Abstract
Background:Evidence is valuable to inform decision making. Understanding stakeholder needs from the evidence of key stakeholders is empirically important to those involved in its generation. Where multiple stakeholders exist, an understanding of whether their questions are homogenous or heterogenous necessitates a dedicated approach that will be of value to those invested in the outcomes of their decisions.The pharmaceutical industry engages with non-pharmaceutical industry stakeholders: Payors, Prescribers and, under carefully controlled circumstances, patients when commercialising its medicines. This original research focussed on the differences between these groups and their pharma aligned stakeholders: Regulatory Affairs, Market Access, Commercial and Medical Affairs using measures of their common quality indicators (QIs) from rare disease patient registries.QIs were solicited using the Jandhyala method for observing item awareness and consensus from list generating questioning. They were compared for homogeneity between individual stakeholder groups and the combined pharma and non-pharma stakeholder group population.Results:All stakeholder groups were unique and suggested items peculiar to their own group.One hundred and eleven discrete QIs were identified: Commercial (8/111; 7.21%), Market Access (6/111; 5.41%), Medical Affairs (4/111; 3.60%), Regulatory Affairs (6/111; 5.41%), Patients (14/111; 12.61%), Payors (6/111; 5.41%), and Prescribers (9/111; 8.11%). Each stakeholder's proportion of unique QIs to the total was not statistically significant to the group as a whole.Non-pharma stakeholders generated 29/111 (26.13%) unique QIs, while pharma stakeholders generated 24/111 (21.62%). The total unique QIs across both stakeholder groups were 53/111 (47.75%). Two QIs were unanimously suggested and agreed upon by all stakeholder groups (7/7; 100%): 'Engages with patients and gains their buy-in' and 'Includes a core data set as part of outcomes'.There was homogeneity in consensus on common QIs between Commercial – Market Access (P=0.006), Market Access – Regulators (P=0.006), Commercial – Prescribers (P=0.001), Market Access – Prescribers (P=0.033), and Commercial – Medical Affairs (P=0.020).Conclusions:There is sufficient evidence to support a multiple stakeholder approach for generating real-world evidence. There was a mismatch between pharma and non-pharma stakeholders of 47.75% indicating redundancies of QIs on each side of this divide. Patients and Payor (non-pharma) groups have been highlighted for greater alignment with pharma stakeholder groups.
Values and Disadvantages of Outsourcing the Regulatory Affairs Tasks in the Pharmaceutical Industry in EU Countries
