Medialis Ltd., Banbury, UK.
Centre for Pharmaceutical Medicine Research, Institute of Pharmaceutical Science, Faculty of Life Science & Medicine, King's College London, London, UK.
Curr Med Res Opin. 2021 Jul;37(7):1249-1257. doi: 10.1080/03007995.2021.1927689. Epub 2021 May 21.
Those invested in the outcome of specific decisions may seek to inform the decision-maker by generating relevant evidence. With multiple decision-makers, the evidence generator benefits from understanding whether their needs differ. This was explored using relevant stakeholders' preferences on quality indicators (QIs) of rare disease patient registries (RDRs), a common vehicle for generating evidence, to support the adoption of new medicines.
The Jandhyala method was used to compare the observed consensus on RDR quality indicators from each stakeholder group (SHG) and between the combined pharmaceutical (pharma) and non-pharmaceutical (non-pharma) SHG population.
All non-pharmaceutical stakeholder groups were equally unique - suggesting QIs peculiar to themselves - Patients (13/102; 12.75%); Payors (5/102; 4.90%); Prescribers (7/102; 6.86%) to the same degree. Non-pharmaceutical stakeholders generated 31/102 (30.39%) unique QIs. Pharma stakeholder generated 25/102 (24.51%). This totals 56/102 (54.90%) QIs without a match. Two QIs were unanimously suggested and agreed upon by all SHGs (7/7; 100%) - "Engages with patients and gains their buy-in" and "Includes a core data set as part of outcomes". There was homogenous consensus on common QIs between: Commercial - Market Access (13/52; 25%; = .005) SHGs; Commercial - Medical Affairs (9/47; 19.15%; = .001) SHGs; Commercial - Payor (11/41; 21.15%; = .012) SHGs, and Market Access - Regulatory Affairs (10/41; 19.61%; = .008) SHGs.
A multiple stakeholder approach for generating real-world evidence can be justified. The potential mismatch of 54.9% between pharma and non-pharma stakeholders indicates a critical misalignment between generator and consumer of its RWE. Pharmaceutical SHG's were highlighted for greater alignment with Patients and Payor (non-pharma) groups.
关注特定决策结果的利益相关者可能会通过生成相关证据来影响决策者。对于多个决策者，证据生成者需要了解他们的需求是否存在差异。本研究使用罕见病患者登记处（RDR）质量指标（QI）的相关利益相关者的偏好来探索这一点，RDR 是一种常见的生成证据以支持新药采用的工具。
使用 Jandhyala 方法比较每个利益相关者群体（SHG）以及制药（pharma）和非制药（non-pharma）SHG 人群对 RDR 质量指标的观察一致性。
所有非制药类利益相关者群体都具有独特性，表明每个群体都有自己特有的 QI——患者（13/102；12.75%）；支付方（5/102；4.90%）；处方医生（7/102；6.86%）。非制药类利益相关者共提出 31/102（30.39%）个独特的 QI。制药类利益相关者提出 25/102（24.51%）个。这总计 56/102（54.90%）个 QI 没有匹配项。有两个 QI 得到了所有 SHG 的一致建议和认可（7/7；100%）——“与患者接触并获得他们的认可”和“纳入核心数据集作为结果的一部分”。商业准入-市场准入（13/52；25%； = .005）SHG 之间对常见 QI 有一致的共识；商业准入-医学事务（9/47；19.15%； = .001）SHG；商业准入-支付方（11/41；21.15%； = .012）SHG；市场准入-监管事务（10/41；19.61%； = .008）SHG。
可以证明采用多利益相关者方法生成真实世界证据是合理的。制药和非制药利益相关者之间潜在的 54.9%不匹配表明，RWE 的生成者和使用者之间存在严重的错位。制药 SHG 与患者和支付方（非制药）群体的一致性更高。
