Additional consensus recommendations for conducting complex innovative trials of oncology agents: a post-pandemic perspective.

In our 2020 consensus paper, we devised ten recommendations for conducting Complex Innovative Design (CID) trials to evaluate cancer drugs. Within weeks of its publication, the UK was hit by the first wave of the SARS-CoV-2 pandemic. Large CID trials were prioritised to compare the efficacy of new and repurposed COVID-19 treatments and inform regulatory decisions.

The National Institute for Health and Care Excellence information specialist development pathway: Developing the skills, knowledge and confidence to quality assure search strategies.

Quality assurance (QA) is an important process in ensuring that systematic reviews and other evidence syntheses are supported by a high-quality search. This paper describes how the National Institute for Health and Care Excellence (NICE) in the UK established a development pathway to ensure its information specialists had the skills, knowledge and confidence to undertake search QA. The key component of the pathway is that it blends technical knowledge with interpersonal skills.

Delayed payment schemes in Central-Eastern Europe and Middle-East.

The need for innovative payment models for health technologies with high upfront costs has emerged due to affordability concerns across the world. Early technology adopter countries have been experimenting with delayed payment schemes. Our objective included listing potential barriers for implementing delayed payment models and recommendations on how to address these barriers in lower income countries of Central and Eastern Europe (CEE) and the Middle East (ME).

Knowledge user survey and Delphi process to inform development of a new risk of bias tool to assess systematic reviews with network meta-analysis (RoB NMA tool).

Background: Network meta-analysis (NMA) is increasingly used in guideline development and other aspects of evidence-based decision-making. We aimed to develop a risk of bias (RoB) tool to assess NMAs (RoB NMA tool). An international steering committee recommended that the RoB NMA tool to be used in combination with the Risk of Bias in Systematic reviews (ROBIS) tool (i.

How can we optimise health technology assessment and reimbursement decisions to accelerate access to new cardiovascular medicines?

Regulatory approvals of, and subsequent access to, innovative cardiovascular medications have declined. How much of this decline relates to the final step of gaining reimbursement for new treatments is unknown. Payers and health technology assessment (HTA) bodies look beyond efficacy and safety to assess whether a new drug improves patient outcomes, quality of life, or satisfaction at a cost that is affordable compared to existing treatments.

Efficient searching for NICE public health guidelines: Would using fewer sources still find the evidence?

Systematic searches are integral to identifying the evidence that is used in National Institute for Health and Care Excellence (NICE) public health guidelines (PHGs). This study analyses the sources, including bibliographic databases and other techniques, required for PHGs. The aims were to analyse the sources used to identify the publications included in NICE PHGs; and to assess whether fewer sources could have been searched to retrieve these publications.

A Review of Economic Models Submitted to NICE's Technology Appraisal Programme, for Treatments of T1DM & T2DM.

In the UK, 4.7 million people are currently living with diabetes. This is projected to increase to 5 million by 2025.

Implementing shared decision-making in UK: Progress 2017-2022.

Shared decision making has been on the policy agenda in the UK for at least twelve years, but it lacked a comprehensive approach to delivery. That has changed over the past five years, and we can now see significant progress across all aspects of a comprehensive approach, including leadership at policy, professional and patient levels; infrastructure developments, including the provision of training, tools and campaigns; and practice improvements, such as demonstrations, measurement and coordination. All these initiatives were necessary, but the last, central coordination, would appear to be key to success.

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.

European collaboration on health technology assessment: looking backward and forward.

The establishment of health technology assessment (HTA) has been an important topic in Europe for many years. There have been a series of activities starting with first projects in 1994 leading to joint actions from the European Network of HTA (EUnetHTA) ending in 2021. This long interval of engagement with HTA structures, methodology, and processes by all member states led to a reliable basis for European collaboration in HTA.

Capturing what matters: updating NICE methods guidance on measuring and valuing health.

In July 2019, the National Institute for Health and Care Excellence (NICE) initiated a major review of its health technology evaluation methods to update its methods guide. This update has recently concluded with the publication of its health technology evaluation manual in January 2022. This paper reports the methods and findings of the review in relation to the recommended approach to use for the measurement and valuation of health-related quality of life (HRQoL) in submissions to NICE.

Immediate birth for women between 34 and 37 weeks of gestation with prolonged preterm prelabour rupture of membranes and detection of vaginal or urine group B streptococcus: an economic evaluation.

