Importance of aligning the implementation of new payment models for innovative pharmaceuticals in European countries.

Introduction: The uptake of complex technologies and platforms has resulted in several challenges in the pricing and reimbursement of innovative pharmaceuticals. To address these challenges, plenty of concepts have already been described in the scientific literature about innovative value judgment or payment models, which are either (1) remaining theoretical; or (2) applied only in pilots with limited impact on patient access; or (3) applied so heterogeneously in many different countries that it prevents the health care industry from meeting expectations of HTA bodies and health care payers in the evidence requirements or offerings in different jurisdictions.

Areas Covered: This paper provides perspectives on how to reduce the heterogeneity of pharmaceutical payment models across European countries in five areas, including 1) extended evaluation frameworks, 2) performance-based risk-sharing agreements, 3) pooled procurement for low volume or urgent technologies, 4) alternative access schemes, and 5) delayed payment models for technologies with high upfront costs.

Recommendations for patient involvement in health technology assessment in Central and Eastern European countries.

Introduction: Meaningful patient involvement in health technology assessment (HTA) is essential in ensuring that the interests of the affected patient population, their families, and the general public are accurately reflected in coverage and reimbursement decisions. Central and Eastern European (CEE) countries are generally at less advanced stages of implementing HTA, which is particularly true for patient involvement activities. As part of the Horizon2020 HTx project, this research aimed to form recommendations for critical barriers to patient involvement in HTA in CEE countries.

Adopting recommendations for implementing patient involvement in cancer research: a funder's approach.

Background: The role of patients in cancer research is undergoing a significant evolution as all stakeholders seek to enhance the level of direct patient involvement in the design and development of clinical trials. However, there are significant hurdles that patients, patient advocates, laboratory researchers, clinical investigators, and funding institutions must overcome to implement relevant patient involvement in all aspects of biomedical research. By using innovative grant funding models, philanthropic organizations can lead the field in overcoming these challenges.

[Changes resulting from the implementation of the new EU Clinical Trial Regulation 536/2014 from the perspective of patients].

The currently valid Regulation (EU) 536/2014 on clinical trials with medicinal products for human use brings some innovations that are of great importance for patients who participate in clinical trials and patients with a need for innovative therapies. These concern patient protection, especially for vulnerable patient groups, as well as the conditions for further use of data obtained in clinical trials. The introduction of the largely publicly available information system CTIS (Clinical Trials Information System) will significantly improve the transparency of ongoing clinical trials.

Building a Healthcare Alliance for Resourceful Medicine Offensive Against Neoplasms in Hematology Added Value Framework for Hematologic Malignancies: A Comparative Analysis of Existing Tools.

Objectives: The Innovative Medicines Initiative-funded, multistakeholders project Healthcare Alliance for Resourceful Medicine Offensive Against Neoplasms in Hematology (HARMONY) created a task force involving patient organizations, medical associations, pharmaceutical companies, and health technology assessment/regulator agencies' representatives to evaluate the suitability of previously established value frameworks (VFs) for assessing the clinical and societal impact of new interventions for hematologic malignancies (HMs).

Methods: Since the HARMONY stakeholders identified the inclusion of patients' points of view on evaluating VFs as a priority, surveys were conducted with the patient organizations active in HMs and part of the HARMONY network, together with key opinion leaders, pharmaceutical companies, and regulators, to establish which outcomes were important for each HM. Next, to evaluate VFs against the sources of information taken into account (randomized clinical trials, registries, real-world data), structured questionnaires were created and filled by HARMONY health professionals to specify preferred data sources per malignancy.

Methods to Generate Innovative Research Ideas and Improve Patient and Public Involvement in Modern Epidemiological Research: Review, Patient Viewpoint, and Guidelines for Implementation of a Digital Cohort Study.

Background: Patient and public involvement (PPI) in research aims to increase the quality and relevance of research by incorporating the perspective of those ultimately affected by the research. Despite these potential benefits, PPI is rarely included in epidemiology protocols.

Objective: The aim of this study is to provide an overview of methods used for PPI and offer practical recommendations for its efficient implementation in epidemiological research.

Core outcome set measurement for future clinical trials in acute myeloid leukemia: the HARMONY study protocol using a multi-stakeholder consensus-based Delphi process and a final consensus meeting.

Background: Acute myeloid leukemia (AML) is the most common acute leukemia in adults and has an unacceptably low cure rate. In recent years, a number of new treatment strategies and compounds were developed for the treatment of AML. There were several randomized controlled clinical trials with the objective to improve patients' management and patients' outcome in AML.