Ewan Gray’s journal round-up for 16th November 2020

Every Monday our authors provide a round-up of the latest peer-reviewed journal publications. We cover all issues of major health economics journals as well as some other notable releases. If you’d like to write one of our weekly journal round-ups, get in touch.

International Journal of Technology Assessment in Health Care

Volume 36, Issue 4

The latest issue of the official journal for the organisation Health Technology Assessment International (HTAi) has a methodological and policy focus as one might expect.

Development-focused HTA (DF-HTA) is offered as an alternative description for ‘early HTA’ or ‘supply-side’ HTA. A commentary presents a set of 10 questions that health economists may find useful when discussing DF-HTA with product developers with a goal of clarity in the objectives of the analysis. As someone with experience of these type of studies, I agree these questions are a useful starting point. Other commentaries discuss multi-criteria decision analysis (MCDA), and there was a timely look at issues facing assessment of drugs for Alzheimer’s disease.

Among methods papers, there was a review of horizon scanning approaches for cell and gene therapies, another review of intersectoral benefits to education from mental health interventions, a comparison of restricted mean survival time and two-state Markov microsimulation for extrapolation of trial results, and development of a benchmarking checklist for HTA agencies. The highlight for me was a review of NICE technology appraisal submissions which demonstrated the inadequacy of validation processes to deal with technical errors in submitted models. In a sample of 41 appraisals, 95% contained one or more errors (usually between one and ten). The errors were roughly evenly split between transcription errors, logic errors, and computational errors. While errors were not always influential for the results of the analysis, it does suggest some value to adding redundancy to the process, and to the use of external review groups in the NICE technology appraisal process.

Ten assessments were published in this edition, although most of these are not economic evaluations. Systematic reviews included a collection of economic evaluations of ventricular assist devices and another of economic evaluations of mixed home care. There was also a review of intersectoral benefits in criminal justice of mental health interventions. In the domain of real-world evidence, there was an investigation of real-world evidence studies in type 2 diabetes and a review looking at real-world evidence use in NICE single technology appraisals. Technology appraisals of precision medicines by the Belgian public health payer, RIZIV-INAMI, were reviewed to assess their methodological quality. Appraisals from another HTA agency – the pan-Canadian Oncology Drug Review (pCODR) – were reviewed to try to determine if indication rarity affects funding decisions. Original evaluations included a cost analysis of 3D printing costs in surgery and an assessment of the effectiveness of wearable cardio-defibrillators in sudden cardiac arrest.

Finally, there were two policy articles. An examination of NICE’s evaluations of products with the more limited European conditional marketing authorization (CMA) or authorization in exceptional circumstances (AEC) showed these were associated with commercial arrangements (price reductions or risk-sharing) reflecting a lack of evidence. A narrative review of a Canadian experience switching from HTA considering only effectiveness and cost-effectiveness (no explicit threshold) to MCDA to inform decision making for drug reimbursement decisions expressed very positive sentiments. However, the impression I was left with was that, while the stated goal of the change was an improvement in consistency and transparency, the resulting process appears not more but perhaps less transparent. Clinical effectiveness evidence and ICERs, as well as new criteria, are reduced to scores on a 4-point scale, which are then qualitatively combined to reach a recommendation. In favour of the new process, it appears to have more conveniently adopted other criteria of safety, severity, unmet clinical need, and equity.

Cost Effectiveness and Resource Allocation

Volume 18, October 2020

As is typical for this journal, the last month’s articles include a truly international selection with six countries represented out of eight articles.

First up, a descriptive study of Taiwan’s policies for mask distribution via local pharmacies during the COVID-19 pandemic. Given Taiwan’s successful control measures, there may be useful lessons for other countries in studying the details of policy implementation. By implementing a rationing system, based on people’s unique national health insurance numbers, it was possible to prevent shortages and control prices early in the pandemic when supplies were scarce.

Also related to universal health insurance, a mixed-methods study from Nepal investigated barriers to achieving roll-out and complete coverage. A major issue seems to be selection out of the system by the better-off, who prefer to use private medical care services.

There were a pair of studies conducted in Iran. Willingness-to-pay for one QALY (life-extension) was elicited using a contingent valuation method. The mean WTP for one QALY was an astoundingly low at $862, around 20% of Iranian per-capita GDP. Next, an unusual review of policies aimed at collating all budgetary allocations for disaster responses over the last 100 years, demonstrating their outsize budget impact.

A big picture review of evaluation methods for health policy provides some food for thought, reminding me of the need to bridge the gap between cost-effectiveness as a decision rule and a more holistic assessment of policy.

A study of resource use and medical costs for tonsillectomy with adenoidectomy on otitis media in South Korea found no difference in the use of otitis media-related services between surgery and non-surgery groups. While this result may appear uninteresting, it may be more important given the context that surgery is justified partly on the basis that it is thought to reduce recurrent interventions.

Rounding out the set are cost-effectiveness analyses of Coblation versus mechanical shaver debridement in patients following knee chondroplasty and nusinersen for patients with infantile-onset spinal muscular atrophy, both set in the USA. The nusinersen study is a truly challenging topic for cost-effectiveness analysis, with large (but uncertain) QALY gains that may be transformative for patients alongside very high costs, producing ICERs in the region of $1,000,000.

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