Articolo
Unlocking the potential of Nordic real-world data for PICO assessments in European HTA
With the complexities of PICOs playing a large part in the JCA process, the utility of using RWD, particularly from the rich registry data in the Nordics, to inform PICOs should be explored.
The increased focus on PICOs
As of 2025, the European Union has moved toward a centralized health technology assessment (HTA) framework through the Joint Clinical Assessment (JCA) process, which operates alongside the regulatory procedures, which aims to accelerate and standardize access to medicines and medical devices across member states. The first categories to pass through JCA are oncology products and advanced therapy medicinal products (ATMPs). To date, several submissions are currently being evaluated within the new process.
The intended outcome of JCA is to establish the relative effectiveness and safety of a new health technology as compared with new or existing technologies. This starts with a PICO (Population, Intervention, Comparator, Outcome) scoping process to inform which research questions must be addressed in the submission package and consequently determine the data that will be requested.
The intended outcome of JCA is to establish the relative effectiveness and safety of a new health technology as compared with new or existing technologies. This starts with a PICO (Population, Intervention, Comparator, Outcome) scoping process to inform which research questions must be addressed in the submission package and consequently determine the data that will be requested.
The final PICOs will be developed based on the member states’ input to ensure that the research questions reflect national policies. Notably, there is neither a dialogue between manufacturers and regulators nor any consideration of available evidence during the PICO scoping phase. This underscores the importance of proactive evidence generation to match final PICOs, where real-world data (RWD) can play an important role.
Nordic health registries to explore PICOs
The possibility of exploring PICOs using RWD could be a powerful tool. The collected data could support manufacturers in anticipating regulatory requirements and streamline the evidence package, which is fundamental for the success of a submission in the context of JCA.
The Nordic countries are renowned for their comprehensive, high-quality, and long-term health registries, often spanning decades. These registries have the potential to act as pivotal sources of RWD, supporting PICO assessments for both pharmaceuticals and medical devices. Importantly, the personal identification number used in the Nordics enables seamless integration of data across multiple sources and facilitates individual-level, multivariable analyses.
The Nordic countries are renowned for their comprehensive, high-quality, and long-term health registries, often spanning decades. These registries have the potential to act as pivotal sources of RWD, supporting PICO assessments for both pharmaceuticals and medical devices. Importantly, the personal identification number used in the Nordics enables seamless integration of data across multiple sources and facilitates individual-level, multivariable analyses.
How can RWD support each PICO dimension?
RWD has the potential to inform all PICO dimensions in various ways. In addition to detailed epidemiology, an understanding of clinical management (e.g. patient journey, drug utilization patterns, complications, and comorbidities) can provide insights to both relevant (P)opulation and (C)omparator. If accessing RWD in time to act on the result, the collected data can guide the need for and the design of future studies within the planned trial program or add-on studies regarding, for example, relevant endpoints (i.e. (O)utcome). For the (I)ntervention dimension, RWD can further be used for post-launch activities (e.g. real-world evidence [RWE] studies on the impact of implementing the therapy in clinical routine and follow-up studies on efficacy/safety requested by regulatory and/or reimbursement bodies). Importantly, timely planning for the use of RWD during the development process is key, particularly given the potential lag time for accessing data depending on the extent and scope.
How useful is Nordic RWD to support a general PICO assessment?
The Nordic health data registries are widely recognized for their unique opportunities to conduct registry-based research, but how applicable would an RWE study based on the Nordics be to predict PICOs for the JCA? Well, there are several aspects substantiating that a Nordic study could indeed be expedient to enhance evidence packages to align with central regulatory expectations:
- Comprehensive coverage: Disease-specific quality registers, in Sweden in particular, support a wide range of therapeutic areas — from oncology and orthopedics to psychiatry and endocrinology.
- Linkability: The use of personal identity numbers allows linkage across registries, enhancing data depth and reliability.
- Generalizability: Although rooted in the Nordic context, the data are sufficiently robust to support broader European population studies. The national registries and many common registries across the Nordic countries cover a very large total population (the total Nordic population amounts to nearly 30,000,000 people).
- Efficiency: With the setup of data sources and an RWD protocol, PICOs can be updated continually with minimal effort, ensuring continuous alignment with current evidence.
- The sheer volume and granularity of disease-specific quality registers (specifically in Sweden) provide detailed data that can significantly enhance HTA evaluations, especially for medical devices where usage patterns and outcomes vary widely.
A study to explore the availability and capacity of Nordic health registries
A review of available registries across the five Nordic countries to assess their applicability to inform each PICO dimension was conducted for two hypothetical products: an oncology drug (Cenpicomab) and a hip replacement device (Cenpicohip). A PICO prediction exercise based on the hypothetical Cenpicomab had been performed previously and presented at ISPOR in 2024, while Cenpicohip was defined for this study.
