The pan-European joint HTA regulation provides a way to evaluate the ethical, social, medical, and economic implications for new health technologies. Health technologies includes medical devices and equipment, pharmaceutical products, diagnostic tests, treatment methods and procedures, and prevention programs.

The goal of a health technology assessment is to compare a new health technology to existing technologies and determine if it is better, worse, or the same by applying pre-defined parameters (e.g. medical, economic, ethical). Policy makers are meant to use the results of a HTA to make decisions about health policies and reimbursement. This particular regulation aims at streamlining clinical data assessments across the EU and reduce duplication of similar assessments for member states.

The EU has created a new mandatory appraisal process called Joint Clinical Assessment (JCAs). An overarching EU HTA body will conduct JCAs for new pharmaceutical products and high-risk medical devices. The JCAs will be available to all EU member states to reduce potential duplicate evaluations. Biopharma companies may benefit because they will only have to submit data to the JCA once instead of sending it to each individual EU member state.

EU member states will also participate in Joint Scientific Consultations (JSCs) to help biopharma companies with clinical study designs, so they are able to produce better “fitting” clinical evidence to address evidence needs for the EU market. The JSCs will provide advice on the requirements for HTA submissions that will help biopharma companies.

The JCA creates a framework for HTA cooperation among member states in the EU. Considering that it is a mandatory appraisal process, biopharma companies must become familiar with it.

The key JCA principles for biopharma to pay attention to are:

Creating a better health policy by:

• Improving access to medicines
• Promoting innovation
• Comparing the effectiveness of products
• Preferring randomized controlled trials (RCTs), but allowing observational data and real-world evidence (RWE) to be used

Enhancing transparency in decision making by:

• Harmonizing methodological requirements
• Focusing on the standards of evidence-based medicine
• Actively involving stakeholders through the stakeholder network

Avoiding redundancies or parallel structures by:

• Having one dossier instead of 27
• Avoiding the need to submit data on the national level after it is already submitted to the JCA
• Providing complementary clinical data on the national level if it is requested

Preserving national decision-making sovereignty by:

• Creating HTA separation for an assessment vs appraisal
• Being free of value judgment and summary on the added medical benefit
• Making the final appraisal on the national level
• Keeping decisions on pricing, pricing regulation, and reimbursement on the national level

It is important to note that JCAs are not legally binding, but they are to be given due consideration as part of the individual country-driven reimbursement decisions.

First, parallel to the regulatory filing, pharmaceutical companies file a letter of intent to formally start the JCA process. Second, the JCA subgroup initiates an assessment scope to request information on critical parameters from all member states following the Population, Intervention, Comparator, and Outcomes (PICO) scheme or survey, to be answered in approximately two weeks.

Third, the JCA subgroup determines the scope and consolidates the PICO scheme. Since each EU member state submits one PICO scheme, all 27 are consolidated into one. Fourth, the dossier is submitted 45 days prior to the expected positive opinion by the Committee for Medicinal Products for Human Use (CHMP), at the latest. Finally, the Coordination Group's assessment report is made 30 days after the EU commission decision.

Biopharma companies will want to pay attention to the EU HTA timeline because it creates centralized market authorization for pharmaceuticals.

2022 – 2024: This is the pilot phase that includes the formation of a Coordination Group with one member per EU country to establish formal and methodological guidance. It also includes the formation of a stakeholder network.

January 12, 2025: JCA becomes mandatory for oncology drugs and advanced therapy medicinal products (ATMPs). The implementation of JSC begins.

January 13, 2028: JCA becomes mandatory for orphan drugs.

January 13, 2030: JCA becomes mandatory for all drugs registered centrally by the European Medicines Agency (EMA).

The biopharma industry needs to be aware of some of the potential concerns about the pan-European joint HTA. For example, EU member states may use clinical evidence for HTA in different ways and may have different views on the relevance of different categories of evidence. The standard of care, which the HTA uses to compare the new technology, can also differ among member states.

Other concerns include:

•  Differences in how member states compare data with some preferring RCTs and others using indirect treatment comparisons through network meta-analysis
• Treatment comparisons made in existing RCTs may not be relevant to some member states if their current standard of care is different
• For some cases, such as treatments for rare diseases, only single-arm clinical studies will be available with or without historical controls
• Differences in how member states accept biomarkers or intermediate outcomes in clinical data and the level of validation they require

The development of the JCAs raises many concerns about methodological and practical guidelines. Biopharma companies need to stay alert and encourage a nuanced approach to the guidelines.

Although the pilot phase of the pan-European joint HTA is not over and will continue until 2024, biopharma companies need to start preparing now. They must pay attention to the development of guidance documents for the HTA process, JCA, or JSC and potentially participate in public consultations to make sure the industry perspective is represented. Collaboration is a key component of any HTA, and biopharma has an important role to play.

Disclaimer: This article is meant to be informative only and should not be relied upon as legal advice.