Rejon-Parrilla Juan Carlos, Epstein David, Mestre-Ferrandiz Jorge, Espin Jaime
Health Technology Assessment Area (AETSA), Andalusian Public Foundation Progress and Health (FPS), Seville, Spain.
Department of Applied Economics, University of Granada, 18071, Granada, Spain.
Pharmaceut Med. 2025 Aug 8. doi: 10.1007/s40290-025-00576-9.
The development of health technologies involves complex, costly, and risky investments, with significant contributions from both public and private sectors. Recent EU pharmaceutical directives propose transparency on public funding to aid pricing negotiations and affordability. However, questions remain regarding how public investments should be measured and their influence on pricing and reimbursement (P&R) decisions. In this paper, we characterise public sector institutions as "payers," "R&D investors," and "regulators". Through a myriad of agencies and decisions, these institutions directly, indirectly or sometime unexpectedly influence risk and return on private research and development (R&D) through P&R, direct investments, and regulatory policy. P&R decisions by payers for innovative therapies influence risk and expected return of future R&D. Value-based pricing offers a more reliable signal of payers' priorities than cost-plus or (international) reference pricing. For greatest impact, public R&D investment should be directed to areas where markets are deficient, such as basic science, translational research, real-world studies, and towards emerging fields like AI and gene editing that will play an increasing role in healthcare and drug development. Applied R&D should be conducted on a financially sustainable basis. Licensing arrangements can be used to recover those investments, while promoting spillover benefits to wider society. Market access regulators are aware of the need for scrupulous transparency and neutrality, but other public sector actors (payers and R&D investors) also must recognise their policies affect the level playing field.
卫生技术的发展涉及复杂、成本高昂且有风险的投资,公共部门和私营部门都做出了重大贡献。欧盟最近的制药指令提议提高公共资金的透明度,以协助定价谈判和提高可承受性。然而,关于如何衡量公共投资及其对定价和报销(P&R)决策的影响,仍然存在问题。在本文中,我们将公共部门机构描述为“支付方”、“研发投资者”和“监管者”。通过众多机构和决策,这些机构通过P&R、直接投资和监管政策直接、间接或有时意外地影响私人研发(R&D)的风险和回报。支付方对创新疗法的P&R决策会影响未来研发的风险和预期回报。基于价值的定价比成本加成或(国际)参考定价更能可靠地反映支付方的优先事项。为了产生最大影响,公共研发投资应指向市场存在缺陷的领域,如基础科学、转化研究、真实世界研究,以及人工智能和基因编辑等新兴领域,这些领域将在医疗保健和药物开发中发挥越来越大的作用。应用研发应以财务可持续的方式进行。许可安排可用于收回这些投资,同时促进向更广泛社会的溢出效益。市场准入监管者意识到需要严格的透明度和中立性,但其他公共部门行为者(支付方和研发投资者)也必须认识到他们的政策会影响公平竞争环境。