Revising EU pharmaceutical legislation: will it foster drug repurposing?

Repurposing off-patent drugs can be a potential source of low-cost treatments for patients with unmet medical needs. Here, we review the proposed new European Union (EU) pharmaceutical legislation in which two articles address drug repurposing. We find certain barriers hindering the adoption of these new incentives by academic and not-for-profit stakeholders, including lack of knowledge on regulatory aspects, pharmacovigilance, and restrictions in data protection.

Factors for a broad technology assessment of comprehensive genomic profiling in advanced cancer, a systematic review.

Comprehensive Genomic Profiling (CGP) allows for the identification of many targets. Reimbursement decision-making is, however, challenging because besides the health benefits of on-label treatments and costs, other factors related to diagnostic and treatment pathways may also play a role. The aim of this study was to identify which other factors are relevant for the technology assessment of CGP and to summarize the available evidence for these factors.

Effectiveness of the Assessment of Burden of Chronic Conditions (ABCC)-tool in patients with asthma, COPD, type 2 diabetes mellitus, and heart failure: A pragmatic clustered quasi-experimental study in the Netherlands.

Background: The Assessment of Burden of Chronic Conditions (ABCC)-tool was developed to optimise chronic care.

Objectives: This study aimed to assess the effectiveness of the ABCC-tool in patients with COPD, asthma, type 2 diabetes, and/or heart failure in primary care in the Netherlands.

Methods: The study had a pragmatic, clustered, two-armed, quasi-experimental design.

How health technology assessment can help to address challenges in drug repurposing: a conceptual framework.

Drug repurposing faces various challenges that can impede its success. We developed a framework outlining key challenges in drug repurposing to explore when and how health technology assessment (HTA) methods can address them. We identified 20 drug-repurposing challenges across the categories of data access, research and development, collaboration, business case, regulatory and legal challenges.

A Blueprint for Multi-use Disease Modeling in Health Economics: Results from Two Expert-Panel Consultations.

Background: The current use of health economic decision models in HTA is mostly confined to single use cases, which may be inefficient and result in little consistency over different treatment comparisons, and consequently inconsistent health policy decisions, for the same disorder. Multi-use disease models (MUDMs) (other terms: generic models, whole disease models, disease models) may offer a solution. However, much is uncertain about their definition and application.

Software with artificial intelligence-derived algorithms for analysing CT brain scans in people with a suspected acute stroke: a systematic review and cost-effectiveness analysis.

Background: Artificial intelligence-derived software technologies have been developed that are intended to facilitate the review of computed tomography brain scans in patients with suspected stroke.

Objectives: To evaluate the clinical and cost-effectiveness of using artificial intelligence-derived software to support review of computed tomography brain scans in acute stroke in the National Health Service setting.

Methods: Twenty-five databases were searched to July 2021.

Exploring the Cost Effectiveness of a Whole-Genome Sequencing-Based Biomarker for Treatment Selection in Patients with Advanced Lung Cancer Ineligible for Targeted Therapy.

Objective: We aimed to perform an early cost-effectiveness analysis of using a whole-genome sequencing-based tumor mutation burden (WGS-TMB), instead of programmed death-ligand 1 (PD-L1), for immunotherapy treatment selection in patients with non-squamous advanced/metastatic non-small cell lung cancer ineligible for targeted therapy, from a Dutch healthcare perspective.

Methods: A decision-model simulating individual patients with metastatic non-small cell lung cancer was used to evaluate diagnostic strategies to select first-line immunotherapy only or the immunotherapy plus chemotherapy combination. Treatment was selected using PD-L1 [A, current practice], WGS-TMB [B], and both PD-L1 and WGS-TMB [C].

The added value of risk assessment and subsequent targeted treatment for epileptic seizures after stroke: An early-HTA analysis.

Introduction: The development of post-stroke epilepsy (PSE) is related to a worse clinical outcome in stroke patients. Adding a biomarker to the clinical diagnostic process for the prediction of PSE may help to establish targeted and personalized treatment for high-risk patients, which could lead to improved patient outcomes. We assessed the added value of a risk assessment and subsequent targeted treatment by conducting an early Health Technology Assessment.

