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

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.