Cost-effectiveness of somatrogon in the Spanish pediatric population with growth hormone deficiency

ABSTRACT

Objective

To analyze the cost-effectiveness of weekly somatrogon compared to daily growth hormones (GH-d) in the pediatric population of Spain with growth hormone deficiency (GHD).

Methods

Markov model with two states (patients with or without GH-d or somatrogon treatment) in prepubertal children (3 to 11 years and 3 to 12 years in girls and boys, respectively) with GHD in isolation or as part of multiple pituitary hormone deficiency and without previous treatment, from the perspective of the National Health System. The simulation of the economic model ends at the age of 18. The costs of hormones and monitoring were obtained from Spanish sources. The utilities were obtained from the literature. Spanish clinical experts validated the assumptions of the model.

Results

In the deterministic analysis, somatrogon would be cost-effective, compared to GH-d, with a cost per QALY (quality-adjusted life year) gained of €19,259 and a clinically relevant QALY gain (0.336). This result was confirmed in deterministic sensitivity analyses. According to the probabilistic analysis, somatrogon would be the dominant treatment, with a 61% probability of a willingness to pay of €25,000 per QALY gained.

Conclusion

Compared to GH-d, somatrogon is cost-effective in the Spanish pediatric population with GHD.

KEYWORDS:

• Cost-effectiveness
• economic evaluation
• GHD
• GH-d
• somatrogon

Article highlights

• Clinical studies of somatrogon have shown that it is not inferior to somatropin. However, the effects of long-term growth hormone deficiency treatment in clinical practice need to be investigated, particularly the implications of weekly (somatrogon) versus daily somatropin (GH-d) dosing on treatment adherence and consequent effectiveness.

• According to the Markov model carried out, somatrogon would be cost-effective, compared to GH-d, with a cost per QALY (quality-adjusted life year) gained of €19,259 and a clinically relevant QALY gain (0.336).

• This result was confirmed in the probabilistic analysis; somatrogon would be the dominant treatment, with a 61% probability of a willingness to pay of €25,000 per QALY gained.

Author’s contribution

C. Rubio-Terres and D. Rubio-Rodríguez have developed the adaptation to the Spanish National Health System of this economic model. M.A. Andreu Crespo, L. Castro-Feijóo, J.I. Labarta-Aizpún, C. Peral and J.A. Barrueta contributed to study conceptualization, design and revision of model. All authors had accessed to data, contributed to study conceptualization, methodology development and manuscript preparation. All authors listed made substantial contributions to the study in conceptualization and/or design of study, analysis and/or interpretation of data and manuscript preparation and/or review. All authors read, edited and approved the final manuscript. C. Rubio-Terres is the guarantor of the overall content of this manuscript.

Declaration of interest

C. Peral and J.A. Barrueta are employees of Pfizer SLU, Madrid, Spain. M.A. Andreu Crespo, L. Castro-Feijóo and J.I. Labarta-Aizpún has received speaker honoraria from Pfizer (Spain) and have participated in Pfizer advisory boards. D. Rubio-Rodríguez and C. Rubio-Terrés are employees of Health Value who received an honorarium from Pfizer (Spain) in connection with the development of this manuscript. The authors have no other 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 apart from those disclosed.

Reviewer disclosures

A reviewer on this manuscript has disclosed that they are an investigator in clinical trials of somatrogon for OPKO Health, a consultant for Pfizer and receive research funding from Pfizer. They are also a consultant for Ascendis which also makes a once weekly growth hormone approved in the US and elsewhere. They are a consultant for GenSci which also makes a once weekly growth hormone available in China. They are a consultant for and receive research funding from Novo Nordisk which also makes a once weekly growth hormone available in the US and elsewhere. Peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.

Supplementary material

Supplemental data for this article can be accessed online at https://doi.org/10.1080/14737167.2023.2256473

Additional information

Funding

This study was sponsored by Pfizer SLU, Alcobendas (Madrid), Spain.