Scientific and Social Value Judgments for Orphan Drugs in Health Technology Assessment

Abstract

Objectives.We explore how broader aspects of a treatment's value and the impact of the condition on patients not captured by routine Health Technology Assessment (HTA) methods using clinical and economic evidence, defined as "other considerations", may influence HTA processes in different settings. Methods.Countries included were England, Scotland, Sweden, and France. Data sources were the publicly available reports on HTA recommendations. Ten drugs with EMA orphan designation and appraised in England were selected. Qualitative thematic analysis was used to systematically identify and code all "other considerations" based on a previously validated methodological framework, which also coded whether it was provided by stakeholders, and how it influenced the decision. Results.A classification framework of scientific and social value judgments was developed and used throughout the study. 125 "other considerations" were identified and grouped into ten subcategories based on the information provided. 18% to 100% of these, depending on the agency, were put forward as one of the main reasons for the final decision potentially contributing to accepting a higher ICER or uncertain evidence. Some of these were nonquantified or non-elicited and pertained to the assessor's judgment. A taxonomy of these value judgments was created to be used in future cases. Results also contributed to better defining the determinants of social value and improving accountability for reasonableness. 3 Conclusions.The systematic identification of the scientific and social value judgments enables to better understanding the dimensions of value, which can be used to improve their transparency and consistent use across decisions and settings.4

Socioeconomic Characteristics of Countries Based on the Presence of Hta Agency

“…The systematic identification of the scientific and social value judgments provides to better understanding of the dimensions of value, which can be used to improve their transparency and consistent use across decisions and settings [8]. However, HTA is developing with uneven speed in middle-income coun- tries and many countries are building on the organizational and methodological experience from established HTA agencies.…”

Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches

“…While most processes, including the standard ones, are mainly interested in severity, unmet need and existence of treatment alternatives, some of the processes explicitly account for rarity or other criteria that may favour appraisal of RDTs (e.g. children, equality or the innovative nature of treatment [19]). Rarity is accounted for in Scotland (as part of its decision modifiers) and Netherlands, where greater uncertainty or unfavourable cost-effectiveness may be more acceptable.…”

Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches

“…While most processes, including the standard ones, are mainly interested in severity, unmet need and existence of treatment alternatives, some of the processes explicitly account for rarity or other criteria that may favour appraisal of RDTs (e.g. children, equality or the innovative nature of treatment [20]). Rarity is accounted for in Scotland as part of its decision modi ers.…”