OBJECTIVES: Assess payer and rare disease (RD) patient perception towards payer reputation, patient-centricity, orphan drug (OD) value, and incorporation of patient perspectives on value of ODs in drug formulary decision-making process in the U.S.

METHODS: In-depth qualitative telephone interviews with 24 payer and patient stakeholders. Participating payers included Managed Care Organizations and Pharmacy Benefit Managers; RD patient group included patient advocates, adult patients with RD and parents of children with RD.

RESULTS: Across all stakeholders, corporate reputation (41%), loyalty (32%), trust (18%) and credibility (16%) were most frequently cited in relation to customer perception of payers in the U.S; 44%/86% of patients/payers respectively identified payers as patient-centric; 41% of patients noted payers to be economically driven, instead of being patient-centric. 88% and 82% of patients noted their lack of knowledge of payer formulary committee composition and its decision-making process respectively. Patients identified several OD attributes delivering value, and 29% noted OD as invaluable/outstanding; 86% of payers noted OD value is low or hard to define. 88% of patients stressed the importance of considering patient input in formulary decisions, and 86% of payers indicated their skepticism/undesirability in considering patient-input, while both stakeholders noted benefit of patient-input in reflecting OD benefit/risk/overall-impact, supporting value judgments. Patients (53%) and payers (71%) noted negative perceptions payers hold towards direct patient-input prevents payer consideration of patient-input. Patients noted surveys/interviews/focus groups/written-correspondence, while payers noted patient advocates as key sources of patient-input. Mandatory review of patient-input and preference data, and incorporation of patient-input in formulary review materials were noted as ideal ways to incorporate patient-input; such action is considered to enhance payer’s patient-centricity, credibility, trust and reputation.

CONCLUSIONS: Formulating a mechanism to formally incorporate patient-input in payer formulary decision-making process could benefit payers by enhancing customer perception of payer’s patient-centricity and reputation, influencing credibility, trust and customer retention.