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We’re well on into 2022, but it’s worth looking back to the last Value in Health issue of 2021. It’s a bumper issue with 18 studies!
On patient-reported outcomes, the issue starts with a study about the quality of their reporting. The authors reviewed 631 RCTs of cancer therapies between 2004-2019, concluding that reporting has improved over time. In another study, Katharine Gries and colleagues report the development and content validity of a new instrument to measure symptoms and impacts experienced by patients with active multiple myeloma, called the MySIm-Q instrument. Additionally, there is a study about how frequently patient-reported outcomes are included in trials of cancer treatments in adolescents and young adults (spoiler: not very much; e.g., only around 21% of therapeutic phase 3 trials).
There are a few studies on patient preferences and quality of life. One is on patient preferences for anxiety and depression screening in cancer care, via a discrete choice experiment to investigate which are the aspects of an anxiety and depression screening program that cancer patients value most. Another study reports the development of a Hungarian tariff set for the ICECAP-A instrument and compares it with the UK tariff. One study looked at ways to address missing EQ-5D-5L data; Suja S. Rajan and colleagues investigated the possibility of asking individuals to fill in questionnaires about their quality of life in the past. The good news is that this approach seems to work, and the quality of life reported by individuals about their past selves is similar to what they had reported in the past. Lastly, there is a study about the psychometric properties of EQ-5D-5L-Y for children and adolescents, and how they compare with the EQ-5D-3L-Y.
There are a few economic evaluation studies. One which caught my eye was Koen Degeling and colleagues’ study on the management of favourable-risk localised prostate cancer. They estimated the health benefits and costs of active surveillance, radical prostatectomy, and radiation therapy from the perspective of the Australian healthcare service. They developed a new discrete event simulation model, informed by risk equations estimated from reconstructed individual-level data from the ProtecT trial (a trial that compared the three management options over a median follow-up of 10 years). They found that choosing active surveillance results in fewer health benefits. This is because 60% of patients on active surveillance eventually receive radical treatment. Also because patients who initially chose active surveillance are at higher risk of clinical progression and metastatic disease than patients who chose immediate radical treatment. It’s a double whammy because active surveillance also results in higher costs. As the authors discuss, the value of active surveillance may not be adequately captured by QALYs from the patient perspective, given the risks of radical treatment. This impressive study, together with the other studies based on the ProtecT trial, are relevant in informing clinical guidelines on the management of prostate cancer.
Keeping with the prostate cancer theme, I couldn’t help but read the interesting cost-effectiveness analysis of prostate cancer screening by Shuang Hao and colleagues. They compared five screening strategies comprising combinations of systematic biopsy, targeted biopsy, and magnetic resonance imaging for early prostate cancer detection in Sweden. It was a model-based analysis, using a pre-existing microsimulation model calibrated to Swedish data. In the model, men were at risk of pre-clinical prostate cancer, which could progress to higher T stages pre-clinically and which could be clinically detected. The authors found that all screening strategies resulted in greater detection of prostate cancer but lower prostate cancer mortality, with the quadrennial screening strategy using magnetic resonance imaging and targeted biopsy being the optimal choice in terms of quality-adjusted life expectancy and costs.
Woojung Lee and colleagues report an excellent study about real option value. As the authors defined, real option value is created when a drug enables a patient to live long enough to benefit from a future innovation. They developed a method to calculate the real option value from ipilimumab for metastatic melanoma in creating the opportunity for patients to have subsequent cancer immunotherapy. To estimate individuals’ life expectancy and take-up of subsequent cancer immunotherapy, the authors used a US database derived from electronic health records (the Flatiron Health database). They found that, when considering the real option value of ipilimumab in terms of the benefits of effective subsequent cancer immunotherapy, the health gained is much greater. The increase depends on the uptake of the subsequent cancer immunotherapy, which in turn depends on the time from when these therapies became available. For example, the QALY gain increased by 53% for those who started first-line treatment when the therapies became available, compared to 1% greater in those who started first-line treatment 24 months before. As far as I’m aware, real option value is not talked about much, but I expect that we’ll see more research about this in the future.
This really is a superb issue, full of interesting studies which I’m unable to cover in full. In addition to the studies discussed above, there is a cost-effectiveness analysis of computed tomography for the diagnosis of hip fractures; a review about how to define and value innovation in health technology assessment; an analysis of the recommendations of the French National Health Authority on the reimbursement and pricing of innovative drugs; a study investigating the use of real-world evidence in the evaluation of medical devices in the US; a study on low-value care, specifically which low-value practices and how often these were used in the context of trauma injuries; a review and meta-analysis of interventions to support informal caregivers of people with dementia; a review of methods to value informal care provided to people with dementia; and a discussion about health technology assessment of antimicrobials.