Pharmaceutical innovativeness index: methodological approach for assessing the value of medicines – a case study of oncology drugs

ABSTRACT

Background

We propose a framework to assess the value of pharmaceutical innovations, with explicit clinical and methodological parameters, based on the therapeutic value and health needs.

Research design and methods

The study was based on the adaptation of health technology assessment methods documented in the literature, which was applied to a sample of oncological drugs. Difficulties and issues during the application of those tools were identified and addressed to develop a new framework with new and revised domains and clear classification criterion for each domain. Scores were assigned to each level and domain according to their relevance to generate the final score of innovativeness.

Results

The Pharmaceutical Innovation Index (PII) includes four domains, two related to clinical and social dimensions – Therapeutic Need and Added Therapeutic Value – and other two about methodological features – Study Design and Quality (risk of bias). The scores combined after assigned to each domain results Index of the Innovativeness of the medicines represents the degree of pharmaceutical innovation.

Conclusion

This work proposes a transparent methodology with well-defined criteria and script; the algorithm developed with authors’ weightings and criteria may be switched to best adjust to other applications, perspective or clinical indications, while keeping the transparency and objectiveness.

KEYWORDS:

• Health technology assessment
• pharmaceutical innovation
• added therapeutic value
• innovativeness
• pharmaceuticals
• value-based innovation

Declaration of interest

The authors report there are no competing interests to declare. The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Author contributions

Concept and design: Ludmila P. Gargano, Juliana Alvares-Teodoro, Francisco de A. Acurcio, and Augusto A. Guerra Jr. Acquisition of data: Ludmila P. Gargano. Analysis and interpretation of data: Ludmila P. Gargano and Augusto A. Guerra Jr. Drafting of the manuscript: Ludmila P. Gargano. Critical revision of the paper for important intellectual content: Juliana Alvares-Teodoro, Francisco de A. Acurcio and Augusto A. Guerra Jr.

Acknowledgments

We thank the researchers from the Collaborating Center for Health Technology Assessment and Excellence (CCATES), especially Ariane A. André and Isabela C. M. de Freitas, for supporting acquisition, analysis, and interpretation of data.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Supplementary material

Supplemental data for this article can be accessed online at https://doi.org/10.1080/14737167.2024.2365985.

Additional information

Funding

This paper was supported by grants from the Minas Gerais State Research Support Foundation (FAPEMIG).