Chris Sampson’s journal round-up for 12th August 2019

Every Monday our authors provide a round-up of some of the most recently published peer reviewed articles from the field. We don’t cover everything, or even what’s most important – just a few papers that have interested the author. Visit our Resources page for links to more journals or follow the HealthEconBot. If you’d like to write one of our weekly journal round-ups, get in touch.

Developing open-source models for the US health system: practical experiences and challenges to date with the Open-Source Value Project. PharmacoEconomics [PubMed] Published 7th August 2019

PharmacoEconomics will soon publish a themed issue on transparency in decision modelling (to which I’ve contributed), and this paper – I assume – is one that will feature. At least one output from the Open-Source Value Project has featured in these round-ups before. The purpose of this paper is to describe the experiences of the initiative in developing and releasing two open-source models, one in rheumatoid arthritis and one in lung cancer.

The authors outline the background to the project and its goal to develop credible models that are more tuned-in to stakeholders’ needs. By sharing the R and C++ source code, developing interactive web applications, and providing extensive documentation, the models are intended to be wholly transparent and flexible. The model development process also involves feedback from experts and the public, followed by revision and re-release. It’s a huge undertaking. The paper sets out the key challenges associated with this process, such as enabling stakeholders with different backgrounds to understand technical models and each other. The authors explain how they have addressed such difficulties along the way. The resource implications of this process are also challenging, because the time and expertise required are much greater than for run-of-the-mill decision models. The advantages of the tools used by the project, such as R and GitHub, are explained, and the paper provides some ammunition for the open-source movement. One of the best parts of the paper is the authors’ challenge to those who question open-source modelling on the basis of intellectual property concerns. For example, they state that, “Claiming intellectually property on the implementation of a relatively common modeling approach in Excel or other programming software, such as a partitioned survival model in oncology, seems a bit pointless.” Agreed.

The response to date from the community has been broadly positive, though there has been a lack of engagement from US decision-makers. Despite this, the initiative has managed to secure adequate funding. This paper is a valuable read for anyone involved in open-source modelling or in establishing a collaborative platform for the creation and dissemination of research tools.

Incorporating affordability concerns within cost-effectiveness analysis for health technology assessment. Value in Health Published 30th July 2019

The issue of affordability is proving to be a hard nut to crack for health economists. That’s probably because we’ve spent a very long time conducting incremental cost-effectiveness analyses that pay little or no attention to the budget constraint. This paper sets out to define a framework that finally brings affordability into the fold.

The author sets up an example with a decision-maker that seeks to maximise population health with a fixed budget – read, HTA agency – and the motivating example is new medicines for hepatitis C. The core of the proposal is an alternative decision rule. Rather than simply comparing the incremental cost-effectiveness ratio (ICER) to a fixed threshold, it incorporates a threshold that is a function of the budget impact. At it’s most basic, a bigger budget impact (all else equal) means a greater opportunity cost and thus a lower threshold. The author suggests doing away with the ICER (which is almost impossible to work with) and instead using net health benefits. In this framework, whether or not net health benefit is greater than zero depends on the size of the budget impact at any given ICER. If we accept the core principle that budget impact should be incorporated into the decision rule, it raises two other issues – time and uncertainty – which are also addressed in the paper. The framework moves us beyond the current focus on net present value, which ignores the distribution of costs over time beyond simply discounting future expenditure. Instead, the opportunity cost ‘threshold’ depends on the budget impact in each time period. The description of the framework also addresses uncertainty in budget impact, which requires the estimation of opportunity costs in each iteration of a probabilistic analysis.

The paper is thorough in setting out the calculations needed to implement this framework. If you’re conducting an economic evaluation of a technology that could have a non-marginal (big) budget impact, you should tag this on to your analysis plan. Once researchers start producing these estimates, we’ll be able to understand how important these differences could be for resource allocation decision-making and determine whether the likes of NICE ought to incorporate it into their methods guide.

Did UberX reduce ambulance volume? Health Economics [PubMed] [RePEc] Published 24th June 2019

In London, you can probably – at most times of day – get an Uber quicker than you can get an ambulance. That isn’t necessarily a bad thing, as ambulances aren’t there to provide convenience. But it does raise an interesting question. Could the availability of super-fast, low-cost, low-effort taxi hailing reduce pressure on ambulance services? If so, we might anticipate the effect to be greatest where people have to actually pay for ambulances.

