Abstract

Introduction

The optimal launch and adoption of novel medicines require effective navigation of the commercial and regulatory landscape, including three key gatekeeper groups: regulators, payors, and prescribers. Traditionally, despite lacking generalisability, the pharmaceutical industry provides Phase III randomized controlled trial evidence to gatekeepers. Despite additional real-world evidence (RWE) improving the generalisability of evidence, the provision of RWE remains in its infancy. The industry may use a multiple stakeholder approach to RWE generation to address each group's priorities and expectations effectively.

Methods

The Jandhyala Method observed a consensus of expert opinion on the expected effects of supplementing randomised controlled trial evidence (RCTE) with RWE on the depth and time to maximal adoption. The study recruited participants from two of the three gatekeeper stakeholder groups (prescribers and payors) and their four aligned industry stakeholder groups (market access, commercial, medical affairs, and regulatory affairs) groups involved in the approval or adoption of investigational medicines.

Results

The observed consensus predicted a shortened time to maximal adoption by 22% and increased maximal adoption by 31% of a new medicine with additional RWE at launch. The suggested rationales for these effects varied across stakeholder groups and between specific effects. The results found the prescriber gatekeeper to be dominant in driving the predicted shortening of adoption. No such dominant subsystem was identified for the expected increase in adoption depth, although a potential rationale was the broader population observed in the supplementary RWE.

Conclusions

The proposed model provides an intuitive temporal framework within which subsystem logic can be further researched to identify and address stakeholder groups’ needs and weigh those needs. Preliminary results appear to support adopting a multiple stakeholder approach to RWE for augmenting the uptake of novel medicines.

中文翻译：

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用于评估产品发布时额外真实世界证据 (RWE) 的预期效果的药物采用模型

摘要

介绍

新药的最佳推出和采用需要对商业和监管环境进行有效导航，包括三个关键的看门人群体：监管者、支付者和处方者。传统上，尽管缺乏普遍性，制药行业仍向守门人提供 III 期随机对照试验证据。尽管额外的真实世界证据 (RWE) 提高了证据的普遍性，但 RWE 的提供仍处于起步阶段。行业可以使用多利益相关者的方法来生成 RWE，以有效地解决每个群体的优先事项和期望。

方法

Jandhyala 方法观察到专家意见的共识，即用 RWE 补充随机对照试验证据 (RCTE) 对最大程度采用的深度和时间的预期效果。该研究从三个看门人利益相关者群体中的两个（处方者和支付者）及其四个相关的行业利益相关者群体（市场准入、商业、医疗事务和监管事务）中招募了参与批准或采用研究药物的参与者。

结果

观察到的共识预测，在发布时具有额外 RWE 的新药的最大采用时间缩短了 22%，最大采用率提高了 31%。这些影响的建议理由因利益相关者群体而异，也因具体影响而异。结果发现，处方看门人在推动预计的采用时间缩短方面起主导作用。尽管潜在的理由是在补充 RWE 中观察到的更广泛的人群，但没有发现这种主要子系统会导致采用深度的预期增加。

结论

所提出的模型提供了一个直观的时间框架，在该框架内可以进一步研究子系统逻辑以识别和解决利益相关者群体的需求并权衡这些需求。初步结果似乎支持对 RWE 采取多方利益相关者的方法来增加新药的使用。