The aim of this policy article is twofold: (i) to provide a summary and update of recent important policy developments, in particular relevant guidance on the use of real-world data/real-world evidence (RWD/RWE) by health technology assessment (HTA) bodies and (ii) to set out our policy recommendations on how the different elements of an “RWE framework” we have previously developed could support, further enhance and facilitate the use of RWE for HTA purposes and by HTA bodies and payers.

We undertook a targeted review and analysis of recent important policy developments. The aim was to build on our recommendations from previous work on the “RWE Framework,” and consider how the relevant tools from our Framework can further enhance and facilitate the use of RWE for HTA purposes and by HTA bodies/payers.

We provide eight conditions that we argue would, in combination, constitute the optimal use and acceptance of RWD/RWE for HTA. We believe that, should the eight conditions hold, RWD/RWE would enable more efficient access to medicines and healthcare technologies for patients.

High-quality, fit-for-purpose RWD/RWE can and should be used more frequently in HTA. Multi-stakeholder and cross-geography collaborative partnerships are needed to align on best practices to optimize the evidence that needs to be generated to satisfy all stakeholders’ needs.

We assessed the implementation of telehealth-supported stewardship activities in acute-care units and long-term care (LTC) units in Veterans’ Administration medical centers (VAMCs).

Before-and-after, quasi-experimental implementation effectiveness study with a baseline period (2019–2020) and an intervention period (2021).

The study was conducted in 3 VAMCs without onsite infectious disease (ID) support.

The study included inpatient providers at participating sites who prescribe antibiotics.

During 2021, an ID physician met virtually 3 times per week with the stewardship pharmacist at each participating VAMC to review patients on antibiotics in acute-care units and LTC units. Real-time feedback on prescribing antibiotics was given to providers. Additional implementation strategies included stakeholder engagement, education, and quality monitoring.

The reach–effectiveness–adoption–implementation–maintenance (RE-AIM) framework was used for program evaluation. The primary outcome of effectiveness was antibiotic days of therapy (DOT) per 1,000 days present aggregated across all 3 sites. An interrupted time-series analysis was performed to compare this rate during the intervention and baseline periods. Electronic surveys, periodic reflections, and semistructured interviews were used to assess other RE-AIM outcomes.

The telehealth program reviewed 502 unique patients and made 681 recommendations to 24 providers; 77% of recommendations were accepted. After program initiation, antibiotic DOT immediately decreased in the LTC units (−30%; P < .01) without a significant immediate change in the acute-care units (+16%; P = .22); thereafter DOT remained stable in both settings. Providers generally appreciated feedback and collaborative discussions.

The implementation of our telehealth program was associated with reductions in antibiotic use in the LTC units but not in the smaller acute-care units. Overall, providers perceived the intervention as acceptable. Wider implementation of telehealth-supported stewardship activities may achieve reductions in antibiotic use.

This paper reports the results of the collaboration within the European initiative of new Reimbursement and aCCess Approaches (EUreccA) which is concerned with the use of real-world evidence (RWE) in health technology assessment (HTA) decision-making. The work grew from the observation of a large, very experienced group of HTA practitioners which found that the use of RWE varied depending on the type of question asked and the particulars of the data source(s) used. We set out to examine how RWE is used in HTA decision-making and to make proposals on its facilitation.

Literature reviews covering earlier reviews of RWE use, academic papers, and HTA agency websites were combined with case studies involving interviews with decision-makers in four countries (England, France, Italy, Sweden) to identify the circumstances of breakdown of RWE use and to build a categorization of the uses of RWE and associated difficulties. This evidence supported the creation of a taxonomy of pairings of data sources and the questions they were used to address. The face validity of the approach was tested at an advisory board of senior HTA practitioners.

In total, 27 questions were identified and 10 types of data source, giving 270 pairings. These pairings were linked to relevant methods guidance and to examples of their use, itemizing HTA issues and decisions made. Reports are being prepared for publication, covering the detail of the methods of the literature searches; methods of the country case studies; a description of the taxonomy; and guidance on governance.

When using RWE in HTA decision-making, the detail of the particular data sources and question addressed matter. Recently, both the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and the Real-World Transparency Initiative have argued for a registry of the uses of RWE. The work described here offers a starting classification of the material that should be held in such a registry, and which in itself could be developed by the stakeholders, both agencies and companies, that use it, furthering trust and confidence.

Antipsychotic depot injections can improve adherence compared to tablets. However, depot prescribing practices differ amongst psychiatrists. Previously, some clinicians perceived an “image” problem for typical antipsychotic depots. This study investigated psychiatrists’ attitudes and knowledge concerning antipsychotic depots (typical and atypical) in an era when patient choice is a pertinent issue.

Cross-sectional postal survey of consultant psychiatrists working in NorthWest England. A pre-existing questionnaire on clinicians’ attitudes and knowledge regarding depots was updated.

The sample comprised 102 consultant psychiatrists (response rate 102/143, 71%). Their use of depots over the past 5 years had: decreased (50%), not changed (27%), increased (23%). In a forced-choice selection of factors that would persuade them to use depots more, the factor cited as most important was ‘having more atypicals available in long-acting depot form’ (43%). Most regarded depots as being associated with better compliance (89%) and reduced relapse rates (98%) compared to oral medication but only 62% agreed that depots can be used for those with first episode psychosis. A significant minority (33%) believed patients always prefer to have oral medication instead of a depot. 68% believed that patients taking medication of their own free choice is more likely for oral than depot.

During the last 5 years, overall depot prescribing rates have reduced. Most regarded depots as offering better adherence and reduced relapse rates but some remain concerned about the acceptability of depots to patients. These clinician concerns are important but, if extreme, could compromise medication choices offered to patients.

Previously, when only typical antipsychotic depot injections were available, some clinicians perceived depots as having an “image” problem despite them being associated with reduced rates of rehospitalisation when compared to tablets. This study investigated psychiatrists’ attitudes and knowledge concerning depots (typical and atypical) and whether they had changed over time.

Cross-sectional postal survey of consultant psychiatrists working in NorthWest England. A pre-existing questionnaire on clinicians’ attitudes and knowledge regarding depots was updated. Results were compared with a former sample (SouthEast England, 2001: N=143).

The sample comprised 102 consultant psychiatrists (response rate 71%). Depot use over the past 5 years had: decreased (50%), not changed (27%), increased (23%). Psychiatrists with decreased depot use had significantly lower scores for the side effects knowledge subscale than those who had unchanged or increased rates of depot use (mean 51.5% vs 54.8%, p=0.029). When compared to psychiatrists sampled five years previously, our current participants had more favourable patient-focussed attitudes (63.5% vs 60.4%, p=0.034); other subscales did not differ. Item-by-item analysis revealed specific changes over time including significantly less respondents regarding depots as: (i) compromising patient autonomy (mean 0.99 vs 1.28, p=0.036); being stigmatising (1.88 vs 2.42, p=0.002); being old fashioned (1.49 vs 2.04, p=0.002).

During the period that an atypical antipsychotic depot has been available, and depot prescribing rates have reduced, some attitudes have changed. These mainly encompass aspects regarding the patient rather than the depot injection and include reducing concerns about stigma and autonomy although concerns about patient acceptance continue.

D.C.O. is psychiatric commissioner on the Commission on Human Medicines and a member of the European Medicines Agency's Scientific Advisory Group on Psychiatry. He chaired the European Medicines Agency's review of the psychiatric use of valproate in pregnancy and women of childbearing potential.