Health Technology Assessment (HTA) and Reimbursement: What is the Difference?

Once a new oncology medicine is developed, it must first be evaluated by the European Medicines Agency (EMA). This agency decides whether the medicine can be used in the 27 EU Member States, as well as Iceland, Liechtenstein, and Norway.

The EMA grants a marketing authorization only if it decides that the medicine’s benefits outweigh its risks.

But approval from the EMA is not enough. Before patients can access the medicine through their public healthcare system, two more questions need to be answered:

• How is this medicine doing comparatively to existing treatments?
• Will the national health system pay for it?

HTA: How is this medicine doing comparatively to existing treatments?

This is the main question answered through health technology assessment (HTA). It is a process used to evaluate a new medicine and how it compares to existing treatments.
HTA looks at:

• How this medicine works
• Whether it is safe
• Whether the additional benefits it offers are worth the cost.

HTA has two main parts:

• Assessment: This is the scientific review. Experts analyze the quality of the trial data — how the trials were conducted, what the results show, and whether the medicine was compared to the right alternatives. This part is focused only on the evidence, not on context (such as who will use the medicine).
• Appraisal: This is where the evidence is interpreted. Decision-makers use the findings from the assessment to decide how the medicine is doing comparatively to existing treatments, and in what situations. In this part, context matters — for example, whether the treatment is for children or for a rare disease.

Reimbursement: Will the national health system pay for it?

Even if a medicine is approved by the EMA and has a positive HTA, it won’t be accessible until it also receives a positive reimbursement decision.

The reimbursement process is conducted by health authorities, often different from the HTA bodies, at the national and/or regional level. They decide:

• If the medicine will be covered by the public system
• For which patients
• Under what conditions (e.g., hospital use only)
• At what price

Price negotiations take place between the health authorities and the pharmaceutical company. It is only after this step that the medicine becomes available to patients through the healthcare system.

In summary

• HTA provides scientific and clinical evidence.
• Reimbursement determines if and how patients can access the medicine.

Download the factsheet –> HTA and Reimbursement_Whats the Difference

How Medicines Are Evaluated in Europe

When new cancer medicines  are developed, pharmaceutical companies must apply for marketing authorisation from the European Medicines Agency (EMA). This process has been in place since 1995 and covers all 27 European Union (EU countries, plus Iceland, Liechtenstein, and Norway (called “Europe” in this factsheet).

The EMA reviews all the clinical trial data to decide whether the benefits of the medicine outweigh its risks—this is called the benefit-risk assessment. Only if the answer is yes, the medicine is authorized for use across Europe.

What Is New Since 2025? A European Joint Health Technology Assessment

As of January 2025, European countries are working more closely together—not just on marketing authorisation, but also on evaluating how new medicines are evaluated comparatively to existing treatments. This is done through a process called Health Technology Assessment (HTA), under a new EU law called the HTA Regulation (HTAR).

HTA answers a different question than EMA’s assessment: “How does this medicine compare to existing treatments—and is it worth the cost?”. To do so, a HTA is done in two main steps:

• Assessment: A scientific review of the evidence (how good are the trial results?).
• Appraisal: A decision-making step that looks at context (e.g. is it for a rare disease? Is it affordable?).

Since 2025, the assessment step is now done jointly at the European level (see Figure 1). The goal is to help patients get quicker access to effective medicines by avoiding repetition of work between countries.

Figure 1 Marketing authorisation and HTA processes since 2025

How a Medicine Gets Authorized in Europe

Pre-submission

Before applying for marketing authorisation, companies prepare by:

• Requesting ‘scientific advice’ from the EMA on how to best design their clinical trials.
• Applying for special status, such as orphan designation for rare diseases and Advanced Therapy Medicinal Product (ATMP) classification for gene or cell therapies
• Submitting a Pediatric Investigation Plan (PIP) if the medicine might be used in children (this is mandatory).

Regulatory evaluation

When ready, the company submits its marketing authorisation application to the EMA. The EMA’s Committee for Medicinal Products for Human Use (CHMP) reviews the evidence and decides whether to recommend approval. If approved, the European Commission grants marketing authorisation. If not, the medicine cannot be used in Europe.

How a Medicine is Evaluated Through European and national HTAs

Two New European-Level Steps in the HTA Process

Since 2025 two new HTA steps are performed at the European level (see Table 1)

Table 1: New European-Level Steps in the HTA Process

 These steps are led by the HTA Coordination Group (HTACG), which includes representatives from national HTA bodies.  The HTACG includes four subgroups:

• One for JSCs
• One for JCAs
• One to identify new and emerging technologies
• One to develop shared methods and procedures

The HTA CG is supported by a Secretariat hosted by the European Commission (HTA CG Secretariat). It ensures everything runs on time by coordinating tasks.

The HTACG is supported by a secretariat hosted by the European Commission known as the HTACG Secretariat. It coordinates tasks and ensures that timelines are met.

To include real-world perspectives, the HTACG works with the HTA Stakeholder Network, which brings together patients, healthcare professionals, researchers, and industry. This network helps identify experts, including patients, who can contribute to JSCs and JCAs.

