A Proposal for Value-Based Managed Entry Agreements in an Environment of Technological Change and Economic Challenge for Publicly Funded Healthcare Systems.

Managed entry agreements (MEA) represent one of the main topics of discussion between the European National Payers Authorities. Several initiatives on the subject have been organized over the past few years and the scientific literature is full of publications on the subject. There is currently little international sharing of information between payers, mainly as a result of the confidentiality issues.

Preparing for tomorrow: Defining a future agenda.

Gene therapy will be the first long-term therapy with potential to produce a functional cure for haemophilia. As a single dose ('once-and-done') therapy with significant uncertainties regarding impact and duration of factor expression, flexibility and adaptability of (1) value framework, (2) health technology assessment (HTA) methodology, and (3) development of alternative payment models will be needed for adoption of this new technology and to facilitate transparent decision-making to support its implementation. The responsibility for each of these currently lies with distinct entities, underscoring a need for enhanced collaboration between all stakeholders, as expanded engagement by key stakeholders will be critical to optimizing the assessment of value, enabling an optimised approach to HTA, and opening receptivity to new and innovative payment models.

Alternative payment models for durable and potentially curative therapies: The case of gene therapy for haemophilia A.

Introduction: The emergence of durable and potentially curative cell and gene therapies (also known as advanced therapy medicinal products) with high price tags is challenging conventional health care payment systems. Among these are gene therapies in various phases of development for haemophilia A and B. The emergence of these therapies comes with clinical and economic uncertainties for payers, providers, patients, and manufacturers.

Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare.

Background: Advanced therapy medicinal products (ATMPs) are beginning to reach European markets, and questions are being asked about their value for patients and how healthcare systems should pay for them.

Objectives: To identify and discuss potential challenges of ATMPs in view of current health technology assessment (HTA) methodology-specifically economic evaluation methods-in Europe as it relates to ATMPs, and to suggest potential solutions to these challenges.

Methods: An Expert Panel reviewed current HTA principles and practices in relation to the specific characteristics of ATMPs.

Ethical Hurdles in the Prioritization of Oncology Care.

With finite resources, healthcare payers must make difficult choices regarding spending and the ethical distribution of funds. Here, we describe some of the ethical issues surrounding inequity in healthcare in nine major European countries, using cancer care as an example. To identify relevant studies, we conducted a systematic literature search.

Studying the Hurdles of Insulin Prescription (SHIP): development, scoring and initial validation of a new self-administered questionnaire.

Background: Although insulin therapy is well-accepted by symptomatic diabetic patients, it is still often delayed in less severe patients, in whom injectable insulin remains under-used. A better understanding of patients' perception of insulin would eventually help physicians to adopt the most appropriate dialogue when having to motivate patients to initiate or to intensify insulin injection.

Methods: The 'Studying the Hurdles of Insulin Prescription' (SHIP) questionnaire was developed based on a list of concepts derived from three diabetic patients' focus groups, and was included into two cross-sectional studies with similar design: SHIP Oral study and SHIP Premix study.