Health technology developers (HTD) planning submission of a centralized Market Authorization Application (MAA) to the European Medicines Agency (EMA) should being preparing today for the implementation of the European Health Technology Assessment Regulation (HTAR). The HTAR will take effect in January 2025 for oncology and/or advanced therapy medicinal product (ATMP) therapies, with other therapies following a staggered implementation process, including orphan drugs in 2028 and all drugs in 2030. This is a mandatory implementation that will be applicable for all HTDs who plan to prepare a centralized MAA submission to EMA.
The market access process in the pharmaceutical industry is complex and usually consists of several steps that require strategic planning, evidence generation, and stakeholder engagement. Companies that have set clear goals from the beginning, have good preparation, conduct research, and have a clear organizational and financial plan, are usually the winning party at market entry and product launch.
The HTAR implementation requires organizational efforts from the HTDs, where internal resources, skills and key roles need to be well defined and aligned with this new way of working. Information on the definition, impact, and how ProPharma can support organizations now with the different steps of the HTAR implementation taking effect in January 2025, can be found in our recent blogs in Part 1 and Part 2.
How to Prepare for Joint Clinical Assessments (JCA) in Europe
Now that we know the impact of the impending HTAR requirements, we need to discuss how to ensure your organization is ready for the new requirements coming early next year. Preparation for the upcoming joint clinical assessment (JCA) will help you create a successful market access plan in Europe. These four steps are based on what the HTAR requires from the HTDs in the new procedure.
Step 1: Introductory Training on HTAR Rules and Implications
To be able to reorganize your internal resources and skills appropriately to accommodate the upcoming requirements in a timely manner, it is important to be informed of the impact the HTAR rules will have on your health technology development. As a first step in this process, we will provide you with an introductory training to the HTAR process and its implications. We will outline the voluntary and mandatory sections and present to you with timelines following a step-by-step approach.
Step 2: Conducting a Technology-Specific Scoping Study
One of the main conditions in the HTAR, is that a joint clinical assessment (JCA) dossier preparation and submission will eventually be a requirement for all health technologies that are going through a centralized MAA with EMA. Joint clinical assessments will incorporate safety, epidemiological and clinical effectiveness considerations through the performance of systematic literature reviews (SLRs) and evidence synthesis. Approximately two months following the submission of a centralized MAA to the EMA, the EU member states will begin to discuss and define each country’s assessment scope and PICO (population, intervention, comparator(s), clinical outcomes) framework for the JCA. The different PICO frameworks are then presented to the Marketing Authorisation Holder (MAH) who will have 90 days to develop their JCA dossier, by incorporating safety, clinical, and epidemiological data following each PICO framework.
The 90-day timeline for the preparation of the JCA dossier is challenging and it is therefore advised to plan ahead. ProPharma can provide your team with support immediately to ensure you are well prepared for the assessment scope by conducting a technology-specific scoping study and developing a plan for the JCA preparations including potential budget outcomes and timelines. By having your technology-specific scoping study and the financial preparations for the JCA prepared, we will make sure that you are ready for the final preparation and submission of the JCA within the defined 90 days.
Step 3: Workshop Preparation to Define Optimal PICO(s)
Following the technology-specific scoping study, ProPharma will arrange a workshop with you to define the different PICO frameworks and how to best incorporate them from a safety, clinical, and epidemiological setting into the development of the JCA dossier. The preparation of the technology-specific scoping study and plan for the JCA dossier development will be arranged in more detail as part of this workshop and next steps forward will be agreed and discussed.
Step 4. Implementation of the Plan
The action steps and timelines for the technology-specific scoping study, the PICO frameworks, and the budget and timelines for the JCA development that have been outlined in Steps 2 and 3 will be implemented.
Joint Consultations Between EMA and HTAb
Joint scientific consultations (JSC) will be offered to HTDs as an opportunity to engage with either HTA bodies (HTAb) representatives alone or jointly with EMA representatives, and this will be implemented at the same time as JCA in January 2025. However, during the interim period, prior to the implementation of JSC, joint consultations can be conducted in parallel with EMA Scientific Advice, termed Parallel EMA/HTAb Scientific Advice.
ProPharma can support with the preparation and participation in these ongoing Parallel EMA/HTAb Scientific Advice meetings, which is strongly recommended to discuss any issues of concerns from EMA and HTAb to ensure that your company’s PICO and trial designs are accurate to facilitate the development plans. We can help you throughout the process from preparation of an application form, preparation of a briefing document to meeting preparation and participation.
Expert European Regulatory Affairs and Market Access Consultants
ProPharma is uniquely positioned to provide comprehensive support for the planning, preparation, and the implementation for upcoming HTAR initiatives in January 2025. Our team combines in-house specialists in both regulatory affairs and HTA, enabling us to offer clients unrivalled joint support throughout the entire process. Furthermore, our consultants remain at the forefront of the field by closely monitoring the latest EU guidance and actively engaging with national stakeholders, regulatory bodies, and HTA organizations.
In addition to our ability to support you with preparation the impending HTAR implementation, our team of regulatory consultants can also assist with anything from early regulatory and HTA interactions, to full MAA support with EMA, JCA preparations to the European Commission (EC), and national HTA preparations and submissions. Our unique ability to support your team throughout the full product life cycle enables us to help you achieve a successful product launch across the EU and beyond.
