Ahead of the much-discussed implementation of an EU-wide legislative framework on joint clinical assessments in 2025, Merck’s Nikola Vesic outlines the scope and aims of the reform and highlights some of the challenges in bridging the gap between theoretical vision and practical implementation.*
In 2022, the European Union (EU) passed new legislation on Joint Clinical Assessments (JCAs), and it is expected that the JCA process will be fully implemented by 2025, starting with oncology products and advanced therapy medicinal products (ATMPs).
The aim of the EU JCA is to harmonize the clinical assessment of medicines across the EU and to provide a single point of entry for companies to submit clinical trial data. The JCA process is intended to improve efficiency, reduce duplication of efforts, and provide faster and more consistent access to innovative medicines for patients across the EU.
The JCA process involves a collaboration between the European Medicines Agency (EMA) and national health technology assessment (HTA) agencies in EU Member States. The aim is to assess the clinical benefit and cost-effectiveness of medicines and to provide recommendations on pricing and reimbursement decisions at the national level.
Overall, the JCA aims to provide a more streamlined and efficient process for clinical assessments, while ensuring that patients across the EU have access to innovative medicines in a timely and consistent manner, but there are concerns that the practical implementation may not fully live up to the theoretical vision.
In theory, the JCA has the potential to significantly improve the drug assessment process in the EU. By coordinating assessments across different regions and countries, the JCA could reduce regulatory barriers and streamline the assessment process, potentially speeding up drug approvals and improving access to new therapies for patients.
However, the success of the JCA in practice will depend on several factors. Firstly, the JCA will need to be implemented effectively across different regions and countries. This will require significant coordination and cooperation between different stakeholders, including regulatory agencies, healthcare providers, and patient groups.
Secondly, the JCA will need to be sufficiently resourced to effectively carry out its mandate. This may require additional funding and resources from different regions and countries, which may be challenging to secure in the context of competing healthcare priorities and budget constraints.
Thirdly, the JCA will need to be flexible enough to accommodate the diverse needs and preferences of different regions and countries. This may require the development of customized assessment processes and protocols to reflect local healthcare priorities and needs.
Overall, while the JCA has the potential to significantly improve the drug assessment process in the EU, its success in practice will depend on effective implementation, sufficient resourcing, and flexibility to accommodate diverse local needs and preferences. Manufacturers will need to closely monitor the progress of the JCA and work closely with local stakeholders to ensure that drugs are accessible and affordable to patients in different regions and countries.
From the perspective of pharmaceutical manufacturers, the new legislation on Joint Clinical Assessments (JCAs) in the European Union (EU) is likely to have potential challenges:
PICO is a framework used in evidence-based medicine and clinical research to identify the key elements of a clinical question. It stands for Population, Intervention, Comparison, and Outcome. From the perspective of the pharmaceutical industry, there are several potential limitations associated with the PICO framework:
- Limited focus on clinical outcomes: While the PICO framework focuses on outcomes, it may not capture the full range of outcomes that are important to patients and healthcare providers. This could lead to a narrow focus on clinical outcomes, rather than broader outcomes such as quality of life or patient satisfaction.
- Limited focus on real-world settings: Clinical trials that use the PICO framework often focus on a narrow population and a specific intervention in a controlled setting. This may not fully capture the real-world experience of patients and healthcare providers, leading to limitations in generalizability and applicability of study findings.
- Limited ability to capture safety data: The PICO framework is primarily focused on efficacy outcomes, which may not fully capture safety concerns related to the intervention being studied. This could lead to an incomplete understanding of the risks associated with a particular drug or treatment.
- Potential for bias in comparison groups: The comparison group used in PICO-based studies may be subject to bias, particularly if it is not a randomized control group. This could lead to challenges in interpreting study findings and making evidence-based decisions.
Overall, while the PICO framework is a valuable tool for clinical research, it is not without limitations and potential cons from the perspective of the pharmaceutical industry. Manufacturers may need to consider these limitations when designing clinical trials and interpreting study findings.
The short timeline for JCA submission may present some challenges and implications, including:
- Increased pressure and resource requirements: The short timeline for JCA submission may increase the pressure and resource requirements for manufacturers, as they will need to ensure that all required data and documentation is available in a timely manner.
