Highlights
Task:
QUESTION 1
For patients with stomach cancer that can be removed by surgery, a randomised controlled trial (RCT) was designed to evaluate whether having chemotherapy after surgery is more effective than the standard practice of not giving chemotherapy. It enrolled patients who had recovered sufficiently after surgery. The RCT will report overall survival (the primary outcome) and quality of life. It will follow patients for 3 years after treatment. It is decided to run the trial with patients and treating clinicians knowing the treatment allocation after randomisation (chemotherapy vs placebo) so they could respond to potential adverse events. The analysis would be done on an intention-to-treat basis.
a) Write out the PICO that describes this randomised controlled trial.
(4 marks)
b) What are two benefits of selecting a disease-specific quality of life instrument (one designed for cancer patients) rather than a generic instrument?
(4 marks)
c) Briefly explain how not blinding (masking) the treatment to patients might affect the accuracy of the trial results.
(8 marks)
d) Suppose the trial reported improved survival among patients who received chemotherapy, and there were no serious concerns about the trial methods. What features of the trial would a doctor need to know in order to apply the results to their patients?
(9 marks)
QUESTION 2
One way that a hospital monitors the quality of care in its maternity unit is by asking women about the care they received during the birth of their baby (ie, the process of care). For the past 5 years, the hospital has measured their experience of care by giving a questionnaire to women as they leave the hospital, which the women post back to the hospital audit department. The hospital has asked you to review their survey process.
a) The survey questions ask if the hospital environment was clean and comfortable, and whether women felt their privacy had been respected. Name one other aspect of patient experience that could be included in a redesigned questionnaire, and give the reason for including it.
(3 marks)
b) Some hospital staff suggest that the survey is a waste of money and that women could use the standard complaint process if they were unhappy with the care they received. Briefly explain why data collected by an internal hospital complaint system is less useful than data from a well-run survey for improving the quality of care.
(6 marks)
c) Other staff suggest giving out the survey to women to complete in the post-natal ward rather than posting the survey to women after they are discharged. Give two reasons why collecting data from women while they are in hospital might lead to biased results.
(6 marks)
d) The Head of Midwifery suggests using a focus group to explore the experiences among women who give birth in the maternity unit. Describe how you would prepare for and run a focus group to measure their patient experience. Include in your answer one difficulty posed by adopting this new approach and how your approach could overcome it.
QUESTION 3
ACE inhibitors are drugs that relax blood vessels and improve blood flow. They are mainly used to treat high blood pressure (hypertension) and heart failure. A multi-group ecological study has found that, between regions of a country, there are large differences in the use of ACE inhibitors, per 1000 residents, among people aged over 65 years. This was a surprise because there are clear treatment guidelines for hypertension and heart failure.
a) To become an evaluation of equity in health care, the study will be extended to measure “need for health care”. Briefly describe the concept of “need for health care”.
(5 marks)
b) Suggest an appropriate measure of need for this evaluation and describe how the study could obtain these data for each region.
(8 marks)
c) What are two reasons that might account for the variation in regional rates of ACE inhibitor use after accounting for clinical need?
(4 marks)
d) Two years ago, one region ran a campaign aimed at doctors to promote adherence to the treatment guidelines on ACE inhibitors. As weekly data were available, a single time-series study was used to assess the impact of the campaign on the use of ACE inhibitors. Why might it be difficult to interpret the results from this study as evidence of the campaign’s effectiveness? Propose a way of improving the time-series study so that the results are easier to interpret.
(8 marks)
QUESTION 4
Venous leg ulcers are a common type of skin ulcer affecting older people. Patients experience pain which affects their mobility and quality of life. To treat the ulcer, it is cleaned and dressed using a compression bandage (standard care). A nurse will do this every week at the patient’s home, and this results in 70% of venous ulcers healing within 12 weeks. However, ulcers can recur and healing may take up to five years.
A randomised controlled trial (RCT) was run to compare clinic-based therapeutic ultrasound in addition to standard care, versus the standard care alone. Patients allocated to the control group received placebo (sham) ultrasound. The primary outcome measure was patient- reported quality of life measured at 3-month intervals for 1 year.
a) An economic evaluation was conducted using data from the trial. Give four examples of health service resources that might be costed in this economic evaluation.
(4 marks)
b) For each of the examples named in part (a), describe the data required by the evaluation to calculate the costs associated with the resources required to treat patients during the trial and follow-up period.
(8 marks)
c) Describe two limitations of RCTs that might result in an economic evaluation using a decision model rather than relying only on the cost and outcome data from this trial.
(4 marks)
d) Give three reasons why it is legitimate for two economic evaluations to come up with different cost-effectiveness ratios even if they use the outcome data from the same trial.
(9 marks)
4
QUESTION 5
Researchers want to evaluate the effectiveness of hip replacement surgery in older people with arthritis. They propose comparing two current methods of surgery: (1) the posterior approach and (2) the direct lateral approach. The posterior approach is easy to perform, but increased rates of dislocation have been reported. The direct lateral approach facilitates the positioning of the replacement hip, which may decrease rates of hip dislocation, but it is a more challenging operation for the surgeon and the patient has an increased risk of a limp.
a) The researchers propose to run the evaluation as a multi-centre randomised controlled trial. Why might the researchers use a randomisation process stratified by centre?
(3 marks)
b) Give two reasons why the surgical operations can be regarded as moderately complex health care interventions. Describe one way that the researchers could reduce the risk of the study’s internal validity being affected by the complexity of the interventions.
(8 marks)
c) Give two reasons why it might prove to be impossible to undertake this evaluation as a randomised controlled trial.
(6 marks)
d) The evaluation is finally run as a prospective cohort study, with patients being asked to participate after they are assessed as appropriate for a hip replacement. The primary outcome is patient-reported physical mobility, which is measured at baseline, 6 and 12 months after surgery. Give two potential sources of bias related to the measurement of this outcome, and for each source, describe what step you could take to limit its impact.
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