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. 2023 Dec 18;13(12):e078134.
doi: 10.1136/bmjopen-2023-078134.

Defining 'therapeutic value' of medicines: a scoping review

Camille E G Glaus  1 , Andrina Kloeti  1 , Kerstin N Vokinger  2
Affiliations

Defining 'therapeutic value' of medicines: a scoping review

Camille E G Glaus et al. BMJ Open. .

Abstract

Objectives: In recent years, discussions on the importance and scope of therapeutic value of new medicines have intensified, stimulated by the increase of prices and number of medicines entering the market. This study aims to perform a scoping review identifying factors contributing to the definition of the therapeutic value of medicines.

Design: Scoping review.

Data sources: We searched the MEDLINE, CINAHL, Embase, Business Source Premier, EconLit, Regional Business News, Cochrane, Web of Science, Scope and Pool databases through December 2020 in English, German, French, Italian and Spanish.

Eligibility criteria: Studies that included determinants for the definition of therapeutic value of medicines were included.

Data extraction and synthesis: Data were extracted using the mentioned data sources. Two reviewers independently screened and analysed the articles. Data were analysed from April 2021 to May 2022.

Results: Of the 1883 studies screened, 51 were selected and the identified factors contributing to the definition of therapeutic value of medicines were classified in three categories: patient perspective, public health perspective and socioeconomic perspective. More than three-quarters of the included studies were published after 2014, with the majority of the studies focusing on either cancer disorders (14 of 51, 27.5%) or rare diseases (11 of 51, 21.6%). Frequently mentioned determinants for value were quality of life, therapeutic alternatives and side effects (all patient perspective), prevalence/incidence and clinical endpoints (all public health perspective), and costs (socioeconomic perspective).

Conclusions: Multiple determinants have been developed to define the therapeutic value of medicines, most of them focusing on cancer disorders and rare diseases. Considering the relevance of value of medicines to guide patients and physicians in decision-making as well as policymakers in resource allocation decisions, a development of evidence-based factors for the definition of therapeutic value of medicines is needed across all therapeutic areas.

Keywords: health economics; health policy; medical ethics.

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Conflict of interest statement

Competing interests: None declared.

Figures

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Figure 1
Flow diagram of search results.
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