The aim of the post below is to review highlights of Plenary Session 1 on November 7, 2022 at the ISPOR conference in Vienna, 2022: “The Convergence of HTA and Regulation: A New Health Technology Assessment Reality and Collaboration with Regulatory Agencies”. Here is how ISPOR introduced the session:
“With the implementation of the new Health Technology Assessment regulation it is expected to have a more systematic and synergic collaboration with regulatory agencies, namely the European Medicines Agency and EU Member States Agencies, taking into consideration the outcomes of the regulatory evaluation of new medicines and new indications. The interaction will be a reality in developing the HTA assessment, but the collaboration can be present in different phases of the life cycle, including horizon scanning, scientific advice, and generation of real-world evidence. This session will discuss how the different activities and remits of both regulatory and HTA assessment can be optimized if we are aiming at more convergence (regulatory & HTA) or the differences in the remits will prevail.”
Marcus Guardian, EUnetHTA, Netherlands, moderated the session, and it included the following speakers i) Emer Cooke, EMA, Netherlands, ii) Iga Lipska, Medical University of Gdańsk, Poland, iii) Carlos Martin, Ministry of Health, Spain, and iv) Rui Santos Ivo, INFARMED, Portugal.
First, the post outlines the plenary session and presents the questions that the moderator put to each speaker and details that emerged.
1. What are the synergies between a robust and strong Regulatory field and the HTA Coordination Group?
2. What are the benefits of collaboration between HTA and Regulatory?
3. How will the HTA regulation impact Central and Eastern European Countries?
4. What benefits are there in synergistic workflows, from regulatory, HTA, to pricing and reimbursement decisions?
5. Will fast-track assessments and conditional authorizations speed access or not?
6. What will a life cycle approach to evidence generation look like, in concrete terms?
7. What plans are there for HTA and Regulatory to close the evidence gap, and actively work together?
8. Key takeaways from the speakers.
Rui Santos Ivo
– Regulatory agencies and HTA have collaborated in the past to provide information to the citizens and patients in a way that connected many areas.
– There is a long-established regulatory system going back to 1965 at the time of the first directive in the EU. There is a strong network of heads of agencies that support the system and work closely with the EMA.
– There is a very strong basis from both Regulatory agencies and HTA agencies to strengthen ongoing interactions, so there will be an influence on both sides.
– There are procedures that demand earlier decisions which will drive greater interaction. Moreso, the regulation establishes concrete elements of collaboration between Regulatory and HTA agencies. Future legislation will add more details to how this collaboration will happen.
– A more integrated approach will benefit everyone, namely, the assessment of new healthcare technologies, medicines, and medical devices, which will be governed by future legislation.
– The interaction between the HTA Coordinating Group and the Regulatory Agencies will drive the convergence of processes and increase capacity, which will require sufficient resources. This is currently a discussion point on both sides, at the level of EMA and HTA agencies.
– From a public health and patient perspective, it’s essential that Regulatory and HTA agencies converge and coordinate the EMA and HTA assessments at an EU level and consider the lifecycle of the product.
– Innovation in healthcare only matters when patients have access. The decision-making system is very complex, with mandates defined for each decision-maker, including Regulatory and HTA agencies.
– To ensure patients have access, we have to connect these decisions, enhance the interfaces, but respect the Regulatory and HTA domains. EMA will continue to be responsible for the benefit risk analysis of healthcare technologies, but they will not be responsible for pricing and reimbursement decisions.
– The EMA will enable national regulators and payers to make decisions in healthcare systems. Priorities and evidence requirements need to be identified to support these decisions. Collaboration between Regulatory and HTA agencies can facilitate and support these decisions.
– Multi-topic and multi-stakeholder contexts between Regulatory and HTA agencies will define the ecosystem, but it will also involve academia, doctors, patients, and developers.
– Joint work on products and methodologies that reinforce collaboration through communication, information sharing, and training will ultimately ensure that patients have access to new healthcare technologies
– Central and Eastern European Countries will benefit from the HTA regulation. Patients in these countries experience an access gap. There are wide disparities of time from regulatory approval to HTA recommendation. For example, in Germany it is about 140 days, and in Poland over 500 days.
– Pharmaceutical companies sequence the launch of new products where applications are submitted much later to HTA agencies and Ministries of Health of Central and Eastern European countries, because of many factors. These delays contribute to the access gap in a patient’s experience.
– HTA processes in Central and Eastern European countries can really work well. For example, in Germany it takes about 89 days in 2021 for an HTA approval, but in Poland it took 82 days.
– Joint clinical assessments can help in the future to reduce this access gap to help patients.
– The new regulation will enable the Regulatory and HTA system to rapidly identify information gaps or uncertain sources to plan appropriately. This approach will assist with deciding the current and potential value of new healthcare technologies and at what price.
– The new system will help with generating timely information for relevant reports, but if we look at it as a workflow, then the system will overall benefit. All the structural elements are included, but a better workflow is needed. The system needs to flow with all information and uncertainties while identifying steps to introduce innovation in a timely manner.
Rui Santos Ivo
– EMA regulatory approval will lead to market authorization, but a HTA analysis will facilitate decision-making at the national level, specifically on pricing and reimbursement of new healthcare technologies and enabling patient access.
– The interaction and convergence of Regulatory and HTA agencies is essential to fill the gaps, enable fast track assessments, and apply conditional authorizations. However, work is needed on identifying the impact afterwards, and how Regulatory and HTA agencies make the final decision.
