The multiple stakeholder approach to real-world evidence (RWE) generation: observing multidisciplinary expert consensus on quality indicators of rare disease patient registries (RDRs)

Abstract

Introduction 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. Methods 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. Results 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%; p = .005) SHGs; Commercial – Medical Affairs (9/47; 19.15%; p = .001) SHGs; Commercial – Payor (11/41; 21.15%; p = .012) SHGs, and Market Access – Regulatory Affairs (10/41; 19.61%; p = .008) SHGs. Conclusions 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.