Objective: What are the costs, benefits and harms of immediate birth compared with expectant management in women with prolonged preterm prelabour rupture of membranes (PPROM) at 34 -36  weeks of gestation and detection of vaginal or urine group B streptococcus (GBS)?

Design: Mathematical decision model comprising three independent decision trees.

Setting: UK National Health Service (NHS) and personal social services perspective.

Population: Women testing positive for GBS with PPROM at 34 -36  weeks of gestation.

Building a model of health technology assessment cooperation: lessons learned from EUnetHTA joint action 3.

Objective: The European Network for Health Technology Assessment (EUnetHTA) was established in 2006. During its final project phase (joint action 3 [JA3]), it undertook an activity to define the scientific and technical principles of a model of health technology assessment (HTA) cooperation in Europe. This policy article presents the key learnings from JA3 partners about developing a model of HTA cooperation.

Innovative Patient Involvement During Covid-19: Keeping Patients at the Heart of HTA.

The COVID-19 pandemic and lockdown measures in the United Kingdom resulted in significant challenges and created opportunities for innovation to keep patients at the heart of HTA. The introduction of the Coronavirus Act 2020 and the associated public health guidance meant that NICE's conventional HTA methods were no longer feasible. NICE introduced rapid, innovative updates to patient and public involvement (PPI), decision-making meetings, and consultations to harness the expertise of patients and the public to ensure guidance addressed the expected concerns and identified barriers which could impact access.

Characteristics and outcomes of patients with COVID-19 with and without prevalent hypertension: a multinational cohort study.

Objective: To characterise patients with and without prevalent hypertension and COVID-19 and to assess adverse outcomes in both inpatients and outpatients.

Design And Setting: This is a retrospective cohort study using 15 healthcare databases (primary and secondary electronic healthcare records, insurance and national claims data) from the USA, Europe and South Korea, standardised to the Observational Medical Outcomes Partnership common data model. Data were gathered from 1 March to 31 October 2020.

Literature searching methods or guidance and their application to public health topics: A narrative review.

Background: Information specialists conducting searches for systematic reviews need to consider key questions around which and how many sources to search. This is particularly important for public health topics where evidence may be found in diverse sources.

Objectives: The objective of this review is to give an overview of recent studies on information retrieval guidance and methods that could be applied to public health evidence and used to guide future searches.

Assessing Technologies for COVID-19: What are the Challenges for Health Technology Assessment Agencies? Findings From a Survey and Roundtable Workshop.

Background: To date, health technology assessment (HTA) agencies have not been at the forefront of decision making regarding the adoption of interventions for coronavirus disease 2019 (COVID-19). Instead, policymakers have prioritised rapid action in response to the pandemic emergency, with no assessment of value for money. As COVID-19 vaccination coverage increases and healthcare systems begin to recover, HTA agencies will be expected to assess technologies for COVID-19.

The Challenges of Measuring and Valuing Quality of Life in Preschool Children: A Retrospective Review of NICE Appraisals.

Health technology assessment agencies evaluate interventions across the lifespan. However, there is no consensus about best-practice methods to measure health-related quality of life (HRQoL) in preschool children (<5 years) and data are often scarce. We reviewed methods used to capture the HRQoL of preschool children in past National Institute for Health and Care Excellence (NICE) appraisals to establish whether there is a need for better methods in this area and if so, to identify priority research areas.

Combination therapies for COVID-19: An overview of the clinical trials landscape.

The COVID-19 pandemic has driven an unprecedented level of global activity in drug discovery and clinical development for effective therapeutics targeting the coronavirus disease. There are currently 744 therapeutics being tested in 2879 clinical trials globally. Almost 90% of these clinical trials are focused on monotherapies.

A method for translating search strategies efficiently into HMIC and SPP.

Background: Small databases, such as Health Management Information Consortium (HMIC) and Social Policy and Practice (SPP), can add value to systematic searches. Search strategies designed for large databases may not be appropriate in small sources. A different approach to translating strategies could ensure that small databases are searched efficiently.

The NICE OECD countries' geographic search filters: Part 1-methodology for developing the draft MEDLINE and Embase (Ovid) filters.

Objective: There are no existing validated search filters for the group of 37 Organisation for Economic Co-operation and Development (OECD) countries. This study describes how information specialists from the United Kingdom's National Institute for Health and Care Excellence (NICE) developed and evaluated novel OECD countries' geographic search filters for MEDLINE and Embase (Ovid) to improve literature search effectiveness for evidence about OECD countries.

Methods: We created the draft filters using an alternative approach to standard filter construction.