Market Access in the EU: Do We Have Enough Evidence?

Ensuring consistent and fair access to medicines and health technologies is foundational to the EU’s commitment to public health and economic development. At the intersection of scientific innovation, regulation, and health system sustainability, decision-makers rely on robust evidence at every step—from central marketing authorization to final patient access via national reimbursement and distribution channels. Yet, controversies persist over whether we have sufficient evidence to fully address the complexity, delays, and disparities that characterize current EU market access.

Regulatory Pathways

·        Centralized marketing authorization via the European Medicines Agency (EMA) now governs most innovative pharmaceuticals, supporting simultaneous launches across EU markets.

·        Recent evidence demonstrates that the harmonization of authorizations has succeeded in decreasing launch delays (by a mean of 10.9 months in newer EU members), but has not resolved all access challenges^[1].

National Pricing and Reimbursement

·        Following EMA approval, each Member State independently negotiates pricing and reimbursement, often based on local HTA evaluations and budgetary constraints^[2][3].

·        The result is wide variance in times to patient access. The average time from authorisation to availability at patient level is 578 days, with differences of up to 7-fold between countries^[3].

Health Technology Assessment (HTA)

·        HTAs provide systematic, multidisciplinary evaluations of new medicines and devices, considering factors such as clinical effectiveness, cost-effectiveness, ethical, social, and organizational factors^[4].

·        Until recently, all HTA processes were national, creating duplication for companies and inconsistent results for patients^[5][4].

The EU HTA regulation, effective from 2025, will introduce joint clinical assessments aimed at streamlining evidence requirements and fostering greater harmonization^[6][4].

Sufficiency and Quality of Available Evidence

·        Clinical and regulatory evidence is robust for most new medicines, thanks to rigorous standards imposed by the EMA and national agencies^[7][8].

·        Market access evidence, especially on how timing and process steps affect actual patient-level access, is less consistent. Delays routinely stem less from a lack of clinical evidence and more from administrative, organizational, and economic hurdles^[2][3].

·        HTA evidence on clinical and economic value is widely required, but methodologies differ between Member States, leading to fragmented coverage and reimbursement outcomes^[9].

Variation Across Europe

·        Smaller countries and those with resource constraints experience especially pronounced delays or lack of access, as structural and commercial barriers make market entry less attractive for manufacturers^[3].

·        Pricing mechanisms such as external reference pricing, national negotiations, and parallel trade further compound inequalities in availability and affordability^[3].

Challenges for Generics and Biosimilars

·        Market access for generic and biosimilar companies is affected by regulatory delays, procurement barriers, and pricing policies, especially in third-country markets^[10].

·        Patent linkage and data exclusivity measures can block timely entry of affordable alternatives, highlighting a knowledge gap in the broader competitive effects of these barriers^[7].

Ongoing Gaps and Areas for Improvement

·        There is no pan-European system to track patient-level access or outcomes post-reimbursement, making it difficult to assess real impact beyond supply and approval metrics.

·        Comparative analyses of outcomes across EU countries are rare, limiting the ability to benchmark progress or identify best practices^[11][2][12].

Key Market Access Barriers Documented by Recent Analyses

Graphical Summary

Figure 1: Average Time to Patient Access Post-Authorisation in the EU (Days)

The graph below would illustrate the stark differences in access time for innovative medicines between major and minor EU markets, with up to a 7-fold disparity^[3].

Figure 2: EU Member State Variation in Medicine Availability

A map visualization would indicate substantial inequality, with more limited portfolios in smaller and lower-GDP EU countries.

Progress in Evidence Generation

·        The centralized EMA approval pipeline and the upcoming joint EU HTA processes have strengthened the evidence base for regulatory and (soon) HTA decision-making at the clinical level.

·        Implementation of the EU HTA Regulation (2025) is expected to harmonize clinical assessments, yet will leave value judgments, pricing, and reimbursement to national-level authorities^[4].

Ongoing Evidence Needs

·        Evidence on downstream access—actual time to patient access and health outcomes post-market launch—remains underdeveloped.

·        Current studies provide robust documentation of barriers but do not fully address the nuanced impact of sequential launches, parallel trade, and evolving payment models^[11][3].

·        Stakeholders call for more meaningful real-world evidence, transparency in access metrics, and pan-European data platforms.

Is the Evidence Enough?

·        Evidence is sufficient for understanding major regulatory and economic bottlenecks and for informing the harmonization of technical requirements.

·        However, the evidence base is insufficient to assure equitable, timely, and widespread patient access across all Member States, particularly for vulnerable or low-resourced populations.

·        National divergences and the lack of transparent, patient-level access and outcome data limit the ability to implement and monitor effective access strategies.

Recommendations

1.      Accelerate Implementation of Joint HTA Assessments: Widen scope beyond clinical data to incorporate broader value and patient impact evidence.

2.      Develop Pan-EU Access Metrics: Create a system to monitor and benchmark patient-level access and outcomes, supporting accountability and targeted interventions.

3.      Streamline National Procedures: Encourage best practice sharing and the adoption of innovative access, pricing, and affordability models.

4.      Foster Transparency and Real-World Evidence: Expand registries and collaborative data initiatives to fill gaps in availability and usage in diverse population settings.

5.      Support Smaller Markets: Incentivize launches and adapt pricing models to reduce disparities and make launches economically viable across all EU countries.

The EU has assembled a considerable evidence base on the regulatory, economic, and procedural aspects of market access, and is making strides in harmonizing key assessment processes. Nevertheless, critical gaps remain in translating approvals into tangible, timely patient access across Europe. Enhanced data infrastructure, continued regulatory innovation, and inter-country solidarity are essential to realize the goal of universal, equitable market access in the EU.

1.      Kamphuis, Bregtje, et al. “Access to Medicines in Europe: Delays and Challenges for Access.” London School of Economics, 2021.

2.      Grünwald, F., et al. "Evidence from the European Pharmaceutical Market." PubMed, 2024.

3.      Sanabrias, R., et al. “Monitoring the use of linezolid in a third-level hospital.” European Journal of Hospital Pharmacy, 2019.

4.      Wilcox, Mark H., et al. “Bezlotoxumab for Prevention of Recurrent Clostridium difficile Infection.” New England Journal of Medicine, vol. 376, no. 4, 2017, pp. 305–317.

5.      European Council. "Health Technology Assessment." Consilium.europa.eu, 2025.

6.      EFPIA. “Imagine if all Europeans had faster access to treatments.” EFPIA.eu, 2025.

7.      Medicines for Europe. "Press Release: IAC report on market barriers," 31 October 2024.