OBJECTIVES: Assess U.S payer and rare disease (RD) patient perceptions of value of drugs in general, and orphan drugs (ODs).

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. Stakeholder perception of value of drugs/ODs was solicited, as part of contextualizing stakeholder decision-making process in RD arena.

RESULTS: When defining drug’s ‘value’ patients and payers have different, albeit, overlapping perceptions. Patients identified treatment efficacy (47%), impact on QOL/ADLs (41%), and affordability (29%) as key attributes defining drug’s value. Payers overwhelmingly (86%) identified “outcome per cost” while defining drug’s value; one payer noted “...whether something is worth it. When I think about value, I usually think about how much we're paying to achieve a particular goal and whether that is realistic or not; the value in a pharmaceutical is what it does for patient outcomes.” More specifically on value of ODs, patients identified ‘delivery of improved outcomes’ (47%), favorable benefit/risk ratio (29%) as key supporting attributes; 29% indicated value of ODs as ‘great/invaluable’. Payers overwhelmingly (86%) noted OD value as hard to define or of low value; 47% identified ‘delivery of improved outcomes’ as a key attribute influencing value of ODs. In relative ranking of OD attributes, patients/payers ranked high or very high – effectiveness (82%/86%), impact on QOL (76%/14%), safety (65%/43%), treatment complexity (35%/0%); 35% of patients ranked ‘out-of-pocket cost’ and 57% of payers ranked ‘overall treatment cost’ as high/very high.

CONCLUSIONS: There are considerable differences in patient/payer perceptions of drug’s value, and most specifically, value of ODs. Payer recognition of these differences and periodic assessment and incorporation of patient stakeholder perceptions in payer formulary decision-making process could enhance customer perception of payer’s patient-centricity.