Topics A Study on the Diverse Values of Pharmaceuticals A Study on the Trend of COVID-19 and DTx, etc.

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Yosuke Nakano, Senior Researcher, National Institute of Biomedical Innovation Policy

1. Introduction

In Research Paper No. 761 ^{), the} authors focused on the various values of pharmaceuticals, especially social values, and investigated the current status of outcome indicators and measurement of these values, as well as the current status of their evaluation overseas (mainly in the UK). The value of pharmaceuticals can change depending on the social conditions and technological progress of the time, and the recent emergence of new coronavirus infection (COVID-19) and digital therapeutics (DTx), etc., due to the advancement of digital technology, are two examples of the various values of pharmaceuticals that can be considered to be of great importance in the study of the various values of pharmaceuticals. We speculate that the emergence of COVID-19 and digital technologies such as digital therapeutics (DTx) may have some impact, large or small, on the consideration of the various values of pharmaceuticals.

In this paper, we focus on the developments related to COVID-19, DTx, and others, and discuss their implications for the various values of pharmaceuticals.

2. considerations based on trends related to COVID-19

Considerations from the COVID-19 pandemic ¹⁾

First, let us consider the impact of the COVID-19 pandemic itself on healthcare and pharmaceuticals in general.

The COVID-19 pandemic, which began to spread globally in early 2020, has had a number of impacts on healthcare. Face-to-face medical care and hospital visits, which had been the norm up to now, became a risk for the spread of infection, and patients were discouraged or avoided receiving medical care (reports indicate that 30% to 40% of patients in Japan discouraged or avoided receiving medical ^{care2, 3)} ). In addition, the physical and mental burden on healthcare workers who had to take the risk of infection and the shortage of manpower also became a major problem. And in some periods and regions around the world, including Japan, the shortage of specialized wards for infectious diseases and ventilators made the collapse of medical care a real crisis. As a result, the question has arisen as to how to manage the healthcare delivery system while efficiently optimizing finite healthcare resources (medical facilities, healthcare workers, etc.) in order to prevent healthcare from collapsing.

In fact, in the UK, in order to keep the supply of physical healthcare resources such as medical facilities and healthcare workers uninterrupted, medical practice guidelines (COVID-19 rapidguideline) for patients being treated for existing diseases to "minimize the risk of infection to patients and healthcare workers while maintaining the quality of care" were ⁽ COVID-19 rapidguideline) has been issued immediately after the COVID-19 outbreak ^.4)

These guidelines were developed by the National Institute for Health and Care Excellence (NICE), the UK's health technology assessment body, and as of the end of August 2021, 19 different guidelines had been published (Table 1). Of these, 17 guidelines (excluding two) are not for the treatment of COVID-19 itself, but for the provision of appropriate medical care for patients being treated for other diseases, even when the medical supply system is under pressure due to COVID-19. Therefore, the guidelines propose not only treatment guidelines for patients, but also comprehensive measures based on "the possibility that uninfected patients may be infected at medical institutions" and "the possibility that patients are potentially infected with COVID-19, which could then be transmitted to healthcare workers.

For example, in the case of cancer treatment (NG161) and rheumatoid arthritis (NG167), it suggests changing to a regimen that requires less frequent visits to the hospital (e.g., from intravenous to oral administration, drugs that can be administered at home, and drugs with longer administration intervals), and the existence of these options itself is beneficial not only to patients but also to healthcare professionals. Prior to the COVID-19 pandemic, drugs that reduced the frequency of hospital visits were primarily viewed as improving convenience and reducing burden on the patient side. However, with the threat of the COVID-19 pandemic, we believe that health care workers, who work at their own risk of infection, have come to realize that they can also contribute to reducing the risk of infection and the burden of infection.

Considerations from COVID-19 therapeutics ¹⁾

Next, let us consider the value of COVID-19 drugs. Currently, research and development of many COVID-19 drugs is underway, and drugs such as oral drugs and antibody cocktail therapies are now available in Japan. The development of new therapeutic agents is expected to continue both domestically and internationally, and here we introduce some examples of the value of COVID-19 therapeutic agents.

In a case ^study5) in which INESSS, a medical technology evaluation organization in Quebec, Canada, conducted an evaluation of Lemdecivir, a COVID-19 therapeutic drug, including its cost-effectiveness and financial impact in 2020, the following references are made from the perspective of improving the efficiency of medical resources and medical tightness.

Thus, it was considered worthwhile to consider the fact that the shortening of the duration of treatment and the reduction in the consumption of medical resources by the drug itself is clearly put forth as a value. Although this value itself has been conceivable in the past, it is highly likely that it would not have been well recognized before the COVID-19 epidemic, and it is thought that it became strongly recognized with the COVID-19 as an opportunity.