This study combines data on Uber market entry in the US, by state and city, with ambulance rates. Between Q1 2012 and Q4 2015, the proportion of the US population with access to Uber rose from 0% to almost 25%. The authors are also able to distinguish ‘lights and sirens’ ambulance rides from ‘no lights and sirens’ rides. A difference-in-differences model estimates the ambulance rate for a given city by quarter-year. The analysis suggests that there was a significant decline in ambulance rates in the years following Uber’s entry to the market, implying an average of 1.2 fewer ambulance trips per 1,000 population per quarter.

There are some questionable results in here, including the fact that a larger effect was found for the ‘lights and sirens’ ambulance rate, so it’s not entirely clear what’s going on. The authors describe a variety of robustness checks for our consideration. Unfortunately, the discussion of the results is lacking in detail and insight, so readers need to figure it out themselves. I’d be very interested to see a similar analysis in the UK. I suspect that I would be inclined to opt for an Uber over an ambulance in many cases. And I wouldn’t have the usual concern about Uber exploiting its drivers, as I dare say ambulance drivers aren’t treated much better.

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Thesis Thursday: Rebecca Addo

On the third Thursday of every month, we speak to a recent graduate about their thesis and their studies. This month’s guest is Dr Rebecca Addo who has a PhD from the University of Technology Sydney. If you would like to suggest a candidate for an upcoming Thesis Thursday, get in touch.

Title
The feasibility of health technology assessment (HTA) in the Ghanaian health system
Supervisors
Jane Hall, Stephen Goodall, Marion Haas
Repository link
http://hdl.handle.net/10453/133353

Why is now the right time to research the feasibility of HTA in Ghana?

In recent years, Ghana has been struggling to financially sustain the National Health Insurance Scheme (NHIS), through which it aims to attain universal health coverage (UHC). As a result, a number of payment methods have been explored, including capitation, but costs to the NHIS continue to escalate. The search for a more efficient NHIS funding resulted in stakeholders visiting the then NICE International, learnt of HTA, and expressed an interest in pursuing it. This interest was strengthened by the World Health Organization 2014 resolution, which encouraged its member states to adopt health interventions and technology assessments in support of UHC. In 2016, a pilot HTA study was conducted with support from international bodies that demonstrated potential cost savings with HTA. Subsequently, the Ghana National Medicines Policy, 2017, made provisions for the use of HTA in the selection of medicines. What remains uncertain is how the policy will be implemented, considering that the limited use of HTA in developing countries has been attributed to a lack of human capacity to undertake it, quality data, and limited resources to support it. With Ghana making progress towards the formal adoption of HTA for health decision-making, it is important to examine its feasibility considering the available national capacity and the health system’s particular characteristics, and to make recommendations on how Ghana can proceed, so that the anticipated positive changes can be realised.

What determines ‘feasibility’ in this context?

The usefulness of HTA to any health system is highly dependent on its availability, the quality of assessment, and the human capacity to conduct country specific appraisals. Thus ‘feasibility’ in this context is determined by the existing health resources and systems that could support the adoption and use of HTA in Ghana. Health resources include human capacity with the needed technical skills to conduct and contribute to HTA, funding for the HTA processes, and the available data, which is of good quality and easily accessible. In addition, potential users of HTA should have knowledge in HTA and be able to interpret its findings. Without these building blocks, HTA in itself cannot be successfully used in Ghana. The systems to consider are health system characteristics such as existing health decision-making processes, and political and social structures. Knowledge of this would aid with planning, design, and introduction of an HTA process that suits the Ghanaian health system’s decision-making context, which would promote its use.

How is HTA perceived by stakeholders in Ghana?

Whilst the majority of Ghanaian stakeholders who participated in my study understood HTA as a decision making tool, others saw it as using technologies such as telemedicine and mobile phone devices for healthcare delivery. Their prior understanding of HTA and its uses drove these differences. In terms of its potential use in the Ghanaian health system, most stakeholders acknowledged the benefits the health system stood to gain should HTA be adopted. They however perceived some barriers to the successful implementation of HTA and made some recommendations to address them. Perceived barriers included lack of knowledge of HTA by potential users, lack of human resource capacity to conduct it, lack of funds to support the conduct, and existing ways of making decisions. Factors perceived to promote HTA use were allocating funds for HTA activities, educating stakeholders on HTA and involving them in the planning, and introduction of HTA for health decision-making in Ghana. Also, stakeholders recommended that data be collated and managed for HTA, and for local Ghanaians to be trained to conduct HTA but rely on experts from other countries where possible.

Was it especially challenging to conduct an economic evaluation in the Ghanaian context?

