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  1. Fulltext Cost-Effectiveness Analysis of Spending on Research and Development to Address the Needs for Innovative Therapeutic Products in Indonesia
    Suwantika AA, Kautsar AP, Zakiyah N, Abdulah R, Boersma C, Postma MJ
    Ther Clin Risk Manag, 2020;16:969-977.
    PMID: 33116546 DOI: 10.2147/TCRM.S260377
    Background: The annual gross domestic expenditure on research and development (GERD) per capita of Indonesia ($24) remains relatively lower than the annual GERD per capita of neighboring countries, such as Vietnam ($36), Singapore ($1804), Malaysia ($361), and Thailand ($111).

    Objective: The aim of this study was to conduct a cost-effectiveness analysis of spending on healthcare R&D to address the needs of developing innovative therapeutic products in Indonesia.

    Methods: A decision tree model was developed by taking into account four stages of R&D: stage 1 from raw concept to feasibility, stage 2 from feasibility to development, stage 3 from development to early commercialization, and stage 4 from early to full commercialization. Considering a 3-year time horizon, a stage-dependent success rate was applied and analyses were conducted from a business perspective. Two scenarios were compared by assuming the government of Indonesia would increase GERD in health and medical sciences up to 2- and 3-times higher than the baseline (current situation) for the first and second scenario, respectively. Cost per number of innovative products in health and medical sciences was considered as the incremental cost-effectiveness ratio (ICER). Univariate sensitivity analysis was conducted to investigate the effects of different input parameters on the ICER.

    Results: There was a statistically significant association (P-value<0.05) between countries' GERD in medical and health sciences with the number of innovative products. We estimated the ICER would be $8.50 million and $2.04 million per innovative product for the first and second scenario, respectively. The sensitivity analysis showed that the success rates in all stages and total GERD were the most influential parameters impacting the ICER.

    Conclusion: The result showed that there was an association between GERD in medical and health sciences with the number of innovative products. In addition, the second scenario would be more cost-effective than the first scenario.

  2. Charting a course for global progress in PIDs by 2030 - proceedings from the IPOPI global multi-stakeholders' summit (September 2023)
    Van Coillie S, Prévot J, Sánchez-Ramón S, Lowe DM, Borg M, Autran B, et al.
    Front Immunol, 2024;15:1430678.
    PMID: 39055704 DOI: 10.3389/fimmu.2024.1430678
    The International Patient Organisation for Primary Immunodeficiencies (IPOPI) held its second Global Multi-Stakeholders' Summit, an annual stimulating and forward-thinking meeting uniting experts to anticipate pivotal upcoming challenges and opportunities in the field of primary immunodeficiency (PID). The 2023 summit focused on three key identified discussion points: (i) How can immunoglobulin (Ig) therapy meet future personalized patient needs? (ii) Pandemic preparedness: what's next for public health and potential challenges for the PID community? (iii) Diagnosing PIDs in 2030: what needs to happen to diagnose better and to diagnose more? Clinician-Scientists, patient representatives and other stakeholders explored avenues to improve Ig therapy through mechanistic insights and tailored Ig preparations/products according to patient-specific needs and local exposure to infectious agents, amongst others. Urgency for pandemic preparedness was discussed, as was the threat of shortage of antibiotics and increasing antimicrobial resistance, emphasizing the need for representation of PID patients and other vulnerable populations throughout crisis and care management. Discussion also covered the complexities of PID diagnosis, addressing issues such as global diagnostic disparities, the integration of patient-reported outcome measures, and the potential of artificial intelligence to increase PID diagnosis rates and to enhance diagnostic precision. These proceedings outline the outcomes and recommendations arising from the 2023 IPOPI Global Multi-Stakeholders' Summit, offering valuable insights to inform future strategies in PID management and care. Integral to this initiative is its role in fostering collaborative efforts among stakeholders to prepare for the multiple challenges facing the global PID community.
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