A blog post based on the Patient Involvement in HTA in Europe project

No two countries have the same process for determining if a medicine should be reimbursed and made available through public healthcare systems. Most use a form of Health Technology Assessment (HTA) to assess the clinical and economic evidence, and many involve patient associations to provide additional insights which inform the decision.

However, how this patient involvement happens varies from country to country, and the precise impact of the patient voice on key decisions is sometimes unclear.

To provide a more harmonised approach to HTA, the European Commission is establishing cross-national collaboration under the Regulation on HTA (HTAR) which comes into force in 2025. This presents an opportunity to consider how best to involve patients in this process.

Identifying good practices

To inform this vital discussion, the EFPIA Patient Think Tank asked the Patient and Citizen Involvement in HTA Interest Group (PCIG) at HTAi to explore stakeholder experiences with patient involvement in HTA, in collaboration with the European Patient Forum (EPF) and the European Patient Academy for Technological Innovation (EUPATI). The project was launched in 2022, with funding support from EFPIA and PhRMA, and combined in-depth interviews with a Europe-wide survey to identify good practices.

One of the key observations is that patient can be involved at every step of the HTA process. Authorities across Europe use various methods to engage patients, including written submissions, interviews, focus groups, deliberative multi-stakeholder discussions in appraisal committees, hearings, consultations, or by having a seat in an organisational committee.

However, no example was found where patients are consistently involved in all steps in one country, and there was low cross-agency consistency in how patients were involved or how their input was used in the HTA report, recommendations, or subsequent decisions.  

Room for improvement

There are a range of shortcomings with current approaches to involving patients in HTA.

These include:

• The challenge of reaching and motivating relevant patients. The survey revealed that many patients say they know little about HTA and how they might be involved in this process.
• Delivering fit-for-purpose training and information to enable effective involvement.
• Guidance for evaluating patient involvement in HTA and giving feedback to those who contributed. This lack of feedback and reporting leads to very low motivation among patients to become involved.

On the other hand, a growing trend was observed that patient stakeholders are becoming more involved at the organisational level. There are examples of standing patient committees (e.g. in Wales) or advisory boards that include patient stakeholders.

Finally, we observed a lack of institutional memory, particularly among the patient community, which may be holding back progress.

In some organisations, patients who had been involved in HTA had moved on and there were no records or other ways to access the input given or any experience related to this process. Due to the lack of such records, it can be assumed that the learning within the patient organisations on how to approach HTA may be limited.

How to involve patients

Our research led to the development of good practice recommendations, including:

• Establish an advisory board or standing committee to advise on patient and public involvement activities.
• Create fit-for-purpose guidance, training, and information for patients and researchers. This could include summaries of technical information in lay language.

Involving patients is a learning process for patient stakeholders, HTA organisations and researchers. The aim should be to make this experience fruitful and productive, and to ensure all stakeholders feel that their time and resources are well spent.

By sharing good practices and recommendations, we are confident that stakeholders can raise the standards of patient involvement in HTA across Europe when implementing the HTAR.

Read the HTA 360 research brief here and the full report here.