Research Synthesis: Health Technology Assessment
V1.0 researched and written by Elise Erickson, edited by Suerie Moon, last updated June 2018
The literature surrounding health technology assessment (HTA) is rich*. One challenge is that there is no set terminology or clear borders when referring to the issue of how to assess the value of a medicine, and its relationship to price. "HTA" is used in some of the literature, whereas others use "pharmacoeconomics" or "cost-effectiveness analysis," and others "value-based pricing." Adding to this challenge, it is a highly technical topic, which means that a significant amount of the literature may be less accessible to non-specialists.
Medicine/drug/pharmaceutical and health technology assessment, cost-effective analysis, pharmacoeconomics, value-based pricing.
Synthesis of the literature
In general, the literature notes the increasing use of HTA in health decision-making as countries continue to spend more on health care, and usually promotes the use of HTA, while also recognizing and critiquing its limitations. Towse and Barnsley (2013) provide an approachable, general overview of the topic, and Henshall and Schuller (2013) provide an overview of the different ways to assess the value of a medicine (see in particular ‘Table 1.Summary of Core and Wider Elements of Value, and Approaches to Measurement and Valuation’). Sorensen, Drummond and Kanavos’ (2008) report "Ensuring Value for Money in Health Care: The Role of HTA in the EU" also provides a useful overview of the surrounding debate.
The WHO’s Guideline on Country Pharmaceutical Pricing Policies (2015), for instance, recommends using HTA as a tool for reimbursement decision-making, and for price setting and negotiation, but recognizes that HTA requires advanced technical capacity and notes the need for accompanying legislative/administrative frameworks, and transparency throughout the process. For a detailed list of studies carried out concerning HTA, see Annex J of the WHO Guidelines on Country Pharmaceutical Pricing Policies. Similarly, a report by Sorensen, Drummond, and Kanavos (2008), which focuses on HTA in Europe, argues that HTA plays an important role in evidence-based decision making, but notes several weak points including the disparate roles of different HTA bodies, the need for more stakeholder involvement in the HTA process (i.e. greater involvement of patients and industry), and lack of transparency in the HTA process (2008).
Much of the discussion focuses on how best to calculate value (i.e. which factors should and should not be included), how to operationalize value-based pricing (VBP), and how to use it in health decision-making (World Health Organization (WHO) 2015; Sussex, Towse, and Devlin 2013). Most studies look at HTA as an independent tool, rather than comparing it to other pricing tools. One study, however, compares HTA with reference pricing and found that HTA is superior (in that it is more nuanced), but it also noted the high cost of carrying out HTAs (Drummond et al. 2010).
Most of the studies focus on high income countries (Drummond et al. 2010). There are country-specific papers for the UK (NICE), Canada, Australia, Sweden, US, and Germany, among others; and some papers that compare processes across countries (Henry, Hill, and Harris 2005). There exist a handful of studies analyzing HTA agencies in particular, including in Europe, the UK, Australia, and many in Canada. While some studies introduce and describe the various agencies (e.g. Canada (Lefebvre, Lafeuille, and Tiggelaar 2017; Menon and Stafinski 2009; Paris and Belloni 2014), and Australia (Hailey 2009)), others examine more specific aspects of the agencies. In Canada, the Common Drug Review (CDR) was introduced in 2003 in place of the 18 separate review processes that previously existed (“CADTH Common Drug Review (CDR),” n.d.).
