Journal round-up: PharmacoEconomics – Open 5(1)

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The issue starts with a commentary by Antony Andrews on the measurement of efficiency of healthcare providers. It makes the important point that healthcare providers cannot operate with maximum efficiency due to the difficulty in responding to changes in demand. After all, healthcare is not like making widgets! The implication is that this excess capacity should be considered when healthcare providers are benchmarked against each other.

If you are into costs, this is the issue for you! There is a study on costs related to the productivity losses of patients with multiple sclerosis in the US, on the costs (and healthcare use) of COPD in Malaysia, on the costs (and healthcare use) of falls and fractures in people with overactive bladder in the US, costs of hospitalisations for respiratory syncytial virus in Colombia, costs (and healthcare use) of patients with porphyria in the US, costs (and hospital length of stay) of neonatal bloodstream infection in Ghana, and costs (and overall survival) of patients with metastatic non-small cell lung cancer in The Netherlands. In addition, the issue includes a study on the hospital length of stay for patients with severe COVID, and on naming a new patient-reported outcome instrument for chronic weight management.

One article that sparked my interest was Melissa H. Roberts and Gary T. Ferguson’s article on payers’ views about real-world evidence and how it can be used to inform decisions on market entry and reimbursement. The paper discusses the role that real-world evidence could have in policy decisions about new drugs, and references some empirical studies, mostly in the area of anticoagulation and COPD. While some of these areas are broadly consensual (e.g. safety monitoring, estimating the risk of hospital admissions with routine care, etc.), others are less so. For example, if the RCT did not compare the new drug to the relevant active comparator, I’d say that the first step would be to investigate if a network meta-analysis is feasible rather than starting with an analysis of real-world evidence to compare the new drug to an active comparator. I agree with the paper that real-world evidence has a lot of potential for HTA, but, as always, the devil is in the detail!

If you’re interested in real-world evidence for HTA, a new organisation is launching on the topic – the GetReal Institute. It is a not-for-profit organisation involving pharma companies, HTA agencies, and universities, whose mission is to facilitate the adoption and implementation of real-world evidence in health care decision-making in Europe.

Continuing with the HTA topic, I read with interest Akira Yuasa and colleagues’ article on the factors influencing HTA decisions across agencies in eight countries. This involved looking at original submissions and resubmissions, which is quite the feat, as these documents often span hundreds of pages. The factors influencing HTA decisions vary by HTA agency. For example, cost/QALY ICERs are used in Canada, the UK, and Australia for reimbursement decisions, while France’s HAS and Japan’s C2H use them for price negotiation. Germany’s IQWIG, for example, used cost analysis for cancer drugs and for some (but not all) of the hepatitis C drugs. Spain’s AETSA and Italy’s AIFA did not mention economic evaluation in their dossiers. As the article mentions, one problem with these cross-agency comparisons is that it depends on what is reported (and publicly available) in the HTA dossiers. Nevertheless, it’s good to know how the practice varies across the world.

Still on HTA, I read the paper on the evidence review group’s perspective of the NICE appraisal of encorafenib with binimetinib (Enco + Bini) versus dabrafenib with trametinib (Dab + Tram) as a first-line treatment for advanced (unresectable or metastatic) BRAF V600 mutation-positive melanoma. This kind of article used to be a regular feature in PharmacoEconomics but has become much rarer in recent years. I find them quite useful to have a quick overview of the appraisal without going through the NICE documentation. In common with many NICE appraisals of cancer drugs, the trial data were immature for overall survival, with the primary outcome being progression-free survival. One remarkable (and unusual) aspect of this appraisal was that the evidence review group concluded that the new drug was equivalent to the comparator, therefore a cost-minimisation analysis was appropriate.

I had a closer look at the cost-effectiveness paper by Adam Fridhammar and colleagues on a hypothetical diagnostic test for prostate cancer in the Swedish setting. They compared 4 options: current clinical practice where men with a specific range of PSA levels have a biopsy, and 3 options using the hypothetical test within a larger or more restricted range of PSA levels. They report the maximum test price for which its use would be cost-neutral and cost-effective at the threshold of SEK 500k/QALY, as well as the relationship to sensitivity and specificity. Their Table 4 shows how it can be misleading to look only at cost, as the strategy with the highest cost-effective price (that is, the cost-effective strategy given the assumed sensitivity and specificity) is the strategy with a negative cost-neutral price (that is, it increases costs).

Finally, I read my colleague Fan Yang et al.’s article on whether rural residents in China understand the EQ-5D-5L. Fan and colleagues recruited and conducted semi-structured interviews with 62 rural farm workers. Interestingly, 6 participants did not understand the ‘anxiety’ domain and 9 the ‘depression’ domain. For example, one participant suggested, “Just ask it simply, like whether you are happy or not”. It may or may not be related that the anxiety/depression domain was selected as the least important by 37 participants. I wonder how well the concept of ‘happiness’ maps to the concept of ‘depression’. Also, how widespread are issues of comprehension of these and other terms vs cultural differences in the concepts? Measuring and valuing health-related quality of life continues to be both important and challenging, to which this article contributes.


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  • Rita is a health economist at the University of York working mainly in economic evaluation. See https://tinyurl.com/y8ogvhjw for her academic profile.

1 thought on “Journal round-up: PharmacoEconomics – Open 5(1)”

  1. I second your point about NICE evidence review group articles being very useful. I suppose their demise (if our assessment is correct) is partly about competition for space in the journals, but mostly about the ERGs just not having the time.

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