- Limited opportunity for feedback and revision: The short timeline for JCA submission may limit the opportunity for manufacturers to receive feedback and revise their submission, which could impact the quality of the assessment.
- Potential delays or rejection: If manufacturers are unable to meet the short timeline for JCA submission, this could result in delays in the assessment process or rejection of the submission.
- Need for careful planning and prioritization: To ensure that they can meet the short timeline for JCA submission, manufacturers will need to carefully plan and prioritize their resources and activities, which could impact other aspects of their operations.
Overall, the short timeline for JCA submission may present some challenges and implications for manufacturers, including increased pressure and resource requirements, limited opportunity for feedback and revision, and the potential for delays or rejection. Manufacturers will need to carefully plan and prioritize their activities to ensure that they are able to meet the timeline and successfully navigate the JCA process.
Lack of stakeholder involvement
- Limited understanding of patient needs: A lack of stakeholder involvement can lead to a limited understanding of patient needs and preferences, which can impact the development of drugs that effectively address patient needs.
- Regulatory challenges: Regulatory agencies may require stakeholder input as part of the drug development and approval process. A lack of stakeholder involvement can result in regulatory delays or rejections of drug applications.
- Limited access to funding: Healthcare payers may be less likely to provide reimbursement for drugs that were developed without stakeholder input. This can impact the commercial success of a drug and limit its market potential.
- Reduced patient engagement: A lack of stakeholder involvement can result in reduced patient engagement and participation in clinical trials, which can impact the quality and relevance of the data collected.
- Limited scientific society involvement: The absence of some scientific societies from the stakeholder network list in the EU JCA (e.g., ESMO) can be viewed as a potential concern due to limited access to expert guidance, no influence on assessment outcomes, variation in recommendations and guidelines, limited collaboration opportunities and representation of diverse perspectives.
- Negative perception of industry: A lack of stakeholder involvement can contribute to a negative perception of the pharmaceutical industry as being focused solely on profits rather than patient needs and outcomes.
Overall, a lack of stakeholder involvement can have significant limitations from the perspective of the pharmaceutical industry, impacting patient needs, regulatory approval, funding, patient engagement, scientific societies, and industry perception. Manufacturers need to actively engage with stakeholders throughout the drug development and approval process to optimize the potential benefits and minimize the potential drawbacks.
Local capabilities and resources
- Increased administrative burden: Manufacturers may be required to provide additional information and documentation to support the JCA process, which could increase the administrative burden and resource requirements.
- Need for local expertise: The JCA process may require manufacturers to have a better understanding of the local healthcare systems and regulatory requirements in each EU member state. This could require additional resources and expertise to navigate the different systems and requirements.
- Variability in local resources: There may be variability in the availability and quality of local resources, such as clinical trial sites and expert reviewers, across different EU member states. This could create challenges in ensuring that the JCA process is implemented consistently and efficiently across all member states and across all assessments
- Potential delays in access: If the JCA process results in delays or additional requirements for marketing authorization, this could impact the timely access to medicines for patients.
- PICOs formulation: Different member states (MS) might face the gaps in formulating PICOs due to limited expertise and resources, variation in healthcare systems, data availability and quality, harmonization challenges, including time and coordination constraints
Overall, while the JCA process is intended to improve the efficiency and consistency of clinical assessments, there may be some concerns regarding the local capabilities and resources required to implement the process effectively. Manufacturers may need to invest additional resources and expertise to navigate the local healthcare systems and requirements in each EU member state, and there may be challenges in ensuring consistent implementation across all member states.
Early Access Programs (EAPs)
Early Access Programs (EAPs) provide a pathway for patients with serious or life-threatening diseases to access investigational drugs before they are approved for commercial use. EAPs are typically used as a last resort when there are no alternative treatment options available, and patients are unable to participate in clinical trials.
From a pharmaceutical perspective, the introduction of the EU Joint Clinical Assessment (JCA) is unlikely to have a significant impact on the availability of EAPs. EAPs are typically managed at a local level by individual regulatory agencies and healthcare providers and are not directly impacted by the JCA.
