METHODS

Standard and new designs for mass balance studies are collected from literature and combined experience available at ICON plc. Rules and regulations that apply to studies with radiolabelled drugs, limitations in study design, and their impact on the MW processes are described. Challenges for MW are identified based on an informal survey on experiences and challenges with mass balance studies among ICON’s early phase MWs.

RESULTS

A variety of study designs is currently used for mass balance studies. Variation is found in labels used, amount of radioactivity administered, excreta collected, duration of studies, dosing sequences, and populations used. A clear description of the procedures followed, the objectives of the study design, and the results obtained is challenging for clinical documents including subject-facing documents.

 

CONCLUSIONS

Mass balance studies are an exciting type of studies with specific challenges for MW. New developments give rise to more opportunities to collect data in wider populations and with lower exposure to radioactive materials. Due to the increasing complexity of these studies, explaining the followed approach in a protocol, clinical study report, or subject-facing document requires more guidance for MW.

METHODS

PubMed literature searches were conducted between 1/06/2015 and 24/08/2022 for RCTs that included the terms ‘Crohn disease’, ‘ulcerative colitis’ or ‘inflammatory bowel disease’. Publications were screened using artificial intelligence to include primary analyses of phase 2–4 pharmaceutical interventional RCTs only. RCT interventions and outcomes were assessed using the ML model Generative Pre-trained Transformer 3. Journal affiliation, impact factor (IF) and RCT phase were assessed manually.

RESULTS

The search returned 1038 publications, of which 138 were RCTs for pharmaceutical interventions (in 35 unique journals). The majority of RCTs (83/138 [60.1%]) were published by society-affiliated journals (n=19). Overall, 30 RCTs reported negative results, of which most (22/30 [73.3%]) were published by society-affiliated journals, and nearly half (13/30 [43.3%]) by two journals (IF, 10.0 [n=7]; IF, 22.7 [n=6]). Most RCTs published in non-society-affiliated journals reported positive results (45/55 [81.8%]; 13 journals); all except for one non-society-affiliated journal published a single negative RCT. Of the 30 negative RCT articles, 63.3% were open access; 14, 10 and 6 publications reported phase 2, 3 and 4 RCTs, respectively.

 

CONCLUSIONS

The results of this exploratory analysis suggest that society-affiliated IBD journals publish more RCTs, including negative RCTs, than non-society-affiliated/general medicine journals. This may have implications for choice of target journal for such data. As only IBD RCTs were assessed, the significance of these findings for other therapy areas is unknown.

METHODS

Analyses were made using claims data of statutory health insurance providers from the German Pharmacoepidemiological Research Database (GePaRD), which covers about 20% of Germany’s general population. Statistical Analysis System (SAS) was the primary tool for data analysis.

RESULTS

The year 2016 had the highest incidence rate and prevalence proportion of 3,60 and 27,17 per 100,000 persons respectively. The age cohort 30-39 almost consistently had the highest prevalence proportions annually, especially year 2016, which was 3,67 per 10,000 persons. This was significantly higher among males belonging to this age cohort, compared to their female counterparts.

 

CONCLUSIONS

About 20%-40% of the population of some sickle cell endemic regions in West and Central Africa have Sickle Cell Trait. Until now, there are some modest increases in the prevalence of sickle cell in Germany, compared to the very high prevalence in West and Central Africa. Recently, universal new born screening for SCD, has been introduced, which will help to identify SCD/SCT early on, and increase awareness of the disease in the light of ongoing migration movements from endemic regions. This is one of the first studies on sickle cell in Germany using claims data. Results reveal dynamic epidemiologic developments, especially after year 2015.

METHODS

GPP 2022 guidelines were compared to GPP3. Search terms including “medical writer”, “writer”, and “writing” were used to extract changes relevant to MWs.

RESULTS

GPP 2022 has expanded the term “publications” to include plain language summaries and enhanced content such as videos, audios, and infographics. Key updates relevant to MWs producing publications include: (a) a statement that MWs enhance the quality of publications; (b) authors must agree to work with a MW before the start of the project, provide direction and initial outline to the MW, and perform the final data check to ensure the quality and accuracy of the publication; (c) a MW may be listed as an author if they fulfil all the ICMJE criteria and is not disqualified from authorship by paid employment as a MW; (d) publications should include the MW’s name, professional qualifications, affiliation, and funding source; (e) MWs should not use their personal social media accounts to share information on company-sponsored research publications; (f) MWs should engage in formal training offered by relevant organisations (e.g., EMWA, AMWA, and ISMPP).

CONCLUSIONS

GPP 2022 provides important clarifications on the transparency, recognition, roles, and responsibilities of MWs. MWs should be aware of these updates and communicate them to their clients and collaborators.