Outcomes Based Agreements (OBAs) are financial arrangements that offer the opportunity to align payment to health outcomes in the real-world, and share the financial risk by providing long-term solutions that grant access to medicines, with reimbursement only when performance is achieved. OBAs are most likely to be useful when there is high uncertainty in the clinical data, but they are difficult to design and implement, and other financial options are usually preferred by payers. As a result, OBAs have been more the exception than the norm, and there is not a clear pattern that indicates if an OBA is likely to succeed in practice.

Through a retrospective OBA exercise with NHS Wales (Project IDEATE: Innovation in Data to Evolve Agreements That Enhance patient health outcomes), we have explored the circumstances under which an OBA might be most appealing to payers, and assessed implementation challenges and solutions, to propose a framework to evaluate the feasibility of a medicine for an OBA.

Along with mitigating some of the clinical uncertainties associated with a lack of mature data at the time of launch, an OBA must also consider other factors: the commercial viability of the agreement, the associated administrative burden, and its cost of implementation. Also, the Health System commitment to a Value-Based Healthcare agenda and, most importantly, its willingness to offer long-term sustainable solutions to optimise treatment, are key to support this approach.

Practical considerations include: how the relevant outcomes are going to be selected and tracked in the real-world, how the whole model is going to fit within the current procurement and finance infrastructures, and how industry works in collaboration with the Health System.

Insights from Project IDEATE will be used to explore how our OBA feasibility framework might be applied in the future.

Recruitment and retention of participants in randomized controlled trials (RCTs) is challenging, and is why many RCTs fail or are not completed on time. Digital approaches such as social media, data mining, email or text messaging could improve recruitment and/or retention, but how well they match these purposes is unclear. We used systematic methods to map the digital approaches that have been investigated during the past 10 years.

We searched Medline, Embase, other databases and relevant web sites in July 2018 to identify comparative studies of digital approaches for recruiting and/or retaining participants in clinical or health RCTs. Two reviewers screened references against protocol-specified eligibility criteria. Studies included were coded by one reviewer (with 20 percent checked by a second reviewer) using pre-defined keywords to describe characteristics of the studies, populations and digital approaches evaluated.

We identified 9,133 potentially relevant references, of which 100, reporting 101 unique studies, met the criteria for inclusion in the map. Among these, 95 percent of studies investigated recruitment but only 11 percent investigated retention. Study areas included health promotion and public health (36 percent), cancer (17 percent), circulatory system disorders (13 percent) and mental health (10 percent). Most study designs were observational (89 percent). The most frequent digital approaches for recruitment were internet sites (53 percent of recruitment studies), social media (42 percent), television or radio (31 percent) and/or email (31 percent). For retention these were email (63 percent of retention studies) or text messaging (38 percent). Time and costs of recruitment were reported in 17 percent and 30 percent of recruitment studies respectively, whilst costs were reported in 19 percent of retention studies.

A wide range of digital approaches has been studied, in many combinations. Evidence gaps include lack of experimental studies; studies on retention; and studies for specific populations (e.g. children or older people) and outcomes (e.g. user satisfaction). More robust experimental studies, perhaps conducted as studies-within-a-trial (SWAT), are needed to address knowledge gaps and ensure that estimates of digital tool effectiveness and efficiency are reliable.

Burkitt lymphoma (BL) is a rare and highly aggressive subtype of non-Hodgkin’s lymphoma. Several studies have identified prognostic factors (PFs) for disease progression and mortality among adults with BL. However, there is no consensus on risk stratification based on PFs. This study aims to identify, critically assess, and synthesize the available evidence on PFs for survival in adults with BL.

A systematic review of the literature was conducted. MEDLINE, Embase, and CENTRAL were searched from inception to 22 February 2022. Randomized or non-randomized clinical trials and longitudinal observational studies were eligible for inclusion. Reference screening, data extraction, and risk-of-bias assessment using the Quality in Prognosis Studies (QUIPS) tool for prognostic factor studies were conducted independently and in duplicate. Publication bias was examined by visual inspection of funnel plots. Effect measures and the corresponding 95% confidence intervals were pooled with an indirect variance estimation in meta-analyses using Review Manager 5, and sensitivity analyses were conducted. Certainty of evidence was assessed using GRADE.

