Patient advocacy group involvement in health technology assessments: an observational study

Res Involv Engagem. 2021 Nov 25;7(1):83. doi: 10.1186/s40900-021-00327-5.


Background: In some jurisdictions, patients and patient groups may be invited to provide input when Health Technology Assessment (HTA) is used to inform decisions about which medicines should be subsidised or funded. This input can help frame the evidence from a patient perspective, address uncertainties in the evidence and interpret it for the local setting. However, there is currently no evidence linking patient involvement with positive reimbursement decisions.

Aim: We aimed to understand the expectations of patient involvement in the reimbursement process, especially among cancer patient advocacy groups (PAGs) in New Zealand (Aotearoa), South Korea and Taiwan.

Methods: We developed an online survey to help understand the role that cancer PAGs play in reimbursement processes and identify knowledge gaps about the processes that might impact the efforts of PAGs. The survey elicited the views of staff and patients affiliated with PAGs (n = 43) on current practices and how the assessment and reimbursement of new cancer drugs might be improved.

Results: There was variability in knowledge of the HTA assessment processes and in experience of being involved in them. Those with HTA experience were more likely to have confidence in the process. Those who had not been involved tended to have little awareness of, or frustration with, decision-making processes. Most identified cost, finances and economic assessments as key considerations in current processes. Some respondents had clear ideas about how their knowledge and involvement could improve processes to determine the value of new medicines. However, for many, a lack of information about the basis for decision making and opportunities to be involved was a barrier to identifying process improvement.

Conclusions: HTA is implemented primarily in countries seeking to have fair and equitable processes for funding medicines. PAGs often recognise the financial challenges of funding new medicines and share the desire for procedural fairness. The connection PAGs make between patient involvement and improved access to new medicines may be based on the belief they can add information to the evidence base, help solve problems, ensure fairness through transparency and/or influence the culture towards increased access to medicines they value.

Keywords: Asia–Pacific; Decision-making; Funding; Health technology assessment; Patient advocacy group; Patient and public involvement; Reimbursement decisions.

Plain Language Summary

WHAT ARE HEALTH TECHNOLOGY ASSESSMENTS?: Health technologies are medicines, devices and services used to prevent, diagnose or treat medical conditions. A Health Technology Assessment (HTA) considers the value of a health technology to help governments make fair decisions about which treatments to fund or subsidise. Patients and Patient Advocacy Groups (PAGs) may be invited to provide input into this process. WHAT DID OUR STUDY LOOK AT?: We surveyed staff and patients from cancer PAGs in New Zealand (Aotearoa), South Korea and Taiwan to understand their experiences and expectations of involving patients in HTA. A total of 43 people completed our survey. WHAT WERE THE RESULTS?: Overall, 20 of the 43 surveyed people (47%) were aware of the assessment process used to decide which medicines were funded in their country. (27 of these 50 people were PAG staff members.) Patients from South Korea and New Zealand were rarely included in the assessment process. Patient involvement was more commonly reported in Taiwan. The impact of input from patients on HTA was rated low by patients in both South Korea and New Zealand and average in Taiwan. A general lack of transparency, feedback on the process, and support from assessment agencies was also reported. Many of the patients surveyed wanted to see more patient involvement in the assessment processes. WHAT IS THE MAIN TAKE-HOME MESSAGE FROM THIS STUDY?: With improved guidance and feedback from assessment agencies, PAGs and patients believe their involvement can improve decision making on treatment funding and increase fairness through transparency.