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Design for an Evaluation of Hemophilia Treatment Center 340B Drug Pricing Programs


"Problem:" Hemophilia is a debilitating, incurable, potentially fatal and catastrophically expensive rare bleeding disorder. Care provided by the US network of 130 regionally organized Hemophilia Treatment Centers (HTC) reduces disorder related mortality, morbidity, and costs. Unfortunately, HTC funding streams and levels have diminished. To generate revenue to meet rising demands, over two-thirds of HTCs operate outpatient pharmacies using federally discounted "340B" prices. However, 340B programs in the HTC setting have never been evaluated. Little is known whether HTC 340B programs are working as intended; about variations and determinants of adoption and implementation; demographic and clinical trends of people enrolled; and patterns of revenue generation and reinvestment per Congressional intent.

The objective of this dissertation is to devise a plan to evaluate HTC 340B Discount Drug Pricing programs. The proposed evaluation aims to examine and explain 340B program adoption and implementation patterns within HTC regions; to assess the impact of 340B adoption on HTC financial resources, capacity and services; and to project the implications of potential 340B program loss on HTC functioning.

"Methods:" The proposed plan for evaluation uses a mixed methods approach, and is guided by the Consolidated Framework for Implementation Research. The design compares 340B adoption, implementation, and impact over time; and solicits future projections, using the HTC as the chief unit of analysis. Data sources include archival retrieval of federal, state, regional, HTC, and HTC host institution documents; new semi-structured qualitative interviews with stakeholders, and a new organizational survey to assess organizational structure and process influences on 340B adoption and implementation. The evaluation's results should be disseminated through a full report, executive summary, and a brief written for a lay audience.

"Summary of findings:" If conducted, the proposed plan for evaluation will provide policymakers and other stakeholders with evidence regarding the adoption, implementation, and impacts of 340B on Hemophilia Treatment Center financial resources, capacity, and services. This evidence will enhance transparency, illuminate organizational and environmental determinants of 340B initiation and dissemination, inform the national 340B debate, and contribute to improved decision making at patient, clinician, and policy levels.

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