The search identified 1,119 references after duplicate removal. Of these, 76 potentially relevant papers were selected for full-text assessment and 36 studies (N=10,882) reported in 39 articles were eligible for inclusion. Older age, higher performance status, and central nervous system involvement were associated with poorer overall survival (OS) and progression-free survival (PFS). Black patients exhibited significantly lower OS and relative survival. Bone marrow involvement and higher albumin levels were associated with poorer OS. Treatment with rituximab and treatment with methotrexate were associated with better OS and PFS. No significant differences in survival were found for HIV status, sex, and risk stratification.

This study, framed within a collaboration with European Reference Network EuroBloodNet, provides a comprehensive and methodologically rigorous evidence review on PFs in adults with BL. Several significant associations of PFs and survival estimates were observed, providing data to inform treatment decisions and to improve patient care.

The aim of this initiative was to examine collaboratively, in a multi-stakeholder team (health technology assessment (HTA) practitioners with patient involvement expertise, health technology industry, patient advocates, health policy experts, patient engagement experts), whether evidence generated through social media research (SMR) fills current information gaps relating to insights on specific aspects of patient experiences, preferences, or patient needs and delivers additional value to HTA.

The framing of the project was done in a co-creative, deliberative multi-stakeholder process. Challenge and refinement happened through discussions with 25 independent stakeholders from HTA bodies, industry, academia, and patient advocacy. For critical themes identified during the framing phase, scoping literature reviews were performed including the state of methods and examples for the use of SMR in HTA.

The framing and stakeholder discussions specified a set of expectations and requirements, and the scoping reviews revealed the current state of methods and usage of SMR in health-policy decision making.

The project concluded that SMR can contribute new, relevant evidence to HTA. It is however recommended to evolve the science through defining best practices when planning, conducting, and using SMR and to conduct multi-stakeholder pilot SMR projects to address questions relevant to current HTAs and to validate and improve the proposed practices.

Although health technology assessment (HTA) and healthcare quality improvement are distinct processes, a greater level of alignment in outcome measures used may increase the quality and efficiency of data collection. This study evaluates the agreement in outcome measures used in oncology for healthcare quality improvement and HTAs, and how these align to the International Consortium for Health Outcomes Measurement (ICHOM) standard sets.

We conducted a cross-sectional comparative analysis of ICHOM sets focusing on oncological indications and publicly available measures for healthcare quality and HTA reports published by the National Health Care Institute from the Netherlands and the National Institute for Health and Care Excellence from the United Kingdom.

All ICHOM sets and HTAs used overall survival, whereas quality improvement used different survival estimates. Different progression estimates for cancer were used in HTAs, ICHOM sets, and quality improvement. Data on health-related quality of life (HRQoL) was recommended in all ICHOM sets and all HTAs, but selectively for quality improvement. In HTAs, generic HRQoL questionnaires were preferred, whereas, in quality improvement and ICHOM sets, disease-specific questionnaires were recommended. Unfavorable outcomes were included in all HTAs and all ICHOM sets, but not always for quality improvement.

Although HTA and quality improvement use outcome measures from the same domains, a greater level of alignment seems possible. ICHOM may provide input on standardized outcome measures to support this alignment. However, residual discrepancies will remain due to the different objectives of HTA and quality improvement.

Consideration of ethical, legal, and social issues plus patient values (ELSI+) in health technology assessment (HTA) is challenging because of a lack of conceptual clarity and the multi-disciplinary nature of ELSI+. We used concept mapping to identify key concepts and inter-relationships in the ELSI+ domain and provide a conceptual framework for consideration of ELSI+ in HTA.

We conducted a scoping review (Medline and EMBASE, 2000–2016) to identify ELSI+ issues in the HTA literature. Items from the scoping review and an expert brainstorming session were consolidated into eighty ELSI+-related statements, which were entered into Concept Systems® Global MAX™ software. Participants (N = 38; 36 percent worked as researchers, 21 percent as academics; 42 percent self-identified as HTA experts) sorted the statements into thematic groups, and rated them on importance in making decisions about adopting technologies in Canada, from 1 (not at all important) to 5 (extremely important). We used Concept Systems® Global MAX™ software to create and analyze concept maps with four to sixteen clusters.

Our final ELSI+ map consisted of five clusters, with each cluster representing a different concept and the statements within each cluster representing the same concept. Based on the concepts, we named these clusters: patient preferences/experiences, patient quality of life/function, patient burden/harm, fairness, and organizational. The highest mean importance ratings were for the statements in the patient burden/harm (3.82) and organizational (3.92) clusters.

This study suggests an alternative approach to ELSI+, based on conceptual coherence rather than academic disciplines. This will provide a foundation for incorporating ELSI+ into HTA.