New EU HTA system for medicines must work for innovators and patients

With many thousands of medicines at various stages of clinical developmentⁱ, this is a period of intense scientific innovation.

The medicines available to patients now, and the ones in the pipeline, are transforming healthcare outcomes for patients in the EU and across the world.

Before they reach patients in the EU, medicines must get regulatory approval to be used. That process is complex. Under the centralized authorization procedure, innovator companies – the developers of new medicines - submit a central marketing authorization application to the European Medicines Agency (EMA). The EMA scientifically assesses the application and provides an opinion for whether the medicine can be used for a given indication. The European Commission grants the EU marketing authorization for the medicine based on the EMA’s opinion.

After a medicine gets EU marketing authorization for use in a given indication, health technology assessments (HTAs) are carried out by national bodies in collaboration with the EMA. These assessments gather evidence about the therapeutic effect of a medicine, as well as evaluating its cost-effectiveness. The multidisciplinary process involves reviewing medical, economic, organizational, social and ethical factors that relate to the use of the medicine in healthcare systems.

In the end, national healthcare payers decide on the price of a medicine, after negotiating with the innovator and weighing all the evidence. Reimbursement decisions for each medicine are made nationally.

New framework

Changes are coming for the route medicines take on their journey to patients in the EU.

From January 2025, EU Member States, following the adoption of the EU HTA Regulation, will be able to jointly assess the clinical evidence for a new medicine. This once-siloed process should become more integrated and efficient, accelerating patients’ access to the latest innovations. A three-year transition period will aim to ensure a new framework that meets the needs of various stakeholders – the European Commission, Member States, HTA agencies, patients groups and innovators. All voices matter and they must be heeded.

The new EU HTA framework will be a key output of the EU Pharmaceutical Strategy whose goals of delivering faster access to innovation and strengthening EU competitiveness are shared by our industry. Member States will be able to use common HTA tools, methodologies and procedures across the EU. But they will still be responsible for assessing a medicine’s non-clinical elements, including economic, social and ethical ones, and making decisions on pricing and reimbursement. Medicines for cancer and rare diseases, and advanced therapeutic medicinal products - mostly cell and gene therapies - will be first to be included in the new system.

New science

The EU HTA Regulation is a step forward but policymakers need to keep pace with science. In the past decade, complex new treatment options have emerged, including cell and gene therapies, diagnostic tools for identifying genetic responses to medicines and tumor-agnostic cancer therapies. The future of healthcare will make more use of digital technologies and artificial intelligence. That will make the task of assessing the scientific, clinical and economic value of medicines more challenging. Innovators in our industry, known as health technology developers (HTDs), use a wide range of clinical trial designs that reflect changing research and development profiles. Regulatory pathways have evolved to reflect epidemiology and product complexity.

The EU HTA Regulation needs to work for everyone, especially innovators and patients. With the recent publication of the draft Implementing Act on Joint Clinical Assessments, a public consultation is under way seeking views on a proposal for how the new system will work in practice.

Workability

BMS is concerned about the workability of the new system. The proposal on the process undermines the capacity of the HTD to provide a high-quality joint clinical assessment submission to the assessors and co-assessor. The design of the system fundamentally challenges the resources needed from HTDs and HTAs. It sets an unbalanced time allocation for scoping the assessment by the HTA body (around 140 days) and for HTD submission (90 days).

The desire to keep the HTD at arm’s length in framing the scope of the assessment has significant repercussions on both sides. The involvement of the HTD in setting the scope is not an end in itself. Rather, it is a means to an end of streamlining the process, helping the assessor and co-assessor in developing a scope to share with Member States, and giving the HTD visibility and transparency to facilitate dossier planning. Involving the HTD will help to reduce the time needed for scoping which, in turn, will give more time to the work of developing the dossier itself.

The PICO, or Population, Intervention, Comparison and Outcome, sets the framework for the evaluation of a medicine and the data requirements for the innovator. HTDs should be involved in creating the scope of joint clinical assessments through the submission of robust and objective epidemiological, clinical and treatment patterns data.

This will help the HTAs to focus quickly and accurately on the PICO most relevant for patients and decision-makers. HTDs have access to important data and clinical practice data which can inform the scope of the joint clinical assessment. This can streamline the assessment process and efficiently arrive at the set of PICOs most relevant for Member States.

It is not sustainable to keep HTDs distanced from the design and operation of the new HTA framework, especially joint clinical assessments. The industry should be at the heart of the process that determines methodology, evidence development and operations. We welcome the proposed meetings on assessment scope but that is not enough. The workability of the new system is placed at risk by the design of the process. Given the complexity of the innovations already developed and the ones to come, ensuring smooth system workability is vital.

Streamlined framework

The EU HTA Regulation is a significant milestone for the European Health Union. It is an exercise in cooperation aimed at reducing fragmentation and duplication in the assessment of medicines by the EMA and national bodies. As we work towards implementation, it is important to ensure a predictable and streamlined EU framework for joint clinical assessments. That includes avoiding just amalgamating fragmented clinical HTA activity in Member States.

All of this work calls for collaboration. In a world of fast-moving science, there is no space for silos. Policymakers, working alongside others in the ecosystem, must design an evaluation framework that adapts quickly to emerging waves of scientific innovation.

Reference:
ⁱ IFPMA