Yes. Conducting a Ghanaian specific economic evaluation was very challenging, especially, in getting the appropriate data. There were no country-specific utility and clinical efficacy data, hence, I had to rely on data from elsewhere, which needed to the transformed to be context specific. The most challenging aspect was with getting appropriate clinical data due to the differences between clinical trial settings and the Ghanaian setting. Applicability issues that were addressed included differences in clinical treatment algorithm, alternative treatments, and epidemiology of disease. Cultural acceptance of available treatment for the study population also defined the appropriate comparator for the evaluation and consequently the clinical data that could be considered. This resulted in having to draw on data from two separate arms of two clinical trials for one of the models I built for my economic evaluation. To ensure applicability of data from other countries to Ghana, the data identified were transformed to be context specific with data input from Ghana either not available or not easily accessible. Therefore, clinical experts were relied upon for such inputs, adding to the limitations of the economic evaluation.

Can HTA processes from other countries be applied in Ghana?

Every health system is unique in its entirety, therefore processes used in one cannot be adopted and applied to the other. The same applies to HTA in Ghana. As part of my thesis, I reviewed a number of HTA organisations across the world to assess if one could be adopted in Ghana. The review revealed that HTA processes vary with each health system in terms of the context under which they were established, the scope or focus of HTA, outcomes, and links to funding decisions and their uses. The establishment of most of these HTA organisations was driven by country specific needs such as curbing the rising costs of healthcare and reducing variations in the availability of quality treatment and care. The available resources, such as human and data, and the health systems characteristics also influenced the HTA processes. Therefore it is not advisable for Ghana to simply adopt and use a model of HTA process from other countries. Rather, Ghana must pursue a country specific HTA process that is informed by relevant country data.

What would be your recommended ‘next step’ for HTA in Ghana?

Firstly, to ensure the acceptance, use and diffusion of HTA in Ghana, stakeholders of health should be educated on HTA and a legal framework stipulating its focus and conduct, and mandating its use, to be adopted.

Secondly, in the short-to-medium term, Ghana can leverage on ongoing collaborations with other countries and foreign organisations, such as the International Decision Support initiative (IDSi), to develop local capacity for HTA. In the long-term, it will be necessary for policy makers to explore the human resource capacity available for HTA in Ghana to guide the development of a human resource plan for HTA.

Thirdly, Ghana has to develop a country-specific methodological guideline or adapt an existing one for the conduct and reporting of economic evaluation studies in Ghana. Subsequently, guidelines for conducting HTA should be developed.

Lastly, to support HTA conduct, Ghana must create a national data repository including a manual on health resource use and their corresponding unit prices. The creation of an HTA standing panel of clinical experts and other stakeholders who could be relied upon to supply inputs for HTA when needed is also recommended. This is very important in the Ghanaian setting where availability and access to data is limited.

Rachel Houten’s journal round-up for 22nd April 2019

Every Monday our authors provide a round-up of some of the most recently published peer reviewed articles from the field. We don’t cover everything, or even what’s most important – just a few papers that have interested the author. Visit our Resources page for links to more journals or follow the HealthEconBot. If you’d like to write one of our weekly journal round-ups, get in touch.

To HTA or not to HTA: identifying the factors influencing the rapid review outcome in Ireland. Value in Health [PubMed] Published 6th March 2019

National health services are constantly under pressure to provide access to new medicines as soon as marketing authorisation is granted. The NCPE in the Republic of Ireland has a rapid review process for selecting medicines that require a full health technology assessment (HTA), and the rest, approximately 45%, are able to be reimbursed without such an in-depth analysis.

Formal criteria do not exist. However, it has previously been suggested that the robustness of clinical evidence of at least equivalence; a drug that costs the same or less; an annual (or estimated) budget impact of less than €0.75 million to €1 million; and the ability of the current health systems to restrict usage are some of what is considered when making the decision.

The authors of this paper used the allocation over the past eight years to explore the factors that drive the decision to embark on a full HTA. They found, unsurprisingly, that first-in-class medicines are more likely to require an HTA as too are those with orphan status. Interestingly, the clinical area influenced the requirement for a full HTA, but the authors consider all of these factors to indicate that high-cost drugs are more likely to require a full assessment. Drug cost information is not publicly available and so the authors used the data available on the Scottish Medicine Consortium website as a surrogate for costs in Ireland. In doing so, they were able to establish a relationship between the cost per person for each drug and the likelihood of the drug having a full HTA, further supporting the idea that more expensive drugs are more likely to require HTA. On the face of it, this seems eminently sensible. However, my concern is that, in a system that is designed to deliberately measure cost per unit of health care (usually QALYs), there is the potential for lower-cost but ineffective drugs to become commonplace while more expensive medicines are subject to more rigor.