When examining the extent to which the listing decisions align with HTA body recommendations, Allen et al. (2016) found “moderate to substantial agreement” between the provincial listing decisions and the CDR. Comparing the quantity drug listings before and after the establishment of Canada’s CDR, Gamble et al. (2011), find that fewer drugs were listed after the agency’s creation, and that the time-to-listing fell in certain smaller Canadian provinces. A study commissioned by the Canadian Agency for Drugs and Technologies in Health examined how patient perspectives were assimilated into Common Drug Review (CDR) assessment reports and recommendations, finding that patient views were used to situate assessments and to interpret the evidence (Berglas et al. 2016). The study also stresses the importance of taking both the recovery process and health sustainability into account when assessing a drug. McCormick, Berescu, and Tadros (2018) examined recommendations for orphan drugs in Canada specifically, finding that positive recommendations have increased over time, usually conditional on a drop in price. In the UK context, a study by Dakin et al. (2015) suggests that NICE decision-making and thresholds for rejection had not changed significantly over time. When examining the extent to which NICE guidance is implemented, Sheldon et al. (2004) find that implementation varied depending on several factors including strong professional support, a clear evidence base, and no increase in cost. Looking at Sweden, a study of the Pharmaceutical Benefits Board (LFN)’s priority-setting procedure suggests that an accountable and reasonable procedure can bring about priority-setting that is “generally perceived as fair and legitimate by the major stakeholders and may increase social learning in terms of accepting the necessity of priority setting in health care” (Jansson 2007).
There are significantly fewer analyses of HTA in LMICs. A study of middle-income countries use of HTA found that their use of HTA is increasing, but developing at an uneven speed (Oortwijn, Mathijssen, and Banta 2010). Sivalal (2009) describe how Malaysia’s HTA agency has increased in terms of both size and resources since its creation in 1995, but note major challenges with regards to sustainability, including: a sufficient level of trained staff and appropriate awareness around the function and value of the agency. Previously, Sivalal (1998) describe an HTA training course in Malaysia shortly after its agency was created. The authors suggest this could be a useful resource for others designing HTA training courses in developing countries. Teerawattananon et al. (2009) describe the various factors that contributed to the development of HTA in Thailand, including studies on the poor distribution of health technologies, followed by an economic recession and the implementation of a universal health coverage plan.
Other studies compare HTA agencies across countries. For instance, a study comparing the value-added assessments (those that go beyond the HTA economic or clinical benefit assessment) between European countries found a number of similarities and differences with regards to the practices of value-assessment, and argued for greater transparency in the value-assessment criteria to enhance resource allocation and therefore societal welfare (Angelis, Lange, and Kanavos 2018). In fact, concerns over transparency in these processes was a reoccurring theme in the literature (Hailey 2009). Clement et al. (2009) compare how effectiveness and cost-effectiveness are used in Canada’s CDR, the UK’s NICE, and Australia’s PBAC. They conclude that the listing decisions varied between the agencies—which is unsurprising considering that the agencies have different mandates and processes. In fact, the authors suggest that the differences in decisions are mostly driven by differences in processes and risk attitudes, rather than the interpretation of clinical or economic evidence. McMahon, Morgan, and Mitton (2006) highlighted various considerations for Canada’s CDR (just after CDR was created) based on lessons learned from the UK’s NICE.
More studies analyzing use or feasibility of HTA in low- or middle-income countries
Analyses of HTA as an input to medicines pricing and incentive for innovation
* For the purposes of this review, we have established three categories to describe the state of the literature: thin, considerable, and rich.
- Thin: There are relatively few papers and/or there are not many recent papers and/or there are clear gaps
- Considerable: There are several papers and/or there are a handful of recent papers and/or there are some clear gaps
- Rich: There is a wealth of papers on the topic and/or papers continue to be published that address this issue area and/or there are less obvious gaps
Scope: While many of these issues can touch a variety of sectors, this review focuses on medicines. The term medicines is used to cover the category of health technologies, including drugs, biologics (including vaccines), and diagnostic devices.
Disclaimer: The research syntheses aim to provide a concise, comprehensive overview of the current state of research on a specific topic. They seek to cover the main studies in the academic and grey literature, but are not systematic reviews capturing all published studies on a topic. As with any research synthesis, they also reflect the judgments of the researchers. The length and detail vary by topic. Each synthesis will undergo open peer review, and be updated periodically based on feedback received on important missing studies and/or new research. Selected topics focus on national and international-level policies, while recognizing that other determinants of access operate at sub-national level. Work is ongoing on additional topics. We welcome suggestions on the current syntheses and/or on new topics to cover.