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Contains fulltext : 294548.pdf (Publisher’s version ) (Open Access), OBJECTIVES: Uncertainty is a fundamental component of decision making regarding access to and pricing and reimbursement of drugs. The context-specific interpretation and mitigation of uncertainty remain major challenges for decision makers. Following the 2021 HTAi Global Policy Forum, a cross-sectoral, interdisciplinary HTAi-DIA Working Group (WG) was initiated to develop guidance to support stakeholder deliberation on the systematic identification and mitigation of uncertainties in the regulatory-HTA interface. METHODS: Six online discussions among WG members (Dec 2021-Sep 2022) who examined the output of a scoping review, two literature-based case studies and a survey; application of the initial guidance to a real-world case study; and two international conference panel discussions. RESULTS: The WG identified key concepts, clustered into twelve building blocks that were collectively perceived to define uncertainty: "unavailable," "inaccurate," "conflicting," "not understandable," "random variation," "information," "prediction," "impact," "risk," "relevance," "context," and "judgment." These were converted into a checklist to explain and define whether any issue constitutes a decision-relevant uncertainty. A taxonomy of domains in which uncertainty may exist within the regulatory-HTA interface was developed to facilitate categorization. The real-world case study was used to demonstrate how the guidance may facilitate deliberation between stakeholders and where additional guidance development may be needed. CONCLUSIONS: The systematic approach taken for the identification of uncertainties in this guidance has the potential to facilitate understanding of uncertainty and its management across different stakeholders involved in drug development and evaluation. This can improve consistency and transparency throughout decision processes. To further support uncertainty management, linkage to suitable mitigation strategies is necessary.

Contains fulltext : 282598.pdf (Publisher’s version ) (Open Access), PURPOSE: Primary congenital glaucoma (PCG) is a form of childhood glaucoma caused by maldevelopment of the anterior chamber. Disease severity differs greatly amongst patients. Ultrasound biomicroscopy (UBM) is a non-invasive technique that can visualize the anterior segment in infants in vivo. The purpose of this narrative review is to make an overview of the UBM data in PCG and study the applicability of UBM in characterizing the disease. METHODS: An online search was performed on PubMed in December 2020. After a critical appraisal of the included articles, study and patient characteristics were summarized. The UBM measurements of the anterior segment in PCG of the different studies were analysed. RESULTS: Six studies were included in this review. All were cross-sectional prospective studies. A total of 221 PCG eyes were examined. PCG eyes showed a larger trabecular iris angle, decreased iris thickness, narrower or absent Schlemm's canal and an increased zonular length compared to controls. Abnormal tissue membrane covering the trabecular meshwork and abnormal insertion of the iris and ciliary process were frequently found. The success rate of glaucoma surgery depended on the severity of anterior segment malformations found with UBM. CONCLUSION: Malformations of the anterior segment in PCG can be demonstrated by UBM in vivo. This imaging can help to characterize disease severity and might support surgical treatment decisions.

A workshop on The Next Generation Gamma-Ray Sources, sponsored by the Office of Nuclear Physics at the Department of Energy, was held November 17–19, 2016 in Bethesda, Maryland. The goals of the workshop were to identify basic and applied research opportunities at the frontiers of nuclear physics that would be made possible by the beam capabilities of an advanced laser Compton beam facility. To anchor the scientific vision to realistically achievable beam specifications using proven technologies, the workshop brought together experts in the fields of electron accelerators, lasers, and optics to examine the technical options for achieving the beam specifications required by the most compelling parts of the proposed research programs. An international assembly of participants included current and prospective gamma-ray beam users, accelerator and light-source physicists, and federal agency program managers. Sessions were organized to foster interactions between the beam users and facility developers, allowing for information sharing and mutual feedback between the two groups. The workshop findings and recommendations are summarized.

Photon strength functions are an important ingredient in calculations relevant for the nuclear synthesis of heavy elements. The relation to the photo-absorption cross section allows to experimentally constrain photon strength functions by investigating the photo-response of atomic nuclei. We determine the photoresponse of 66Zn in the energy region of 5.6 MeV to 9.9 MeV and analyze the contribution of the decay channel back to the ground state. In addition, for the elastic channel electric and magnetic dipole transitions can be separated. Nuclear resonance fluorescence experiments were performed using a linearly-polarized quasimonoenergetic photon beam at the High Intensity Gamma-ray Source. Photon beam energies in the energy range from 5.6 MeV to 9.9 MeV with an energy spread of about 3 % were selected in steps of 200 - 300 keV. Two High-Purity Germanium detectors were used for the subsequent gamma-ray spectroscopy. Full photo absorption cross sections are extracted from the data making use of the mono-energetic character of the photon beam. For the ground-state decay channel the average contribution of electric and magnetic dipole strength is disentangled. The average branching ratio back to the ground state is determined as well. The new results show lower cross sections compared to the values extracted from experiments using bremsstrahlung. In the latter the average branching ratio to the ground state needs to be estimated from statistical model calculations in order to analyze the data. The calculations underestimate this branching ratio compared to the values extracted within the present analysis, which would partly explain the high cross sections determined from the bremsstrahlung data.

