Implementing Coverage With Evidence Development for Medicines With Uncertain Clinical Benefit: Insights From the Netherlands
Author(s)
Jan-Willem Versteeg, MSc, PharmD1, Noraly Stam, MSc, PharmD2, Aukje Mantel-Teeuwisse, PhD1, Lonneke Timmers, PhD2, Wim Goettsch, PhD2, Christine Leopold, PhD1.
1Utrecht University, Utrecht, Netherlands, 2Zorginstituut Nederland, Diemen, Netherlands.
1Utrecht University, Utrecht, Netherlands, 2Zorginstituut Nederland, Diemen, Netherlands.
OBJECTIVES: Promising new orphan medicinal products (OMPs), conditionals (CMA), and exceptionals often face reimbursement delays and consequential delays in patient access due to limited evidence on their clinical effectiveness and high costs per patient. In 2019, the Dutch National Healthcare Institute therefore introduced a coverage with evidence development (CED) program for these medicines. This study aims to provide a comprehensive and transparent overview of the policy and its procedures, the medicines included in the program, and an analysis of the factors that have facilitated or hindered its implementation.
METHODS: This policy review included all medicines that have been part of the Dutch CED program since its start in 2019 up to May 1st, 2025. For each product, relevant information was extracted across key categories such as medicine type, evidence requirements, and program duration. Data was extracted from a range of documents, including process descriptions, progress reports, evaluation reports, health technology assessment reports, and European Public Assessment Reports. Expert consultations were used to validate findings.
RESULTS: Two flowcharts were developed: one outlining the CED program from a health systems perspective, and one detailing the procedural steps and stakeholder roles. Case studies of included medicines showed that the program was used for different types of medicines, indications, and evidence gaps. Despite initial plans to include 2 medicines per year, only a limited number have been included as of yet.
CONCLUSIONS: The CED program is gathering clinical effectiveness data while providing patient access to OMPs and CMA products. Important facilitating factors for the program were identified, including the involvement of all stakeholders, the only-in-research approach of data gathering, and the case-by-case evidence generation requirements and duration. Further evaluation is needed as the program does not yet include the expected number of medicines. No conclusion can be drawn yet on the quality of the collected data.
METHODS: This policy review included all medicines that have been part of the Dutch CED program since its start in 2019 up to May 1st, 2025. For each product, relevant information was extracted across key categories such as medicine type, evidence requirements, and program duration. Data was extracted from a range of documents, including process descriptions, progress reports, evaluation reports, health technology assessment reports, and European Public Assessment Reports. Expert consultations were used to validate findings.
RESULTS: Two flowcharts were developed: one outlining the CED program from a health systems perspective, and one detailing the procedural steps and stakeholder roles. Case studies of included medicines showed that the program was used for different types of medicines, indications, and evidence gaps. Despite initial plans to include 2 medicines per year, only a limited number have been included as of yet.
CONCLUSIONS: The CED program is gathering clinical effectiveness data while providing patient access to OMPs and CMA products. Important facilitating factors for the program were identified, including the involvement of all stakeholders, the only-in-research approach of data gathering, and the case-by-case evidence generation requirements and duration. Further evaluation is needed as the program does not yet include the expected number of medicines. No conclusion can be drawn yet on the quality of the collected data.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HPR119
Topic
Clinical Outcomes, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Coverage with Evidence Development & Adaptive Pathways, Reimbursement & Access Policy
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology, Rare & Orphan Diseases