Dataset Information

Changing platforms without stopping the train: experiences of data management and data management systems when adapting platform protocols by adding and closing comparisons.

ABSTRACT: BACKGROUND:There is limited research and literature on the data management challenges encountered in multi-arm, multi-stage platform and umbrella protocols. These trial designs allow both (1) seamless addition of new research comparisons and (2) early stopping of accrual to individual comparisons that do not show sufficient activity. FOCUS4 (colorectal cancer) and STAMPEDE (prostate cancer), run from the Medical Research Council Clinical Trials Unit (CTU) at UCL, are two leading UK examples of clinical trials implementing adaptive platform protocol designs. To date, STAMPEDE has added five new research comparisons, closed two research comparisons following pre-planned interim analysis (lack of benefit), adapted the control arm following results from STAMPEDE and other relevant trials, and completed recruitment to six research comparisons. FOCUS4 has closed one research comparison following pre-planned interim analysis (lack of benefit) and added one new research comparison, with a number of further comparisons in the pipeline. We share our experiences from the operational aspects of running these adaptive trials, focusing on data management. METHODS:We held discussion groups with STAMPEDE and FOCUS4 CTU data management staff to identify data management challenges specific to adaptive platform protocols. We collated data on a number of case report form (CRF) changes, database amendments and database growth since each trial began. DISCUSSION:We found similar adaptive protocol-specific challenges in both trials. Adding comparisons to and removing them from open trials provides extra layers of complexity to CRF and database development. At the start of an adaptive trial, CRFs and databases must be designed to be flexible and scalable in order to cope with the continuous changes, ensuring future data requirements are considered where possible. When adding or stopping a comparison, the challenge is to incorporate new data requirements while ensuring data collection within ongoing comparisons is unaffected. Some changes may apply to all comparisons; others may be comparison-specific or applicable only to patients recruited during a specific time period. We discuss the advantages and disadvantages of the different approaches to CRF and database design we implemented in these trials, particularly in relation to use and maintenance of generic versus comparison-specific CRFs and databases. The work required to add or remove a comparison, including the development and testing of changes, updating of documentation, and training of sites, must be undertaken alongside data management of ongoing comparisons. Adequate resource is required for these competing data management tasks, especially in trials with long follow-up. A plan is needed for regular and pre-analysis data cleaning for multiple comparisons that could recruit at different rates and periods of time. Data-cleaning activities may need to be split and prioritised, especially if analyses for different comparisons overlap in time. CONCLUSIONS:Adaptive trials offer an efficient model to run randomised controlled trials, but setting up and conducting the data management activities in these trials can be operationally challenging. Trialists and funders must plan for scalability in data collection and the resource required to cope with additional competing data management tasks.

SUBMITTER: Hague D

PROVIDER: S-EPMC6540437 | biostudies-literature | 2019 May

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Changing platforms without stopping the train: experiences of data management and data management systems when adapting platform protocols by adding and closing comparisons.

Hague Dominic D Townsend Stephen S Masters Lindsey L Rauchenberger Mary M Van Looy Nadine N Diaz-Montana Carlos C Gannon Melissa M James Nicholas N Maughan Tim T Parmar Mahesh K B MKB Brown Louise L Sydes Matthew R MR

Trials 20190529 1

<h4>Background</h4>There is limited research and literature on the data management challenges encountered in multi-arm, multi-stage platform and umbrella protocols. These trial designs allow both (1) seamless addition of new research comparisons and (2) early stopping of accrual to individual comparisons that do not show sufficient activity. FOCUS4 (colorectal cancer) and STAMPEDE (prostate cancer), run from the Medical Research Council Clinical Trials Unit (CTU) at UCL, are two leading UK examp ...[more]

PMID: 31138292

Similar Datasets

Project description:Background:Each year, an estimated 17 million children suffer from severe acute malnutrition (SAM) and 33 million from moderate acute malnutrition (MAM), with many of the most severe cases found in extremely food insecure contexts or conflict situations. Current global outpatient treatment protocols for uncomplicated SAM and MAM, adapted by most countries for use at national level, call for SAM and MAM to be managed separately, however global-level stakeholders have recently begun evaluating simplified and/or combined protocols managing acute malnutrition. Methods:This study analyzes national policy discussions and decision-making around outpatient acute malnutrition treatment for uncomplicated cases in emergency situations in Niger, Nigeria, Somalia, and South Sudan. Data collection (March-July 2018) included semi-structured in-depth interviews with 50 respondents (N?=?11-15 per country) from government, funding agencies, and implementing partners, as well as 11 global and regional stakeholders. We also conducted a document analysis (N?=?10-15 per country and at global level) to situate debates and evaluate current policy. Data were analyzed iteratively using thematic content analysis. Results:We find that while combined/simplified protocols for outpatient management of uncomplicated cases of acute malnutrition are being used in emergency situations in all four countries, there is widespread confusion about protocol terminology and content, stemming from a lack of coherence at the global level. As a result, national-level stakeholders express diverse, if overlapping, rationales for modifying current protocols, which vary given the intensity and scope of the emergency. Without specific global-level guidance, combined/simplified protocols are often used on an ad hoc basis, although the processes for triggering them were at least nominally controlled at the national level. Decisions about when and where to enact "exceptional" modifications to country protocols were often based on inconsistent determinations of what constitutes an "emergency." Respondents said more evidence is needed on both clinical and operational aspects of these protocols, and they awaited clear guidance from global norm-setting agencies. Conclusions:Based on these findings, global-level stakeholders should urgently improve coordination and communication around existing protocols. Standardized guidance based on the available evidence is required to clarify best practices for combined management of SAM and MAM, particularly in emergency contexts (which should be defined) and in situations of limited resources. Given the complexity of governance arrangements in conflict situations, both guidance and updates on research must be disseminated in a rational, systematic, and digestible way to the multiplicity of field actors.

Dataset Information

Changing platforms without stopping the train: experiences of data management and data management systems when adapting platform protocols by adding and closing comparisons.

Publications

Changing platforms without stopping the train: experiences of data management and data management systems when adapting platform protocols by adding and closing comparisons.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets