arXiv:2605.06520cs.GTcs.LG2026-05

通过分阶段试验与补贴,提升高价值药物研发的社会效益。

Optimizing Social Utility in Sequential Experiments

论文配图:Optimizing Social Utility in Sequential Experiments
图 1 · 摘自论文原文
  • 分阶段随机试验+部分成本补贴,优化研发效率
  • 社会效用提升超35%,优于传统非序列方案
  • 适用于高风险高回报药物研发,适合政策制定者

高风险领域如药物研发的监管审批需大规模随机对照试验提供安全性和有效性证据。但高昂成本使开发者在缺乏确定疗效时望而却步,抑制了可能带来巨大社会效益的‘突破性’产品开发。本文提出一种统计实验协议:开发者分阶段进行随机对照试验,监管方部分补贴成本。通过信念马尔可夫决策过程建模,证明可通过动态规划高效求解最优策略。进一步发现,社会效用是补贴水平的分段线性凸函数,因此可通过分治法高效求得最优补贴。基于抗生素研发与审批公开数据的仿真显示,该协议相较标准非序列方案可提升社会效用超过35%。

原文摘要 · Abstract (English)

Regulatory approval of products in high-stakes domains such as drug development requires statistical evidence of safety and efficacy through large-scale randomized controlled trials. However, the high financial cost of these trials may deter developers who lack absolute certainty in their product's efficacy, ultimately stifling the development of `moonshot' products that could offer high social utility. To address this inefficiency, in this paper, we introduce a statistical protocol for experimentation where the product developer (the agent) conducts a randomized controlled trial sequentially and the regulator (the principal) partially subsidizes its cost. By modeling the protocol using a belief Markov decision process, we show that the agent's optimal strategy can be found efficiently using dynamic programming. Further, we show that the social utility is a piecewise linear and convex function over the subsidy level the principal selects, and thus the socially optimal subsidy can also be found efficiently using divide-and-conquer. Simulation experiments using publicly available data on antibiotic development and approval demonstrate that our statistical protocol can be used to increase social utility by more than $35$$\%$ relative to standard, non-sequential protocols.

社会效用随机试验补贴机制动态规划

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