However, the JCA may indirectly impact EAPs by potentially speeding up the approval process for new drugs, reducing the need for EAPs in some cases. If new drugs can be approved and made available to patients more quickly through the JCA, the need for EAPs may be reduced.
Additionally, the JCA may impact the availability and design of clinical trials, which can impact the availability of EAPs. If clinical trials are designed to meet the requirements of the JCA, they may be more streamlined and efficient, potentially reducing the need for EAPs.
Overall, while the JCA is unlikely to directly impact EAPs, it may indirectly impact their availability and design by potentially reducing the need for them in some cases. Manufacturers will need to closely monitor the impact of the JCA on drug development and commercialization, and work closely with local stakeholders to ensure that drugs are accessible and affordable to patients, including those who may need access to EAPs.
The question we should ask ourselves is: What really delays access – HTA or health system budget?
While the JCA process aims to address some of HTA challenges through standardized assessments, it is essential to acknowledge that achieving pan-EU access within a short timeframe requires addressing challenges and issues beyond HTA, such as healthcare system budget scarcity, regulatory variations, stakeholder engagement, and political considerations. The relative importance of these factors can vary depending on the specific healthcare system and the type of drug in question. Manufacturers must navigate these factors to ensure that drugs are accessible and affordable to patients, working closely with local stakeholders to understand the specific needs and constraints of different healthcare systems.
In many cases, the HTA process can be a significant contributor to delays in patient access to new drugs. HTA processes can be time-consuming and require significant resources to complete, which can result in delays in drug approval and reimbursement decisions.
On the other hand, healthcare system budgets can also be a significant constraint on patient access to new drugs. Country healthcare budgets will continue to be an important factor in determining the availability and affordability of drugs, particularly in the context of price negotiations and reimbursement decisions. The budget constraints may limit the ability of these countries to reimburse and provide timely access to innovative therapies, even if the assessments are streamlined through the JCA process. Addressing budget scarcity requires broader discussions and potential policy changes at the national level to allocate sufficient resources to healthcare and pharmaceutical spending. Limited healthcare budgets may result in difficult choices for healthcare providers, who must prioritize spending on different treatments and interventions. This can result in delays in drug approval and reimbursement decisions, as well as limitations on patient access to certain treatments.
While the JCA may help to improve access to new drugs by reducing regulatory delays and speeding up the assessment process, it is unlikely to eliminate the challenges associated with local healthcare budgets. However, the JCA may help to facilitate more coordinated and consistent approaches to drug assessments across different regions and countries, potentially leading to more consistent reimbursement decisions and greater access to innovative therapies for patients. Additionally, by reducing duplication of efforts and increasing efficiency in the assessment process, the JCA may help to reduce development costs for manufacturers, potentially leading to more affordable drugs and greater access for patients.
Theory vs practice – vision vs reality
Overall, while the JCA is unlikely to eliminate the challenges associated with local healthcare budgets, it may help to improve access to innovative therapies for patients by streamlining the assessment process and reducing regulatory delays.
The manufacturers must start with the internal processes now, to be able overcome the challenges of JCA and meet the opportunities. They will need to start with the internal preparation early, fully understanding the new JCA requirements, establish the new operation processes for dossier preparation and closely monitor its development. Manufacturers will need to continue to work closely with local stakeholders, including healthcare providers and payers, to ensure that drugs are accessible and affordable to patients in different regions and .
One aspect that may introduce complexity and uncertainty for manufacturers is the potential double process that could occur at both the local level and within the EU JCA framework. The ultimate goal is to achieve harmonization and convergence between the local and EU assessments, reducing duplicative efforts and enabling timely and consistent access to medicines across Member States.
It is expected that more clarity on the EU JCA process will be provided before its official implementation. As the process develops, regulatory bodies, such as the EMA and the European Commission, will likely release additional guidance, documents, and communications to address the uncertainties and provide more details to stakeholders, including manufacturers. This is a common practice to ensure that stakeholders have sufficient information to understand and prepare for upcoming changes.
* The content of this publication has been developed by the author. Consequently, statements on this publication are the personal opinion of the author and do not reflect the Merck company position (as employer of the author)
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