The process to obtain reimbursement for enzymatic debridement in clinically deep burns.

INTRODUCTION: Burn eschar removal by enzymatic debridement with NexoBrid® (EDNX) results in a maximum preservation of all viable tissue, which is the main advantage over traditional tangential excision. The authors participated in a marketing authorization holder process to obtain reimbursement from the national health authorities in Belgium. MATERIAL AND METHODS: The reimbursement process consisted of three phases, as specified by the reimbursement regulations required by the Belgian National Institute for Health and Disability Insurance (NIHDI). RESULTS: Forty-one patients with clinically deep 2nd and 3rd degree burns, treated with EDNX in two Belgian burn centers, were included in the registry for the first phase of the reimbursement process. The total success rate of the EDNX treatment was 95.1% (39/41). Over half of the burn wounds treated with NexoBrid® (55.2%) did not require any additional surgical debridement or skin grafting. To obtain definitive reimbursement, an extra 16 patients were included. In this population, 51.4% did not require any surgical intervention. The total success rate of the EDNX debridement in this group was 100%. Based on an estimated market share of 12% and around 75 patients in the third year after final reimbursement, a market access consultant calculated that NexoBrid® will realize yearly savings for the Belgian Healthcare budget of at least €30.000. CONCLUSION: Based on the results of this Belgian registry study in combination with the yearly healthcare budget savings, the NIHDI granted a final reimbursement for EDNX treatment in adults, endorsed by the Minister of Health on November 5th, 2019.

Critical Reflections on Reimbursement and Access of Advanced Therapies.

Background: The health economic literature has questioned the cost-effectiveness and affordability of advanced therapies, proposed adjustments to value assessment frameworks, and discussed the use of outcome-based managed entry agreements and staggered payments in the last few years. The aim of this manuscript is to conduct a critical reflection on assessment criteria and access conditions for reimbursement of advanced therapies. Methods: A narrative review of the peer-reviewed literature and grey literature was conducted in April 2021 by searching PubMed; Google Scholar; policy and legislative documents; websites of health technology assessment agencies, advanced therapy organisations, governmental advanced therapy innovation programmes, consultancy agencies; ISPOR conference abstracts and presentations. Results: Based on the available evidence, this manuscript argues that: a) advanced therapies can be cost-effective at high prices set by manufacturers; b) the economic evaluation framework adopted by many payers under-values these products; c) advanced therapies can be affordable and may not require spread payments; d) outcome-based managed entry agreements are theoretically attractive, but challenging in practice; e) the cost-effectiveness of advanced therapies depends on the outcome-based managed entry agreement and payment approach; f) there is a role for multinational collaborations to manage reimbursement and access of advanced therapies. Conclusions: This manuscript shows that there is no single approach to reimbursement and access of advanced therapies. Instead, we support a more tailored assessment of health economic aspects of advanced therapies, which considers the heterogeneity of these products and their target populations.

Prolonged enoxaparin therapy to prevent venous thromboembolism after primary hip or knee replacement. A cost-utility analysis.

BACKGROUND: Among patients undergoing elective total hip or knee replacement, prolonged prophylaxis with low-molecular-weight heparin significantly reduces the risk of symptomatic venous thromboembolism. Whether implementing routine prolonged prophylaxis is cost-effective remains uncertain. METHODS: We performed an economic modeling study to compare the costs and health outcomes of standard (12 days) with prolonged (42 days) enoxaparin prophylaxis against venous thromboembolism after elective total hip and knee replacement. The primary economic perspective was that of a societal healthcare payer, taking Belgium as a case country. We used cost-utility analysis, a form of cost effectiveness analysis in which costs are reported in monetary terms (euros) and health outcomes are converted into quality-adjusted life years (QALYs) gained, thereby incorporating a measure of quality of life (utility) into the health outcomes. Costs for diagnosis and treatment of proximal and distal deep vein thrombosis, pulmonary embolism, postphlebitic syndrome, and major bleeding were obtained from a Delphi panel (orthopaedic surgeons) and the official reimbursement rates (Federal Ministry of Health). QALYs for these health outcomes were based on utility scores as reported in the literature. The main outcome measure was the incremental cost-utility ratio, reported as the incremental cost per quality-adjusted life year gained (euros/QALY). The incremental cost-utility ratio refers to the amount of money needed to produce one additional QALY. We also performed sensitivity analyses on clinical and economic parameters to identify important model uncertainties. RESULTS: In the base-case analysis, incremental costs of prolonged prophylaxis amounted to 58 euros and 114 euros per patient, with an additional gain in QALY of 0.0083 and 0.0018 after total hip and knee replacement, respectively. Thus, a strategy of prolonged enoxaparin prophylaxis was associated with a cost-utility ratio of 6,964 euros/QALY and 64,907 euros/QALY after total hip and knee replacement, respectively. This tenfold difference in incremental cost-utility ratios between hip and knee replacement might have important practical implications. According to recent European guidelines, an intervention costing less than 20,000 euros per QALY is said to exhibit strong evidence for adoption, whereas one costing 20,000-100,000 euros exhibits moderate evidence for adoption. By current European guidelines, the cost of 6,964 euros and 64,907 euros per QALY gained would give strong evidence for adoption of prolonged enoxaparin prophylaxis among total hip replacement patients, but moderate evidence for adoption among total knee replacement patients. Sensitivity analyses using 20% changes from the base-case analysis showed this outcome to be robust. CONCLUSIONS: Our findings indicate that, among patients undergoing elective total hip or knee replacement, prolonged enoxaparin prophylaxis leads to increased health benefits at increased cost. Given the additional costs that healthcare decision makers in Europe are usually prepared to pay for a gain in utility, prolonged prophylaxis with enoxaparin is cost-effective after elective total hip replacement, and our data provide strong evidence for adoption of prolonged enoxaparin prophylaxis after elective total hip replacement.