– In the Regulatory area, fast track assessments and conditional authorizations should not be a norm, which applied during the pandemic with rolling reviews, and is now being discussed while they review legislation. Ensuring that companies enter the different markets in a certain period will also be considered in this review.
– We should examine all elements in the new regulation to deepen them, locating them in the correct context, and avoiding a situation where a joint EU assessment results in difficulties for national decision-makers.
– The impact of regulatory decisions may have implications for health systems, their capacity, and sustainability. All procedures should be interlinked and support robust decision-making, especially on reimbursement, as this ensures access at a national level.
– An HTA recommendation is only one part of the overall system. Despite the Regulatory and HTA decisions that are provided, most countries have a pricing and reimbursement process, including pricing negotiations. Central and Eastern European countries are afraid of pressure to reimburse because of financial resources in their healthcare systems.
– Joint clinical assessments establish the expectation among patients that reimbursement would be provided, perhaps based on signals from EUnetHTA, the new HTA regulation, and initiatives on joint procurement by the Heads of HTA agencies.
– Regulators need to think about and develop a life cycle approach to evidence generation. Post licensing evidence has been of great value and generated opportunities to collaborate under the joint work plan. This is especially true for medicines where unmet needs and real-world evidence are needed after initial decision making.
– The Darwin EU project is a Pan-European Network of real-world data that the EMA put together with network partners to provide decision makers with valid and reliable real-world evidence. For example, additional information will be available on diseases, patient populations, and the safety and the effectiveness of medicines.
– Different evidence is available across the lifecycle of medicines, from clinical trials to real world evidence, during the early research stages to evidence generated after they launch the medicine. As new medicines come to market, we need earlier discussions on evidence requirements, especially for medicines addressing unmet needs under accelerated access schemes, which may include gene therapies, or histology independent indications in oncology.
– Regulators need to prioritize which medicines best address public healthcare needs. The EMA report on the 5-year experience with the priority medicines scheme will strengthen collaboration with downstream decision makers to enable patient access medicines for unmet medical needs.
– A life cycle approach to evidence generation depends on strengthening integration and collaboration across a broad range of decision makers in public health bodies. We also needed input from patients, healthcare professionals, academia, and developers of innovative healthcare technologies. We therefore need a stronger framework to allow developers to discuss their evidence plans across decision makers, and close identifiable gaps.
Rui Santos Ivo
– A gap may emerge with the development of assessments by Regulatory and HTA agencies and enabling the basis for national decisions. Ultimately, it’s the latter decision that will allow access.
– Generating evidence is necessary when available from developers, but it may also be put together from available sources and used when needed.
– The Darwin EU project run by EMA and other regulatory agencies is a very important structural project to generate evidence, but adding the heads of HTA agencies will also be beneficial because they will use of the evidence in post market authorization.
– Randomized control trials are still the gold standard. However, real-world evidence is also important for post launch evidence generation and reimbursement decisions, especially for reassessments. For example, with gene therapies, it will be essential to consider real world evidence.
– Guidelines are being produced by international organizations like ISPOR and EUnetHTA, so a continued focus on evidence generation is needed. The evidence requirements at each stage of the decision-making process must be deliberated.
Rui Santos Ivo
– Efforts to close the gap require that regulatory and HTA agencies analyze the consequences and types of decisions taken with available evidence. An overly simplistic approach may not work with various levels of evidence, uses of evidence, and types of decision-making, including pricing and reimbursement. More work is needed to develop methodologies.
– Horizon scanning is an important tool in HTA regulation and where future collaboration with EMA will be needed in terms of evidence needs and exchange of information.
– Closing the evidence is probably the most important thing that Regulatory and HTA agencies can do. The joint EMA / EUnetHTA 21 Work Plan identifies nine specific areas that will close evidence gaps. Activities include joint scientific consultation, exchange of information, methods and guidelines for real world evidence, methodologies for engagement of patients, extrapolation, horizon scanning, etc. These are the concrete steps that facilitate collaboration and deliver concrete endpoints.
· The current iteration of EUnetHTA will end September 2023, but a strong collaboration with EMA will endure through institutionalized collaboration between the HTA Coordination Group and EMA.
· The connected lifecycle approach to evidence is what will strengthen collaboration between HTA and Regulatory Agencies.
· Evidence requirements are different for the different decision makers. The HTA Regulation will strengthen the interfaces between Regulatory Agencies and HTA bodies.
· The EUnetHTA 21 consortium and joint work plans establishes the basis to progress the technical work, improving access to innovative and valuable medicines for patients.
· Identify clinical uncertainty and information gaps as early as possible. The process could be managed with all stakeholders aimed at introducing innovation in a timely manner, whilst managing financial uncertainty and enabling countries to remain budget sustainable.
· Evidence decision-making processes are critical, it’s all about the evidence.
· We need to be prepared for the future of HTA regulation and mapping national legislation framework at a local level because in January 2025 the process will start with oncology drugs.
· If the national legislation framework is not prepared on time, then patients will not have access to innovative medicines. There are other key success factors, like transparency and quality, so guidelines will be needed in the future to facilitate this work.
Rui Santos Ivo
· We have a robust system which should be implemented carefully and supported with good collaboration between all stakeholders, including the European Commission, the EMA, and all the national agencies.
· The new system is a tool to work towards equity and sustainability through our implementation plans and to ensure the best outcome is achieved.
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