Stakeholder point of view on prescription drug affordability - a systematic literature review and content analysis.

OBJECTIVES: The objectives of this research were to: 1) understand perspectives on affordability of pharmaceutical drugs from the point of view of stakeholders as reported in published peer-reviewed journals and conferences; 2) evaluate if (and how) perspectives on affordability overlapped across stakeholders. METHODS: The systematic literature review followed Cochrane and PRISMA guidelines. Content analysis with iterative and systematic coding of text was conducted, to identify themes. RESULTS: A total of 7,372 unique citations were eligible, and 126 articles included for final synthesis. For patients, 6 core themes emerged: financial barriers, adherence, access, patient-provider communication, financial distress, and factors that impact affordability. For payers, 5 core themes: financing schemes, cost-effectiveness, budget impact, private vs. public preferences, and ethics. For providers, 3 themes: patient-provider communication, physician prescribing behavior, and finding alternatives to support patient access. For policymakers, 2 themes: measuring affordability and the role of government. Limited articles representing the manufacturer perspective were identified. Perspectives of decision makers (payers, policymakers) did not overlap with those affected by affordability (patients, providers). CONCLUSIONS: This research highlights the multi-dimensionality of drug "affordability." Multiple factors beyond cost influence patient affordability implying interventions can help alleviate affordability issues for some patients. The lack of overlap highlights potential hazards that decisions related to out-of-pocket spending, insurance coverage, reimbursement, and rationing occur without explicitly considering patient and provider perspectives.

Budget projections and clinical impact of an immuno-oncology class of treatments: Experience in four EU markets.

BACKGROUND: Immunotherapies have revolutionized oncology, but their rapid expansion may potentially put healthcare budgets under strain. We developed an approach to reduce demand uncertainty and inform decision makers and payers of the potential health outcomes and budget impact of the anti-PD-1/PD-L1 class of immuno-oncology (IO) treatments. METHODS: We used partitioned survival modelling and budget impact analysis to estimate overall survival, progression-free survival, life years gained (LYG), and number of adverse events (AEs), comparing "worlds with and without" anti-PD-1/PD-L1s over five years. The cancer types initially included melanoma, first and second line non-small cell lung cancer (NSCLC), bladder, head and neck, renal cell carcinoma, and triple negative breast cancer [1]. Inputs were based on publicly available data, literature, and expert advice. RESULTS: The model [2] estimated budget and health impact of the anti-PD-1/PD-L1s and projected that between 2018-2022 the class [3] would have a manageable economic impact per year, compared to the current standard of care (SOC). The first country adaptations showed that for that period Belgium would save around 11,100 additional life years and avoid 6,100 AEs. Slovenia - 1,470 LYGs and 870 AEs avoided; Austria - respectively 4,200, 3,000; Italy - 19,800, 6,800. For Austria, the class had a projected share of about 4.5 % of the cancer care budget and 0.4 % of the total 2020 healthcare budget. For Belgium, Slovenia, and Italy - respectively 15.1 % and 1.1 %, 12.6 %, 0.6 %, and 6.5 %, 0.5 %. CONCLUSION: The Health Impact Projection (HIP) is a horizon scanning model designed to estimate the potential budget and health impact of the PD-(L)1 inhibitor class at a country level for the next five years. It provides valuable data to payers which they can use to support their reimbursement plans. POLICY SUMMARY: The model is a strategic tool which allows decisionmakers to assess the implications of policy decisions, such as additional investment, or accelerated access to IOs. It can drive tangible population health benefits by eliminating the questions around PD-(L)1 inhibitor spending and its related outcomes.