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Abstract

BACKGROUND

Randomised controlled trials (RCTs) are widely accepted as the best way to assess the outcomes and safety of medical interventions, but are sometimes not ethical, not feasible, or limited in the generalisability of their results. In such circumstances, routinely available data could help in several ways. Routine data could be used, for example, to conduct 'pseudo-trials', to estimate likely outcomes and required sample size to help design and conduct trials, or to examine whether the expected outcomes observed in an RCT will be realised in the general population.

OBJECTIVES

The project was undertaken to explore how routinely assembled hospital data might complement or supplement RCTs to evaluate medical interventions: in contexts where RCTs are not feasible for defining the context and design of an RCT for assessing whether the benefits indicated by RCTs are achieved in wider clinical practice.

METHODS

The project was based on the system of linked Scottish morbidity records, which cover 100% of acute hospital care episodes and statutory death records from 1981 to 1995. Three case studies were undertaken as a way of investigating the utility of these records in different applications. First, an attempt was made to analyse the link between the timing of surgery for subarachnoid haemorrhage (SAH) and subsequent outcomes (a question not easily susceptible to RCT design). A subsample was derived by excluding patients for which a diagnosis of SAH may not have been established or that may not have been admitted to a neurosurgical unit, and the data were assessed to attempt to inform the design of a trial of early versus late surgery. Transurethral prostatectomy (TURP), the second case study, has become the surgery of choice for benign prostatic hyperplasia without systematic assessment of its effectiveness and safety, and an RCT would now be considered unethical. However, there is a need to investigate long-term effects and the influence of co-morbidities on outcomes. A retrospective comparison of mortality and re-operation following either open prostatectomy (OPEN) or TURP was, therefore, undertaken. Patients for whom it was not possible to establish the initial procedure were excluded. The third case study compared coronary artery bypass grafting (CABG) with percutaneous transluminal angioplasty (PTCA) for coronary revascularisation. RCTs have been conducted in limited patient subgroups with short follow-up periods. A meta-analysis of RCTs could be augmented by routine data, which are available for large populations. This would allow assessment of subgroup effects, and outcomes over a long period. A subgroup of patients was therefore constructed for whom relevant routine data were available and who reflected the entry criteria for major RCTs, thus enabling a comparison between the results expected from this subgroup and those of the general population.

RESULTS AND CONCLUSIONS

The uses of routine data in these contexts had strengths and weaknesses. The SAH study suggested a means of assessing outcomes and survival rates following haemorrhage, which could have value in informing the design of more precise trials and in evaluating changes in outcome following the introduction of new treatments such as embolisation. However, the potential of the data was not realised because their scope and content were insufficient. For example, lack of data on the time of onset of symptoms and patients' conditions at hospital admission made it difficult to establish the link between timing of surgery and the outcome, and there was insufficient information on patients' conditions at discharge to enable a comparison of outcomes. The prostatectomy study was able to address questions not answered by RCT literature because the large number of cases it included allowed exploration of subgroup effects. (ABSTRACT TRUNCATED)

Abstract

BACKGROUND

Randomised controlled trials (RCTs) are widely accepted as the best way to assess the outcomes and safety of medical interventions, but are sometimes not ethical, not feasible, or limited in the generalisability of their results. In such circumstances, routinely available data could help in several ways. Routine data could be used, for example, to conduct 'pseudo-trials', to estimate likely outcomes and required sample size to help design and conduct trials, or to examine whether the expected outcomes observed in an RCT will be realised in the general population.

OBJECTIVES

The project was undertaken to explore how routinely assembled hospital data might complement or supplement RCTs to evaluate medical interventions: in contexts where RCTs are not feasible for defining the context and design of an RCT for assessing whether the benefits indicated by RCTs are achieved in wider clinical practice.

METHODS

The project was based on the system of linked Scottish morbidity records, which cover 100% of acute hospital care episodes and statutory death records from 1981 to 1995. Three case studies were undertaken as a way of investigating the utility of these records in different applications. First, an attempt was made to analyse the link between the timing of surgery for subarachnoid haemorrhage (SAH) and subsequent outcomes (a question not easily susceptible to RCT design). A subsample was derived by excluding patients for which a diagnosis of SAH may not have been established or that may not have been admitted to a neurosurgical unit, and the data were assessed to attempt to inform the design of a trial of early versus late surgery. Transurethral prostatectomy (TURP), the second case study, has become the surgery of choice for benign prostatic hyperplasia without systematic assessment of its effectiveness and safety, and an RCT would now be considered unethical. However, there is a need to investigate long-term effects and the influence of co-morbidities on outcomes. A retrospective comparison of mortality and re-operation following either open prostatectomy (OPEN) or TURP was, therefore, undertaken. Patients for whom it was not possible to establish the initial procedure were excluded. The third case study compared coronary artery bypass grafting (CABG) with percutaneous transluminal angioplasty (PTCA) for coronary revascularisation. RCTs have been conducted in limited patient subgroups with short follow-up periods. A meta-analysis of RCTs could be augmented by routine data, which are available for large populations. This would allow assessment of subgroup effects, and outcomes over a long period. A subgroup of patients was therefore constructed for whom relevant routine data were available and who reflected the entry criteria for major RCTs, thus enabling a comparison between the results expected from this subgroup and those of the general population.

RESULTS AND CONCLUSIONS

The uses of routine data in these contexts had strengths and weaknesses. The SAH study suggested a means of assessing outcomes and survival rates following haemorrhage, which could have value in informing the design of more precise trials and in evaluating changes in outcome following the introduction of new treatments such as embolisation. However, the potential of the data was not realised because their scope and content were insufficient. For example, lack of data on the time of onset of symptoms and patients' conditions at hospital admission made it difficult to establish the link between timing of surgery and the outcome, and there was insufficient information on patients' conditions at discharge to enable a comparison of outcomes. The prostatectomy study was able to address questions not answered by RCT literature because the large number of cases it included allowed exploration of subgroup effects. (ABSTRACT TRUNCATED)

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