An investigation of age dependency in Dutch and Chinese values for EQ-5D-Y.

Aims: The primary aim was to explore the age dependency of health state values derived via trade-offs between health-related quality of life (HRQoL) and life years in a discrete choice experiment (DCE). The secondary aim was to explore if people weigh life years and HRQoL differently for children, adolescents, adults, and older adults.

Methods: Participants from the general population of the Netherlands and China first completed a series of choice tasks offering choices between two EQ-5D-Y states with a given lifespan.

Cost of illness of patients with small fiber neuropathy in the Netherlands.

Neuropathic pain is associated with substantial healthcare costs. However, cost-of-illness studies of small fiber neuropathy (SFN) are scarce. Our aim was to estimate the healthcare, patient and family, and productivity costs of patients with SFN in the Netherlands from a healthcare and societal perspective.

The value of correctly diagnosing axial spondyloarthritis for patients and society.

Objective: To demonstrate the value of diagnosing axSpA, by comparing health and costs associated with available diagnostic algorithms and perfect diagnosis.

Methods: Using data from SPACE and other cohorts, a model was developed to estimate health (quality-adjusted life-years, QALYs) and costs (healthcare consumption and work productivity losses) of different diagnostic algorithms for axSpA amongst patients with low back pain referred to a rheumatologist, over a 60-year horizon. The model combined a decision-tree (diagnosis) with a state-transition model (treatment).

Oral Azacitidine for Maintenance Treatment of Acute Myeloid Leukaemia After Induction Therapy: An Evidence Review Group Perspective of a NICE Single Technology Appraisal.

The National Institute for Health and Care Excellence (NICE) invited the manufacturer (Celgene) of oral azacitidine (ONUREG), as part of the Single Technology Appraisal (STA) process, to submit evidence for the clinical effectiveness and cost-effectiveness of oral azacitidine for maintenance treatment of acute myeloid leukaemia (AML) after induction therapy compared with watch-and-wait plus best supportive care (BSC) and midostaurin. Kleijnen Systematic Reviews Ltd, in collaboration with Maastricht University Medical Centre+, was commissioned to act as the independent Evidence Review Group (ERG). This paper summarises the company submission (CS), presents the ERG's critical review on the clinical and cost-effectiveness evidence in the CS, highlights the key methodological considerations and describes the development of the NICE guidance by the Appraisal Committee.

Budget Impact of Three Improvement Targets for Compression Therapy for Patients with Deep Venous Thrombosis in The Netherlands.

Background And Objective: Compression therapy following deep venous thrombosis in the Netherlands is suboptimal. We assessed the budget impact of targeted care improvements.

Methods: We calculated the per-patient and population healthcare resource use and costs concerning 26,500 new patients each year in the Netherlands for the current pathways in region North Holland (divided into two parts: NH-A and NH-B) and region Limburg.

Comprehensive Review of Methods to Assess Uncertainty in Health Economic Evaluations.

Uncertainty assessment is a cornerstone in model-based health economic evaluations (HEEs) that inform reimbursement decisions. No comprehensive overview of available uncertainty assessment methods currently exists. We aimed to review methods for uncertainty assessment for use in model-based HEEs, by conducting a snowballing review.

Headroom Analysis for Early Economic Evaluation: A Systematic Review.

Objectives: The headroom analysis is an early economic evaluation that quantifies the highest price at which an intervention may still be cost effective. Currently, there is no comprehensive review on how it is applied. This study investigated the application of the headroom analysis, specifically (1) how the headroom analysis is framed (2) the analytical approach and sources of evidence used, and (3) how expert judgement is used and reported.

Cost-effectiveness of proton radiotherapy versus photon radiotherapy for non-small cell lung cancer patients: Exploring the model-based approach.

Introduction: Proton radiotherapy (PT) is a promising but more expensive strategy than photon radiotherapy (XRT) for the treatment of non-small cell lung cancer (NSCLC). PT is probably not cost-effective for all patients. Therefore, patients can be selected using normal tissue complication probability (NTCP) models with predefined criteria.