The 9th European Medical Writers Association (EMWA) symposium, entitled ‘Clinical Trials – New Trends’ took place on 6 May. The symposium explored the latest trends in the design, conduct and reporting of clinical trials over 3 sessions, focusing on insights relevant to medical writers and communicators.
A summary of session 1 of the symposium is provided below to benefit those who were unable to attend, and as a timely reminder of the key topics for those who did.
Session 1: general overview
Session 1, chaired by Beatrix Doerr (Coriuvar Consulting), set out to provide an overview of new trends in clinical trials.
A medical writer’s guide to scientific evidence types: it’s child’s play!
Simon Page (Ipsen) opened the session with a guide to the different types of studies and their position in the ‘hierarchy of evidence’. Reviewing the characteristics of outputs from systematic reviews/meta-analyses, randomised controlled trials (RCTs), observational studies, case reports and basic research, Page highlighted how evidence gained from each type of study provides complementary insights. While systematic reviews and meta-analyses top the hierarchy by providing the highest quality of evidence, they rely on the studies lower in the hierarchy to contribute evidence to amalgamate. RCTs definitively test efficacy under closely controlled conditions, but observational studies can provide insights on effectiveness in real world settings. When it comes to reporting study results, Page highlighted the guidelines to be followed for each study type, which can all be found on the EQUATOR Network website.
New trends in medical device trials: from basket to umbrella – implementing learnings from pharma trials
Autumn Lang (confinis CPM) then considered developments related to trials of medical devices, focusing on study design strategy, risk assessment and cross-communication. ‘Basket’ and ‘umbrella’ study designs are among the innovations being used in the assessment of medical devices, drawing on experience in fields such as oncology. A ‘basket’ design allows the same device to be tested in multiple groups of patients with different diseases, while in an ‘umbrella’ study, multiple devices are tested in a single patient population. Adaptive approaches are also being used, where the study design can be modified based on interim results. Lang also noted the growing drive for medical device studies to provide evidence not only of technical performance (for example, sensitivity, specificity and precision of a diagnostic device) but also of clinical utility and the extent to which health outcomes are improved.
Lang also noted the growing drive for medical device studies to provide evidence not only of technical performance…but also of clinical utility and the extent to which health outcomes are improved.
Risk assessments are an important consideration when trialling medical devices, although, currently, no clear guidelines exist. Lang rounded off her presentation by highlighting the valuable role medical writing professionals can play in communicating the results of clinical evaluations of medical devices, specifically with reviewing and evaluating the clinical literature to allow comparison with competitor devices.
New trends in in vitro diagnostic trials: case example COVID-19 tests
Turning to trials of in vitro diagnostics, Holger Kloess (QUNIQUE) took the evaluation of COVID-19 antigen tests as a case study. While these tests gained registration based on evaluations conducted by individuals with expertise in processing the tests, and were originally developed for use by nurses and other healthcare professionals, they are now being handed out for use by the general public. This raises questions around the extent to which the performance demonstrated in registration studies is reproducible when testing is carried out by untrained individuals. It also highlights the need for real world evidence on top of that from highly controlled studies.
Kloess then considered the evolving regulatory landscape in in vitro diagnostics. Diagnostic assays are increasingly incorporated into devices, many linked to mobile phone apps – these and the associated control software all require assessment. While 9% of in vitro diagnostics are currently registered as devices, this is expected to rise dramatically. These devices will need to be assessed according to the recently updated ISO 14155 guidelines, which require application of Good Clinical Practice in device trials, robust trial designs, pre-defined risk tolerance limits and clinical safety thresholds, and full quality control.
New trends in veterinary medicine trials
In the penultimate talk of the session, Rachel Dean (VetPartners) reviewed developments in animal medicine and the particular challenges of running, reporting, and researching veterinary clinical trials. While the methodological basis of an RCT is the same regardless of whether it is conducted in humans or animals, veterinary trials have the added complication of covering a wide range of species and settings, ranging from pets in the home to animals in the wild. At the same time, many of the challenges in veterinary trials are seen across species, and include:
- studies not addressing the right questions
- poor design and execution of studies
- studies not being reported, or being reported poorly
- lack of access to, or awareness of, studies.
The majority (~70%) of veterinary trials are currently run by the pharmaceutical industry for licensing purposes…end users are rarely consulted, or their needs addressed.
Dean highlighted that the majority (~70%) of veterinary trials are currently run by the pharmaceutical industry for licensing purposes, and that end users are rarely consulted, or their needs addressed. Trials are often too small and lack clearly defined, appropriate outcomes. There is no registry for veterinary trials and negative studies are often not published, leading to a positive bias in available results. When studies are published, they often sit behind journal paywalls and, with the exception of CAB Abstracts, most bibliographic databases cover only around half of the journals relevant to veterinary medicine.
There is no registry for veterinary trials and negative studies are often not published, leading to a positive bias in available results.
Looking to the future, Dean hopes that trials will be more reflective of the real world and the needs of end users rather than those of the pharmaceutical industry and academia. Trial designs should be bolder, bigger and simpler, with trials carried out in real life settings. Greater use should be made of electronic patient records and all veterinary trials should be registered, although progress on this objective has been limited to date. Greater involvement of professional medical writers was discussed as making a positive contribution to the quality of reporting of veterinary studies.
Patient perspective on new trends in clinical trials: think about patient value, not provider value!
The take-home message from the last presentation in the session by Jan Geissler (Patvocates) was that studies need to focus on the value for patients, not providers. Developments in digital technology are opening up a range of opportunities to design studies that are more aligned with the needs of patients than researchers.
Developments in digital technology are opening up a range of opportunities to design studies that are more aligned with the needs of patients than researchers.
It is increasingly feasible to move care and research to where the patient is, rather than requiring the patient to travel to the research centre. Remote monitoring and mobile apps can provide greater support, faster access to test results and improved safety. At the same time, it is important to consider the downsides. The shift to remote interactions runs the risk of leaving individuals isolated: people can be left cut off from care if they fall on the wrong side of the digital and educational divide. Finally, there are complex ethical issues on privacy and data ownership that need to be addressed.
When designing studies for the digital, decentralised era, it is essential to engage patients as co-designers of trials. Geissler considered that, at present, patients are often engaged as ‘beta testers’ rather than designers, with studies being developed in their interests but without their input. Studies need to reflect the fact that not all patients are the same, and the assessment of risks and benefits needs to account for variations in patient characteristics and preferences. Medical writers have an important role to play in reporting trial results in ways that are meaningful and relevant to patients.
When designing studies for the digital, decentralised era, it is essential to engage patients as co-designers of trials.
The additional costs involved in engaging patients early in the study design process were raised, and discussions noted that it was important to consider the cost impact across the study lifecycle. Investing in patient engagement has the potential to enhance the design of studies and improve patient retention, thus saving costs down the line, as well as aligning with the principle of ‘No research about us without us’.
Written as part of a Media Partnership between EMWA and The Publication Plan.
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