The rising cost of oncology care has motivated efforts to quantify the overall value of cancer innovation. This study aimed to apply the MACBETH approach to the development of a value assessment framework (VAF) for lymphoma therapies.

A multi-attribute value theory methodological process was adopted. Analogous MCDA steps developed by the International Society for Health Economics and Outcomes Research (ISPOR) were carried out and a diverse multi-stakeholder group was recruited to construct the framework. The criteria were identified through a systematic literature review and selected according to the importance score of each criterion given by stakeholders, related research and expert opinions. The MACBETH method was used to score the performance of alternatives by establishing value functions for each criterion and to assign weight to criteria.

Nine criteria were included in the final framework and a reusable model was built: quality adjusted life years (QALYs), median progression-free survival, objective response rate, the incidence of serious adverse events (grade 3–4), rates of treatment discontinuation due to adverse events, annual direct medical costs, dosage and administration, the number of alternative medicines with the same indication and mechanism, mortality of the disease. The weights of each criterion in the order presented above are 17.43 percent, 16.11 percent, 14.39 percent,13.54 percent,11.83 percent,11.30 percent,7.08 percent,4.59 percent, and 3.73 percent.

A criterion-based valuation framework was constructed using multiple perspectives to provide a quantitative assessment tool in facilitating the delivery of affordable and valuable lymphoma treatment. Further research is needed to optimize its use as part of policy-making.

This study explored patient involvement in healthcare decision-making in the Asia Pacific region (APAC) by identifying roles and factors influencing differences between healthcare systems. Proposed recommendations to enhance patient engagement were made.

This systematic literature review was conducted using studies from Australia, China, Japan, Malaysia, New Zealand, the Philippines, South Korea, Singapore, Taiwan, and Thailand. Studies were included if they provided data on patient involvement in health technology assessment (HTA) and/or funding decisions for medicines. Extracted data were scored according to eleven parameters adapted from the National Health Council (NHC) rubric, which assessed the level of patient involvement in healthcare system decision-making.

We identified 159 records between 2018 and 2022, including methodology guidelines from Government websites. Most mentioned parameters were patient partnership, patient-reported outcome, and mechanism to incorporate patient input. Limited information was available on diversity and patient-centered data sources. Tools for collecting patient experience included quality-of-life questionnaires, focus groups, interviews, and surveys, with feedback options like structured templates, videos, and public sessions.

Beyond input in assessment process, involvement of patients in decision-making phase has evolved within HTA bodies over time with considerable variation. Few APAC healthcare systems involve patients in the appraisal process as members of the recommendation or decision-making committee.

The findings indicate that while patient involvement in pharmaceutical reimbursement decisions exists, improvements are needed. Effective integration of patient input requires transparency, education, and resource planning. This study establishes a baseline to track progress and assess the long-term impact of patient involvement.

The aim of this study is to propose and validate a value assessment framework for Health Technology Assessment (HTA) for rare diseases drugs in Brazil.

A scoping review was performed to identify criteria used by HTA agencies in countries with public healthcare systems when evaluating orphan drugs. Based on the findings, a criteria framework for rare disease drugs was proposed for Brazil. Content validity was conducted over three rounds using Delphi technique and content validity ratio (CVR) approach was employed to evaluate the ratings from the eighteen stakeholders (experts and patients).

Twenty-nine HTA criteria for rare disease drugs were identified to compose the Brazilian framework. After three Delphi rounds, the final value framework comprised fifteen criteria categorized into four domains: disease-related factors, treatment-related factors, social and political factors, and economic factors. Among the most well-rated criteria by the CVR, considering the relevance attribute, were “relevance of outcomes for a rare disease,” “impact on patient’s quality of life,” “price negotiation,” and “adjusted cost-effectiveness threshold.” On the other hand, “budget impact threshold,” “innovative nature of treatment,” and “willingness to accept greater uncertainty in clinical evidence” received negative evaluations and were excluded from the final framework.

A value assessment framework validated by key stakeholders of rare diseases in Brazil could contribute to improve HTA transparency, decision making, and efficiency of the healthcare system, and inspire the development of a local guidance for rare-disease HTA.