Considerations from the COVID-19 vaccine

Next, we would like to consider this from the perspective of the value in the vaccine against COVID-19 (hereinafter referred to as "COVID-19 vaccine").

The COVID-19 vaccine, which began to be administered in Japan in February 2021, is currently available in Japan at no cost to the individual (free of charge) (the government, however, bears the cost of purchasing the vaccine). However, as with the influenza vaccine, it is possible that the government will eventually shift to self-pay (fee-based) vaccination, and before that time, the appropriateness of the purchase cost by the government may be debated. At that time, it is presumed that an evaluation of the value of the COVID-19 vaccine, including its cost-effectiveness, will be conducted. And in that case, it will be an important point of contention as to what kind of cost and within what scope of impact the value evaluation will be conducted. In July 2020, the U.S. government signed a contract to purchase 100 million doses of Pfizer/Biontec's vaccine for "about $2 billion (about $40 per person)," and the sales price to other developed countries is expected to be at the same ^level6).

In their 2021 paper, Bloom et al. stated that standard health economic evaluations of vaccines focus only on relatively narrow health benefits and do not properly assess the value of ^vaccines7). However, with COVID-19, many people have experienced and understood the broad impact of the pandemic, and there is growing recognition that the COVID-19 vaccine has a wide range of economic and social benefits (social value) beyond avoiding morbidity and mortality from infection and reducing health care costs. The report also notes that the COVID-19 Panels are the first to be published in the United States. He also noted that the revelation of the broad impact of the COVID-19 pandemic may help convince policy makers that the socioeconomic benefits of the COVID-19 vaccine should be properly assessed.

Appleby also points out in his 2020 paper that the UK government has allocated a large budget for measures against COVID-19, in addition to healthcare (Figure 1), and the financial burden is considerable. He then mentions that if the widespread use of the COVID-19 vaccine can reduce these expenditures, the vaccine should be a cheap purchase for the ^government8). In other words, the paper asks whether vaccine evaluations and decisions should be made in light of these effects on national finances and society as a whole.

In Japan, supplementary budgets were compiled three times to implement various measures related to COVID-19, and the total increase in general account expenditures for FY2020 amounted to 76.8 trillion ^yen9) (however, approximately 30 trillion yen was carried over to the next year as a result). This represents an increase of more than 70% over the initial budget, and Japan is similarly suffering from the enormous impact of the infectious disease pandemic on national finances and, by extension, on the entire socioeconomy.

It remains to be seen how the value of the COVID-19 vaccine, which has the potential to prevent a pandemic of this magnitude, will be evaluated in Japan and abroad.

3. considerations from trends in digital health such as digital therapeutics (DTx)

Value assessment based on the characteristics of digital health

The development of new medical technologies related to digital health, such as DTx and programmed medical devices, has been active and may be helpful in considering the various values of pharmaceuticals, so the study was also conducted from the perspective of value evaluation in medical devices.

In August 2020, CureApp's application for nicotine dependence treatment received regulatory approval in Japan (and was covered by insurance from December of the same year), becoming the first DTx in Japan and a hot topic. However, it is said that the current evaluation of the value of medical technologies related to digital health, including DTx and programmed medical devices, has yet to fully evaluate their characteristics.

Under such circumstances, in September 2020, the Center for Medical Devices released "Recommendations from the Study Group on Insurance Reimbursement of Medical Technologies in Light of Advances in Digital Health (abbreviated as the Study Group on AI and Digital Health ⁾¹⁰⁾. In it, the study group indicated the issues involved in the evaluation of medical technologies related to digital health, including DTx, and made five recommendations for evaluation based on their characteristics (Figure 2).

What caught the author's attention in the recommendations is that one of the characteristics of medical technology for digital health is "reduction of the burden on healthcare professionals," and that a system is needed to evaluate this characteristic as a value.

Specifically, "the effects of medical cost containment achieved by improving the quality of medical care, significantly saving medical resources, and shortening service delivery time brought about by medical technologies related to digital health are also positively evaluated," and "the reduction of the burden on medical professionals and the shortening of medical treatment time, which are major characteristics of medical technologies related to digital health, are also important as evaluation indicators. "The reduction of the burden on healthcare professionals and the shortening of medical treatment time, which are major features of digital health-related medical technologies, are also important perspectives as indicators for evaluation. After collecting data on these factors for a certain period of time, a mechanism will be introduced to reevaluate the medical technology (addition or subtraction may be possible).

In the aforementioned discussion related to COVID-19, we referred to the value of reducing the burden on healthcare professionals and improving the efficiency of healthcare resources, but it is very interesting to note that the importance of appropriately evaluating the value of DTx and other digital health technologies based on their characteristics is also mentioned, although in a different context. This is very interesting.

