unmet need that lack mature evidence for conventional HTA and P&R pathways, and outlines key principles and processes that can be applied at a national and pan-European level.
- The framework covered four key phases: eligibility criteria, initial assessment, reassessment, and exit criteria. Within these phases, key components included clearly defined eligibility criteria, a comprehensive data collection agreement, pricing that reflects expected value throughout the process, and enforceable exit mechanisms.
- While the framework is designed to be implemented initially at the national level, it is intended to evolve alongside EU efforts to harmonise HTA processes, ultimately supporting faster, evidence-based access to promising treatments across Europe.
- Additional policy dialogue is required to address outstanding challenges. OHE developed a set of recommendations to progress the implementation of APACE including developing consistent methods for assessing uncertainty, defining relevant and feasible outcomes, ensuring use of appropriate P&R models, and defining how APACE will align with new EU-wide HTA regulations and processes.
In recent years, the European Medicines Agency (EMA) has introduced several accelerated pathways (APs) to enable earlier access to promising therapies where there is high unmet medical need, particularly in oncology. However, while regulatory acceleration has been achieved, value assessment processes as part of Health Technology Assessment (HTA) and pricing & reimbursement (P&R) across EU member states have not changed consistently in response. National HTA and P&R processes have differing evidentiary requirements and timelines compared to regulatory approval, leading to fragmented access pathways in individual member states. As a result, patients in Europe often do not receive fast access to these treatments, and significant inconsistencies persist across countries. HTA agencies typically request more robust data than what is available at the point of accelerated regulatory approval, and flexible reimbursement and HTA models, such as risk-sharing agreements and reassessment, are underutilised. The upcoming Joint Clinical Assessment (JCA) process may support alignment, but its uptake and impact remain uncertain.
To address these challenges, the Office of Health Economics (OHE) has developed an Accelerated Patient Access to Cancer Care in Europe (APACE) framework for promising oncology treatments targeting conditions with high unmet need, that are perceived to lack mature enough evidence for traditional HTA and P&R pathways. The framework lays out the key principles and processes for the HTA and P&R components of an accelerated pathway. This framework should initially be implemented at the national level, with countries agreeing and consistently adhering to the key principles. As the EU moves toward greater harmonisation of HTA processes, the APACE framework should evolve in parallel, supporting access and evidence generation at a multi-national level. While pricing and reimbursement (P&R) decisions will remain under the authority of national payers, the intrinsic link between value assessment and P&R highlights the importance of developing core principles that can be adapted at the national level.
The APACE framework was developed through a four-phase structured engagement process conducted between November 2024 and February 2025, involving European stakeholders—including regulators, HTA bodies, payers, patient representatives, economists, and industry representatives—from six countries.
The APACE framework outlines a structured pathway for managing the uncertainty of promising treatments for oncology. Key components include:
- Eligibility Phase:
- Treatments must address a condition with a significant and urgent unmet need and show promise of demonstrating effectiveness for HTA appraisal
- Initial Assessment:
- HTA bodies assess expected value and scientific uncertainty. Treatments qualify for managed access if uncertainties are resolvable to an acceptable level and if expected value justifies provisional reimbursement.
- A Data Collection Agreement (DCA) defines the studies needed to resolve specific uncertainties to an agreed upon level, with reassessment timelines.
- Reassessment:
- Takes place at the agreed-upon reassessment point. If available, new evidence informs updated expected value and assessment of whether uncertainties have been resolved to an acceptable level.
- Depending on evidence, or lack thereof, treatments may enter traditional reimbursement pathways, with either the same or an adjusted price, or are delisted.
- Resolution mechanisms for remaining uncertainties are set out.
- Exit Phase:
- Outlines conditions for entering the traditional reimbursement pathway or removal from reimbursement status, including obligations to patients and penalties for non-compliance.
There was broad support for the APACE framework among stakeholders. However, certain issues remained unresolved, including how to quantify eligibility criteria, define relevant outcomes and finalise a suitable P&R model under uncertainty. The resulting recommendations for further work and actions to progress the implementation of the framework are:
- Generate and agree clear eligibility criteria – Develop measurable and balanced criteria for eligibility based on unmet needs and urgency of condition, supported by stakeholder consensus and EU-level validation.
- Align regulatory and HTA requirements – Strengthen collaboration between regulators and HTA bodies to streamline eligibility and evidential standards and improve access timelines.
- Define relevant and feasible outcomes – Create EU-endorsed criteria for relevant outcomes and involve patients early to capture relevant, meaningful endpoints to be used in HTA assessments.
- Agree on principles and methods for replicable assessments of uncertainty – Adopt consistent methods to measure and manage evidential uncertainty in early and ongoing assessments.
- Strengthen national and pan-European real-world evidence infrastructure – Enhance national data collection systems, cross-country data integration and quality to support broad, effective real-world evidence use in evaluation.
- Progressing implementation of alternative P&R models – Encourage outcome-based agreements aligned with treatment value and evidence generation incentives.
- Creating legal enforcement mechanisms for exit process – Introduce enforceable legal frameworks to ensure timely delisting of treatments that no longer meet APACE criteria.
- Define funding arrangements – Establish dedicated short-term funds at the national level and long-term plans to sustainably finance accelerated access to ATs.
- Determine how APACE will integrate into EU HTA regulation (EU HTAR) processes – Improve EU-wide alignment in terms of timelines of assessments, evidence requirements and payment models used in accelerated pathways, to ensure APACE can fit well into JCA and other EU HTAR-related processes.
The APACE framework provides a structured, stakeholder-endorsed approach to accelerating access to oncology medicines targeting unmet needs across Europe. It seeks to bridge the gaps between regulatory approval and reimbursement by enabling conditional access paired with evidence development. To realise its potential, further policy work is needed to resolve outstanding issues and ensure consistent implementation across countries.