Given the lack of a value framework for assessing health technologies in Asian settings, a value framework incorporating multiple-criteria decision analysis for new drugs under universal healthcare coverage in Taiwan was established.
The development process included (1) the adoption of 5 value domains (ie, Overall clinical benefit, Disease burden, Alignment with patient concerns, Economic value, and Feasibility of adoption into the health system) and 26 corresponding indicators, derived from the literature and expert discussions; (2) the creation of separate weighting schemes for 3 drug types—new oncology, new orphan, and other new drugs—based on inputs from multiple stakeholders (n = 86) using various weighting methods; and (3) the application of the value framework to cases of new oncology drugs.
Overall clinical benefit had the highest preference weight, irrespective of drug type, (ie, mean values [95% CIs] for new oncology, new orphan, and other new drugs: 32.5 [30.4–34.6], 30.6 [28.1–33.1], and 30.6 [28.7–32.6], respectively), weighting method, and stakeholder type. The 5 domain-derived weights (from the point allocation method) were comparable to the 26 indicator-derived weights (from the direct rating method), suggesting that the value framework with a short-form (domain-derived) weighting scheme is sufficient to support decision making under time and resource constraints.
A country-specific value framework incorporating multiple-criteria decision analysis for new drugs was developed in an Asian setting under universal healthcare coverage. It allows multiple stakeholders to systematically appraise all drug value attributes and provides a structured process for adapting and refining value assessments.
This research addresses the need for a structured way to assess the value of new health technologies, particularly drugs, in Taiwan's universal healthcare system. As new treatments, especially for cancer and rare diseases, become more expensive and their benefits more uncertain, it is crucial to have a reliable method for evaluating their overall value. Traditional assessment methods often focus mainly on clinical effectiveness and cost. However, this can overlook other important aspects such as patient concerns and social implications.
To address this, the study introduces a value framework based on multiple-criteria decision analysis (MCDA). This framework consists of 5 major areas: (1) overall clinical benefit (like how well a drug works and how safe it is), (2) disease burden, (3) alignment with patient concerns, (4) economic value, and (5) feasibility of adoption into the health system. Each area contains specific criteria to help decision makers fully evaluate a drug’s value. For instance, the area of overall clinical benefit includes factors like efficacy, safety, and unmet medical needs.
The researchers engaged various stakeholders, including healthcare professionals, policy makers, and patient representatives, to develop and weight these areas. The results showed that overall clinical benefit was the most important factor across different types of drugs, including new oncology and orphan drugs. This suggests that all parties recognize the significance of treatment effectiveness. The framework also allows for the prioritization of drug assessments based on specific contexts, which is essential for informed decision making regarding healthcare resource allocation.
This value framework offers a structured approach for stakeholders to systematically evaluate new drugs, leading to more transparent and consistent decisions. It aims to enhance the quality of health technology assessments in Taiwan, thereby improving patient outcomes and ensuring that resources are allocated effectively. Overall, the study highlights the importance of incorporating diverse value attributes in drug assessments to meet the needs and expectations of all involved in the healthcare system. Future research and broader stakeholder engagement will be essential for refining and adapting this framework to the evolving healthcare landscape.