Payer–patient engagement framework to strengthen ethical formulary decision-making in rare disease arena in the USA

Aim: Develop a Payer–Patient Engagement Framework (PPEF) in rare disease (RD) to incorporate patient preferences/input into payer formulary decisions in the USA. Materials & methods: Targeted literature reviews related to drug value assessment, ethical decision-making and corporate social performance theories were conducted, and integrated with results from 24 payer and patient stakeholder interviews in the USA to construct PPEF. Results: Published literature revealed scant use of patient preferences/input by payers, and the potential relationship between payer's ethical formulary decisions and payer's moral intent/behavior/actions, and their external credibility and reputation. Payer/patient interviews identified formal and informal ways to solicit patient input and incorporate them in drug formulary review materials. PPEF was formulated to engage patients, assess, and act on evidence and perform transparent external communication. Conclusion: PPEF could enable payers to formally solicit and utilize patient input to strengthen RD formulary decisions and enhance orphan drug access and RD patient outcomes.