OBJECTIVES: Acute on Chronic Liver Failure (ACLF), occurring in approximately 6 to 20 per 1’000 person years, is an acute orphan disease with a high short-term mortality and high unmet medial need. There is no approved treatment for ACLF and standard medical treatment has only remained supportive of disease-defining organ dysfunction and failures. We have investigated and identified potential key parameters required for access and reimbursement decision-making for ACLF treatment.

METHODS: Healthcare payer and administrators were interviewed in November 2021 on health economics parameters between current ACLF treatment and hypothetical blinded potential new drugs. Results were analyzed to identify key reimbursement and access parameters.

RESULTS: In total, five US healthcare payer and administrator professionals participated, which consisted of one Managed Care Organization (MCO), two Hospital Pharmacists, and two hospital administrators. All participants enumerated that reduction in ICU stay is important (5 out of 5). Length of hospital stay, and transplant-free survival were deemed important by 4 of 5, while disease progression was mentioned by 3 of 5. Hospital readmission rates within 90 days and drug listed in medical guidance. In addition, potential new drug might be considered for a faster reimbursement if it was deemed a breakthrough treatment (i.e., orphan drug designation).

CONCLUSIONS: Reduction length of stays (days in ICU and hospital), transplant-free-survival, disease progression, hospital readmission rates, drug listed in medical guidance, and reduction in the number of organ failures were deemed key parameters for ACLF treatment access and reimbursement decision-making. These parameters should be further considered and developed as evaluation framework for acute orphan disease access and reimbursement evaluation in ACLF. Furthermore, future research needed in creating both ICD and DRG codes in ACLF.