Unique Czech model puts patients at centre of orphan drug reimbursement decisions

The EU's January launch of a new joint system for assessing health technologies has already been enhanced by Czechia, which has gone a step further by giving patients a voice in reimbursement decisions.

When a new amendment to the Czech public health insurance law came into effect in 2022, it gave patients a role in deciding whether certain medicines – specifically, those for rare diseases – should be reimbursed. In an interview with Euractiv, Eva Doleželová, Head of Medicines Evaluation Unit at the State Institute for Drug Control (SÚKL), said that in a European context, this is a unique model.

“It brings in the patient perspective – something that assessors usually do not have,” Doleželová explained. “They help us understand how the disease affects quality of life, family dynamics, and financial stability.”

In the Czech Republic, the reimbursement process for orphan medicinal products is managed by SÚKL, which prepares an assessment report. SÚKL acts as a non-decision-maker in this process.

What is unique about the Czech legal framework in the reimbursement process of orphan medicinal products is that patients and medical societies are official parties to administrative proceedings.

Not only can they submit input to the assessment report, but they also have representatives on the Advisory Board for the Reimbursement of Medicines for the Treatment of Rare Diseases at the Ministry of Health.

The final decision published by SÚKL is based on the Ministry of Health's decision, following the recommendation of the Advisory Board.

Informed patients matter

Doleželová emphasised that the process works best when patients are well-informed about how Health Technology Assessment (HTA) works.

“Patients who understand the HTA principles can better comprehend treatment options and health technologies. It allows them to take an active role in decisions about their care,” she says.

She added that such understanding contributes to patient safety, trust in the healthcare system, and helps patients advocate for their needs in broader policy debates. “Informed patients can better represent their interests, engage in health policy discussions, and even contribute to legislative development,” she notes.

To support this, SÚKL maintains regular communication with patient organisations.

“We now hold biannual meetings with patient groups, up from once a year. The agenda for these meetings is defined by the patient organisations themselves, based on their priorities and needs,” Doleželová explains.

Collaboration in practice

Whereas patients are formally part of the decision-making process for orphan drugs, their involvement in other reimbursement procedures remains optional. SÚKL welcomes their input in those cases, too. “Each statement – whether from patient organisations or medical societies – is considered during the assessment process,” said Doleželová.

Still, she acknowledges the reality of limited capacity. “Most patient organisations rely on volunteers, so it’s natural that they focus on processes where they have a real impact.”

To make patient participation easier, SÚKL has developed a structured submission form for patient organisations with guiding questions. “The form is used to conduct guide interviews with patients, ensuring the collection of relevant and meaningful information. I think this works well.”

New EU HTA regulation

Since January 2025, a new EU regulation on HTA has been in effect. The European system is focused on the clinical assessment of new health technologies. Decisions about reimbursement remain the responsibility of each member state.

“There was no previous official EU HTA system – this is completely new,” says Doleželová. “It’s similar to centralised regulatory procedures SÚKL is already familiar with.”

SÚKL was involved in the earlier EUnetHTA pilot project and spent considerable time preparing for the EU regulation. “We’ve only had training experience so far,” Doleželová says.

The first real assessments are expected in the first half of this year. “If all goes well, we might have practical experience with a full assessment by the end of the year,” she adds.

The EU framework also includes Joint Scientific Consultations (JSC), where HTA developers (pharmaceutical companies) can seek advice before entering the HTA process.

Patient representation is also built into the European HTA structure. “There are representatives from patient organisations who will be involved in the assessment process at the EU level,” Doleželová confirmed.

“We see patients’ willingness to learn and understand HTA as extremely positive. It’s not just about patients – it’s with patients,” she added.

[Edited by Vasiliki Angouridi, Brian Maguire]