Value Contribution of Palopegteriparatide in Adult Patients with Chronic Hypoparathyroidism using Multicriteria Decision Analysis (MCDA).

Abstract

Introduction: The study objective was to determine the value contribution of palopegteriparatide to the treatment of adults with chronic hypoparathyroidism, a rare endocrine disease, through a multi-criteria decision analysis (MCDA).

Methods: The Orphar-SEFH group framework for evaluating orphan drugs was used, along with the weighting performed by 98 national and regional evaluators. A multidisciplinary panel of seven experts individually scored the evidence matrix. The scores were collected and analysed using Microsoft Excel software programmed for MCDA study analysis. The results were discussed in a reflective discussion session.

Results: The expert panel considered chronic hypoparathyroidism to be a moderate-to-severe disease (mean ± SD: 3.3 ± 0.5) due to its multiorgan impact and associated comorbidities, with significant unmet needs (3.6 ± 1.0), particularly related to the lack of the physiological effect due to insufficient levels of parathyroid hormone. The experts found palopegteriparatide as an add-on treatment to conventional therapy (calcium and active vitamin D) to be much more effective than placebo plus conventional therapy, with this criterion receiving the highest score. The safety profile was considered acceptable (1.6 ± 1.1). The participants highlighted the positive impact of palopegteriparatide on patient-reported outcomes (2.9 ± 0.9) compared to placebo because of improvements in quality of life (QoL). Palopegteriparatide was seen as a potentially disease-modifying treatment, which could lead to savings in "other medical costs" (3.1 ± 1.1) and "non-medical/indirect costs" (2.0 ± 0.8), although no evidence was available, especially in the long term. The quality of the evidence was perceived as high (3.6 ± 0.5). The overall value contribution of palopegteriparatide was 0.58 (+ 1 = maximum value) compared to placebo. The study has some limitations, including a relatively small panel size and the exclusion of treatment cost as a criterion due to lack of pricing information at the time of evaluation.

Conclusion: According to MCDA, palopegteriparatide represents a valuable therapeutic option for chronic hypoparathyroidism treatment, particularly due to its demonstrated efficacy, which had an impact on patients' QoL, and the current unmet needs in this therapeutic area.