欧盟联合临床评估（JCA）：申办方实施指南

Contents Introduction3Areas of challenge for sponsors4How sponsors can prepare for the JCA implementation6Case study: JCA preparation in action10Optimize your JCA readiness11Authors12 Introduction The Joint Clinical Assessment (JCA) framework (Regulation (EU) 2021/2282) was implementedon January 12, 2025 for all new medicinal products for oncology, and advanced therapy medicinalproducts (ATMPs). From 2028, this process will apply to orphan medicinal products, and from 2030onwards, for all other products not previously included. Biosimilars, generics, and all products withexisting marketing authorization by European Medicines Agency (EMA) in the European Union (EU) areexcluded, even if applications are submitted for new indications. Who are the 27 EU member states? The countries participating in the EU JCA: Austria, Belgium, Bulgaria, Croatia,Cyprus, Czechia, Denmark, Estonia, Finland, France, Germany, Greece, Hungary,Ireland, Italy, Latvia, Lithuania, Luxembourg, Malta, Netherlands, Poland, Portugal,Romania, Slovakia, Slovenia, Spain and Sweden The goal of the JCA framework is to improve the process for evaluating the clinical evidence of newmedicines across the 27 EU member states, addressing concerns regarding equity, timeliness andaccess. The output of the JCA process is a report that provides a single, harmonized evaluation of the clinicalevidence for new treatments for all EU countries. It is a factual description of the relative effectsobserved for analyzed health outcomes, including numerical results, confidence intervals, and ananalysis of scientific uncertainty and evidence quality. The report is expected to deliberately avoid valuejudgments, rankings, conclusions on overall benefit or clinical added value, and recommendations on themedicinal product’s use or positioning within therapeutic strategies. While not EU members, Norway, Iceland and Liechtenstein (as part of the EuropeanEconomic Area) also participate in the EU Health Technology Assessment (HTA)cooperation. Member states retain responsibility for determining the clinical added value of medicinal productswithin their specific healthcare contexts, as the relevance of analyses in the JCA report may vary bycountry. This approach allows for flexibility in considering local factors while utilizing the JCA reportas a foundation. The JCA report is intended to lay the groundwork for pricing and reimbursementnegotiations with HTA bodies at the member state level, facilitating timely decisions within each market. JCA is expected to increase collaboration and efficiency by avoiding duplication of efforts. Thisapproach could facilitate more equitable and timely access to new, innovative treatments acrossmember states, particularly benefiting smaller markets that historically face delays in product launchesor have limited resources for conducting their own HTA evaluations. As of July 2025, the JCA process is underway for six oncology products, including ATMPs, withassessments expected to be published in 2026. For 2025, it is estimated that 17 oncology productsand eight ATMPs will undergo JCA evaluation. Areas of challenge for sponsors Based on our experience, we recognize six main areas of challenge for sponsors (known as healthtechnology developers or HTDs in JCA nomenclature): 1.Identifying and defining the PICO (Population, Intervention, Comparator, Outcome) relevant toeach member state The standard of care may vary across member states, leading to variations in the PICO elementsrequested by different EU member states and HTA bodies. The diversity in healthcare practicesand priorities often results in a high number of requested PICOs in the final scope, even after JCAconsolidation. Additionally, the final scope will not specify which member states requested particularPICOs. Consequently, sponsors will need to proactively anticipate HTA requirements to effectivelyprioritize and facilitate the generation of relevant evidence. What is PICO? The PICO framework provides a standard format for specifying research questions: P (population)– including full patient population and/or relevant subpopulationsI (intervention)C (comparator[s])– including approved or off-label comparatorsO (outcomes) If a comparison against multiple comparators is required, each will have aseparate PICO. 2. Preparing evidence simultaneously for all member states 5. Addressing resource and organizational challenges Smaller sponsors may be particularly impacted by the growing resource demands of theJCA process, that runs alongside the regulatory process and includes providing additionaldata requested, meeting submission timelines and preparing evidence/strategy in advance –particularly without a European or local Market Access and Health Economics and OutcomesResearch (HEOR) function. As JCA includes evidence requirements for all 27 European member states, evidence will need tobe generated at the same time (e.g., indirect treatment comparisons), where previousl