In brief:

• The HTACG Subgroups conduct the work
• The HTACG Secretariat (European Commission) supports coordination
• The HTA Stakeholder Network brings in expert input, including patients and clinicians.

1.Joint Scientific Consultation (JSC)

A JSC helps companies improve their trial design before studies begin, ensuring they generate the kind of evidence needed for future HTA. While similar to the scientific advice provided by the EMA, the purpose differs: EMA advice focuses on designing safe and effective trials for marketing authorization, whereas JSCs aim to support future HTA decisions (see Table 2).

Table 2: Similarities and differences between early advice and JSC

Patients, clinicians, and other experts such as statisticians and health economists contribute to ensure planned studies address the most relevant questions.

Clinicians use the PICO framework—Patient, Intervention, Comparator, and Outcomes (see Table 3)—both when advising on study design during Joint Scientific Consultations (JSCs) and when reviewing medicines during Joint Clinical Assessments (JCAs). Patients, on the other hand, use a structured template to provide input during JSCs, and contribute using the PICO framework only during JCAs.

Table 3: PICO Framework

 2.Joint Clinical Assessment (JCA)

A JCA is a joint scientific report that compares a new medicine to current treatments:

• It’s written by two designated HTA bodies (the assessor and co-assessor) and serves as a shared scientific base for all countries.
• The process begins when a company applies to the EMA for marketing authorization.
• At that point, the company submits the intended use of the medicine (its claimed indication) to the HTACG Secretariat—but not yet the clinical data.

Next, the assessor and co-assessor draft a proposed assessment scope using the PICO framework. This draft is circulated to all European countries through a “PICO survey” to gather national needs and priorities. To ensure that patient perspectives are considered, both European-level and national patient experts may be invited to contribute.

Once the final PICOs are agreed, the company submits a dossier of evidence that addresses each PICO element. This dossier forms the basis for the scientific assessment by the assessor and co-assessor HTA bodies. Table 4 summarizes what a JCA —and what it is not..

Table 4: What JCA is and isn’t

We also examined how current patient involvement processes can inform the JCA framework. It identifies opportunities to replicate effective practices and proposes strategies to embed patient voices meaningfully into the JCA process.

Read the full article –> Patient Involvement in Health Technology Assessments: Lessons for EU Joint Clinical Assessments

National assessments and appraisals

Once the JCA is complete, each country’s HTA body uses the report as a common scientific base and adds its own assessment and appraisal according to national priorities and practices.

In some countries, such as Italy and Spain for example, HTA processes also take place at regional or local level—and in some cases even at hospital level—to determine how and where the medicine will be used.

A positive national HTA outcome therefore does not automatically guarantee that new medicine will be included in regional or hospital formularies. Additional approval steps are required. These steps are usually carried out by national, regional, or local health authorities—often distinct from HTA bodies. They decide:

• If the medicine will be covered by the public system
• Which patients will have access
• How it will be used (e.g. in hospitals only)
• At what price

These decisions are based on:

• National or regional/local health priorities
• The medicine’s impact on the national or regional/local healthcare budget
• Whether the treatment is cost-effective

Download the factsheet –>How Medicines Are Evaluated in the EU

Want to Learn More?

Download and read our companion factsheets on how patients are involved at European and national levels:

• Involvement of French Patients in Health Technology Assessment –> French patient involvement in HTA
• Involvement of German Patients in Health Technology Assessment

Impact of patient involvement in HTA

Our members and other patient groups representing those with acute leukemia have contributed to over 90 Health Technology Assessments (HTAs) for treatments conducted by HTA agencies across Canada, England, France, Germany and Scotland from 2020 to early 2023. Despite this significant effort, there is little that places our collective efforts into the wider context of who, how, where, when and what difference our submissions make to the recommendations made by HTA agencies that shape patient access to new treatments for acute leukemia.

We looked at he opportunities for patient involvement in HTA across six countries in Europe and Canada, comparing the approach each HTA agency takes to patient involvement. It is based upon HTA agencies websites, including their native language sources, as well as the wider literature published between January 2020 and April 2023.

Read the full report –> Opportunities and exploring the impact of patient involvement in HTA

We also looked at patient involvement in the HTA of medicines for the purpose of reimbursement across seven countries. That research was qualitative, describing the opportunities for involvement informed by a review of HTA agency websites, their annual reports and a rapid evidence review of the recent literature.

This quantitative analysis has looked at the scale of cancer patient group submissions to HTA agencies in Canada, England, France, Germany and Scotland and recommendations reached with and without patient group submissions to add to the picture given by the qualitative work.

This research highlights how often cancer patient groups put in the work to submit to HTA agencies. HTA agencies also have to put in work to manage the process. More research is needed to explore the difference all this effort makes to HTA decision-making and how to ensure it both has an impact and is also as efficient as it can be.

Read the full report –> Exploring the difference that patient involvement in HTA makes in cancer

We also looked into NICE and PACE.

Read the full report –> Report – NICE and Appealing

Read the full report –> PACE at SMC