OBJECTIVES: Health Technology Assessment (HTA) provides quantitative estimates of the efficacy and safety of new drug entities as well as cost-effectiveness to provide guidance for reimbursement and market access decisions. There is a growing recognition that when balancing costs and effectiveness, it is important to include inputs reflecting the patients’ voice.
METHODS: A review of HTA regulatory guidance documents and supporting literature was conducted for the US, Britain, Canada, Germany, and Australia. The role of the patient voice in relation to product appraisal was compared across these differing countries.
RESULTS: Examples of Patient Reported Outcomes (PROs) that have been evaluated within the quantitative HTA include impact on quality of life, utility, and functional status. Beyond PRO, the patient voice has been captured through advisory boards, patient advocacy groups and other direct patient input mechanisms. Of the five countries, Britain (NICE) has the most experience with both patient and public involvement in HTA via direct consultation, citizen groups and partners groups, supported by formal policy. The patient voice directly in policy decisions is emerging in both Canada and Australia as guidance documents and processes are updated. In Germany, the patient voice is sought via consultation with patient groups though their focus is on efficacy rather than issues of HRQL. In the US, comparative effectiveness research provides a vehicle for the patient voice with patient and consumer participation in priority setting and information dissemination as supported by proposed legislation.
CONCLUSIONS: The role of the patient voice in HTA extends beyond that of PRO measures and utility analysis. Direct input from patients and consumer advocacy groups are being included in policy decisions in all the countries whose guidelines were reviewed for this study. This can be seen as an essential step in applying the results of the quantitative HTA.