A workshop on The Next Generation Gamma-Ray Source sponsored by the Office of Nuclear Physics at the Department of Energy, was held November 17-19, 2016 in Bethesda, Maryland. The goals of the workshop were to identify basic and applied research opportunities at the frontiers of nuclear physics that would be made possible by the beam capabilities of an advanced laser Compton beam facility. To anchor the scientific vision to realistically achievable beam specifications using proven technologies, the workshop brought together experts in the fields of electron accelerators, lasers, and optics to examine the technical options for achieving the beam specifications required by the most compelling parts of the proposed research programs. An international assembly of participants included current and prospective gamma-ray beam users, accelerator and light-source physicists, and federal agency program managers. Sessions were organized to foster interactions between the beam users and facility developers, allowing for information sharing and mutual feedback between the two groups. The workshop findings and recommendations are summarized in this whitepaper.

info:eu-repo/semantics/published

Objectives: Patient preference information (PPI) is gaining recognition among the pharmaceutical industry, regulatory authorities, and health technology assessment (HTA) bodies/payers for use in assessments and decision-making along the medical product lifecycle (MPLC). This study aimed to identify factors and situations that influence the value of patient preference studies (PPS) in decision-making along the MPLC according to different stakeholders. Methods: Semi-structured interviews (n = 143) were conducted with six different stakeholder groups (physicians, academics, industry representatives, regulators, HTA/payer representatives, and a combined group of patients, caregivers, and patient representatives) from seven European countries (the United Kingdom, Sweden, Italy, Romania, Germany, France, and the Netherlands) and the United States. Framework analysis was performed using NVivo 11 software. Results: Fifteen factors affecting the value of PPS in the MPLC were identified. These are related to: study organization (expertise, financial resources, study duration, ethics and good practices, patient centeredness), study design (examining patient and/or other preferences, ensuring representativeness, matching method to research question, matching method to MPLC stage, validity and reliability, cognitive burden, patient education, attribute development), and study conduct (patients' ability/willingness to participate and preference heterogeneity). Three types of situations affecting the use of PPS results were identified (stakeholder acceptance, market situations, and clinical situations). Conclusion: The factors and situation types affecting the value of PPS, as identified in this study, need to be considered when designing and conducting PPS in order to promote the integration of PPI into decision-making along the MPLC.

Background: Research has been mainly focused on how to elicit patient preferences, with less attention on why patients form certain preferences. Objectives: To assess which psychological instruments are currently used and which psychological constructs are known to have an impact on patients' preferences and health-related decisions including the formation of preferences and preference heterogeneity. Methods: A systematic database search was undertaken to identify relevant studies. From the selected studies, the following information was extracted: study objectives, study population, design, psychological dimensions investigated, and instruments used to measure psychological variables. Results: Thirty-three studies were identified that described the association between a psychological construct, measured using a validated instrument, and patients' preferences or health-related decisions. We identified 33 psychological instruments and 18 constructs, and categorized the instruments into 5 groups, namely, motivational factors, cognitive factors, individual differences, emotion and mood, and health beliefs. Conclusions: This review provides an overview of the psychological factors and related instruments in the context of patients' preferences and decisions in healthcare settings. Our results indicate that measures of health literacy, numeracy, and locus of control have an impact on health-related preferences and decisions. Within the category of constructs that could explain preference and decision heterogeneity, health locus of control is a strong predictor of decisions in several healthcare contexts and is useful to consider when designing a patient preference study. Future research should continue to explore the association of psychological constructs with preference formation and heterogeneity to build on these initial recommendations.

Background: Patient preferences (PP), which are investigated in PP studies using qualitative or quantitative methods, are a growing area of interest to the following stakeholders involved in the medical product lifecycle: academics, health technology assessment bodies, payers, industry, patients, physicians, and regulators. However, the use of PP in decisions along the medical product lifecycle remains limited. As the adoption of PP heavily relies on these stakeholders, knowledge of their perceptions of PP is critical. Objective: This study aimed to characterize stakeholders’ attitudes, needs, and concerns with respect to PP in decision making along the medical product lifecycle. Methods: Semi-structured interviews (n = 143) were conducted with academics (n = 24), health technology assessment/payer representatives (n = 24), industry representatives (n = 24), patients, caregivers and patient representatives (n = 24), physicians (n = 24), and regulators (n = 23) from seven European countries and the USA. Interviews were conducted between April and August 2017. The framework method was used to organize the data and identify themes and key findings in each interviewed stakeholder group. Results: Interviewees reported being unfamiliar (43%), moderately familiar (42%), or very familiar (15%) with preference methods and studies. Interviewees across stakeholder groups generally supported the idea of using PP in the medical product lifecycle but expressed mixed opinions about the feasibility and impact of using PP in decision making. Interviewees from all stakeholder groups stressed the importance of increasing stakeholders’ understanding of the concept of PP and preference methods and ensuring patients’ understanding of the questions asked in PP studies. Key concerns and needs in each interviewed stakeholder group were as follows: (1) academics: investigating the validity, reliability, reproducibility, and generalizability of preference methods; (2) health technology assess