The paper provides some insight into what drives a decision to undertake a full HTA in Ireland. The NICE fast-track appraisal system operates as an opt-in system where manufacturers can ask to follow this shorter appraisal route if their drug is likely to produce an ICER of £10,000 or less. As my day job is for an Evidence Review Group (opinions my own), how things are done elsewhere – unsurprisingly – captured my attention. The desire to speed up the HTA process is obvious but the most appropriate mechanisms in which to do so are far from it. Whether or not the same decision is ultimately made is what concerns me.

NHS joint working with industry is out of public sight. BMJ [PubMed] Published 27th March 2019

This paper suggests that ‘joint working arrangements’ – a government-supported initiative between pharmaceutical companies and the NHS – are not being implemented according to guidelines on transparency. These arrangements are designed to promote collaborative research between the NHS and industry and help advance NHS provision of services.

The authors used freedom of information requests to obtain details on how many trusts were involved in joint working arrangements in 2016 and 2017. The declarations of payments made by drug companies are disclosed but the corresponding information from trusts is less readily accessible, and in some cases access to any details was prevented. Theoretically, the joint working arrangements are supposed to be void of any commercial influence on what is prescribed, but my thoughts are echoed in this paper when it asks “what’s in it for the private sector?” The sheer fact that some NHS trusts were unwilling to provide the BMJ with the information requested due to ‘commercial interest’ rings huge alarm bells.

I’m not completely cynical of these arrangements in principle, though, and the paper cites a couple of projects that involved building new facilities for age-related macular generation, which likely offer benefits to patients, and possibly much faster than could have been achieved with NHS funding alone. Some of the arrangements intend to push the implementation of national guidance, which, as a small cog in the guidance generation machine, I unashamedly (and predictably) think is a good thing.

Does it matter to us? As economists, it means that any work based on national practice and costs is likely to be unrepresentative of what actually happens. This, however, has always been the case to some extent, with variations in local service provision and the negotiation power of trusts with large volumes of patients. A national register of the arrangements would have the potential to feed into economic analysis, even if just as a statement of awareness.

Can the NHS survive without getting into bed with industry? Probably not. I think the paper does a good job of presenting the arguments on all sides and pushing for increasing availability of what is happening.

Estimating joint health condition utility values. Value in Health [PubMed] Published 22nd February 2019

I’m really interested in how this area is developing. Multi-morbidity is the norm, especially as we age. Single condition models are criticised for their lack of representation of patients in the real world. Appropriately estimating the quality of life of people with several chronic conditions, when only individual condition data are available, is incredibly difficult.

In this paper, parametric and non-parametric methods were tested on a dataset from a large primary care patient survey in the UK. The multiplicative approach was the best performing for two conditions. When more than two conditions were considered, the linear index (which incorporates additive, multiplicative, and minimum models with the use of linear regression and parameter weights derived from the underlying data) achieved the best results.

Including long-term mental health within the co-morbidities for which utility was estimated produced biased estimates. The authors discuss some possible explanations for this, including the fact that the anxiety and depression question in the EQ-5D is the only one which directly maps to an individual condition, and that mental health may have a causal effect on physical health. This is a fascinating finding, which has left me somewhat scratching my head as to how this oddity could be addressed and if separate methods of estimation will need to be used for any population with multi-morbidity including mental health conditions.

It did make me wonder if more precise EQ-5D data could be helpful to uncover the true interrelationships between joint health conditions and quality of life. The EQ-5D asks patients to think about their health state ‘today’. Although the primary care dataset used includes 16 chronic health conditions, it doesn’t, as far as I know, contain any information on the symptoms apparent on the day of quality of life assessment, which could be flaring or absent at any given time. This is a common problem with the EQ-5D and I don’t think a readily available data source of this type exists, so it’s a thought on ideals. Unsurprisingly, the more joint health conditions to be considered, the larger the error in terms of estimation from individual conditions. This may be due to the increasing likelihood of overlap in the symptoms experienced across conditions and thus a violation of the assumption that quality of life for an individual condition is independent of any other condition.

Whether the methodology remains robust for populations outside of the UK or for other measures of utility would need to be tested, and the authors are keen to highlight the need for caution before running away and using the methods verbatim. The paper does present a nice summary of the evidence to date in this area, what the authors did, and what it adds to the topic, so worth a read.

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