General findings
All assessed registries were categorized by therapeutic area (alternatively, all diagnoses/no diagnosis/other) (Figure 1).
Figure 1. Nordic health registries by therapeutic area
Figure 1. Nordic health registries by therapeutic area
Figure 2. Nordic health data registries by country
Figure 2. Nordic health data registries by country
The number of registries per each specialty assessed was aggregated by country. Denmark and Sweden had the highest representation across all therapeutic areas (Figure 2). Notably, Sweden had registries covering all assessed therapeutic areas. Subsequently, a multilayered RWD study could likely be supported by Swedish registries for most conditions.
Next, relevant registries were evaluated for their ability to support population characterization, intervention tracking, comparator identification, and outcome measurement for the two hypothetical therapies.
Sources for registry data to support a PICO assessment for both the fictional oncology and medical device products were identified in each of the Nordic countries. There were seven relevant registries for the oncology product (Cenpicomab), of which four were available in all the Nordic countries and one was a disease-specific quality registry available in three countries.
For the medical device (Cenpicohip), there were four relevant registries, of which one was available in all countries and two were quality registries specific to the therapeutic area available in three countries.
Next, relevant registries were evaluated for their ability to support population characterization, intervention tracking, comparator identification, and outcome measurement for the two hypothetical therapies.
Sources for registry data to support a PICO assessment for both the fictional oncology and medical device products were identified in each of the Nordic countries. There were seven relevant registries for the oncology product (Cenpicomab), of which four were available in all the Nordic countries and one was a disease-specific quality registry available in three countries.
For the medical device (Cenpicohip), there were four relevant registries, of which one was available in all countries and two were quality registries specific to the therapeutic area available in three countries.
Figure 2. Nordic health data registries by country
Usefulness for respective PICO dimensions
(P)opulation
To explore the target population, registries such as the National Patient Registry (NPR), Prescribed Drug Registry (PDR), and various clinical registries (CRs) were found. For Cenpicomab, additional sources like cancer registries (CaR), lung cancer CRs, and palliative care CRs could provide disease-specific insights. For Cenpicohip, hip arthroplasty CRs and medical implant registries were key sources.
(I)ntervention
Post-launch activities could be informed by NPR, CRs, and PDRs. For the oncology drug, lung cancer CRs were particularly relevant, while the high-value hip arthroplasty and implant registries were relevant for the hip replacement device.
(C)omparator
To identify appropriate comparators, the same registries applicable for a population provide drug utilization data and clinical management.
(O)utcome
An assessment of relevant outcomes would require registries to report relevant data. For Cenpicomab, outcome data could be retrieved from NPR, PDR, CaR, lung cancer CRs, cause of death registries, and pathology registries. For Cenpicohip, overlapping sources were relevant (NPR, PDR, and CRs), but for assessing specific outcomes, hip arthroplasty CRs are fit for purpose.
Final remarks
The Nordic countries continue to set the gold standard for RWD infrastructure. Their health registries offer unparalleled depth, coverage, and linkability, making them ideal for informing PICO assessments in HTA processes. As Europe moves toward greater harmonization through JCA, readiness for the evidence requirement could lead to a rapid and effective process. Ultimately, this contributes to improved patient access to innovative therapies and devices across Europe, which is indeed the very intention of the new process.
This article summarises Cencora’s understanding of the topic based on publicly available information at the time of writing (see listed sources) and the authors’ expertise in this area. Any recommendations provided in the article may not be applicable to all situations and do not constitute legal advice; readers should not rely on the article in making decisions related to the topics discussed.
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Sources
- Directorate-General for Health and Food Safety. Updated: List of ongoing joint clinical assessments. https://health.ec.europa.eu/latest-updates/updated-list-ongoing-joint-clinical-assessments-2025-09-02_en
- European Commission. Joint Clinical Assessments. https://health.ec.europa.eu/health-technology-assessment/implementation-regulation-health-technology-assessment/joint-clinical-assessments_en
- Joline Schmidt K, Konstanski M, Sauer A-K, et al. Understanding Joint Clinical Assessment scoping requirements in oncology: results of a rapid PICO prediction exercise via an in-house expert network. https://www.ispor.org/docs/default-source/euro2024/isporeurope24hta200posterfinal145403-pdf.pdf?sfvrsn=323db99a_0
- Laugesen K, Ludvigsson JF, Schmidt M, et al. Nordic health registry-based research: a review of health care systems and key registries. Clin Epidemiol. 2021;533-554.
- Member State Coordination Group on HTA (HTACG). 2024. Guidance on the scoping process.
- Nordforsk. Joint Nordic registers and biobanks: a goldmine for health and welfare research. https://www.nordforsk.org/2014/joint-nordic-registers-and-biobanks-goldmine-health-and-welfare-research
- The Swedish Research Council (n.d.). Guide till register- och hälsodata. https://dataguiden.se/
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