DTx valuation case study in UK NICE

With regard to the aforementioned value of "reducing the burden on healthcare professionals" based on the characteristics of digital health, we will introduce a case study of NICE in the U.K. as a specific example for DTx.

In the UK, the National Health Service (NHS) and NICE are working together to improve access to psychological therapies (Improving Access to Psychological Therapies Program, IAPT). ^{11, 12)} The IAPT evaluates candidate DTx from the perspective of treatment content, digital technology elements, clinical evidence, and resource impact, and publishes the results. In this section, we focus on the "impact on resources," which may be related to "reducing the burden on healthcare professionals" among the evaluation perspectives.

As of August 31, 2021, 14 products (counted separately for each indication) have been evaluated, and 6 products have received a recommendation. The following is an example of the reference to "impact on resources" in two of these products.

Mentioned case study in ^Deprexis13)

Deprexis is a DTx already covered by insurance in Germany and is an online cognitive behavioral therapy program for depression used in conjunction with face-to-face treatment. In terms of "resource implications," the use of Deprexis could free up therapists' free time, allowing more people to receive face-to-face cognitive-behavioral therapy. And he mentions that it could provide treatment at a lower cost than in-person therapy. The fact that human resources, such as therapists, could be made more efficient by combining online treatment programs is also specified in the evaluation, and is mentioned as a characteristic of online treatment programs.

On the product website of Deprexis, ¹⁴⁾ the product characteristics shown in Figure 3 are listed.

The following two points, in particular, were considered to have DTx-specific value other than those mentioned above, and are introduced here as well.

Space from Depression is another online cognitive-behavioral therapy program for depression that has been recommended and evaluated for use in clinical practice. The evaluations of therapists and patients were generally positive.

In the "Resource Impact" section of the evaluation results, a comparison of the time spent in standard face-to-face cognitive-behavioral therapy and Space from Depression in real-life settings was published (total time spent for one course of treatment: 229.6 minutes vs. 92.8 minutes). Although the authors note that in some cases the standard face-to-face cognitive-behavioral therapy time includes other treatment time, making simple comparisons difficult, the time spent in the online program was less than half of the face-to-face therapy time.

Although this is only one case study, it was thought that if such data could be collected and accumulated in the real world as a suggestion for pharmaceuticals, it could serve as one of the grounds for advocating a reduction in the burden on healthcare professionals, etc.

In the aforementioned proposal by the Center for Medical Devices, it is mentioned that "medical technologies related to digital health have a higher affinity for collecting outcome data than conventional medical technologies," and it will be interesting to see what kind of evaluation mechanisms will be proposed by DTx and other organizations once data that can be used for value evaluation is collected. It remains to be seen what kind of evaluation mechanism will be proposed by DTx and others as data that can be used for value evaluation is collected.

The evaluation included comments from patients and therapists who actually used Space from Depression, and we would like to introduce them here.

First, in the patient survey, 66.3% of the respondents (n=134) were "completely satisfied" or "mostly satisfied" with the online therapy. While some said that this online therapy was ideal for them and that they were satisfied with the flexibility and adequate support, some negatively commented that they were dissatisfied with the lack of contact with their therapists and felt isolated without support.

Among therapists, there were many positive comments about the content and ease of use of the online therapy. However, some pointed to issues related to education and work management by the development company and the functionality of the system used.

In addition, it was noted that the use of Space from Depression was useful in maintaining and managing the clinic staff, as it changed the way therapists work and enabled them to work from home.

4. Conclusion

This paper focuses on trends related to COVID-19 and DTx, etc., and discusses the implications and other issues related to the various values of pharmaceuticals. The COVID-19 pandemic has reminded us that medical resources are limited, and it is important to consider the value of pharmaceuticals as a means of reducing the burden on medical personnel. The COVID-19 pandemic reminded us that healthcare resources are limited, and triggered the entire society to recognize the importance of reducing the burden on healthcare professionals and the contribution that pharmaceuticals can make to this.

On the other hand, looking at digital health such as DTx, a system to appropriately evaluate the value of medical technology according to its characteristics is needed.

There are many issues to be addressed in evaluating new value, and although the systems for pharmaceuticals and medical devices are different, there are some points that can be referenced and applied to each other when considering value evaluation.

In terms of "reduction of the burden on medical professionals," at present, one possible way to demonstrate the value is to obtain data on, for example, the time saved in the treatment process and the number of human resources saved, and then add the monetary cost to the data to show the monetary value. However, to obtain a more appropriate quantitative evaluation, it may be necessary to develop outcome indicators and tools that can directly evaluate "burden reduction.

In addition, in evaluating the cost-effectiveness of the COVID-19 vaccine, there is a need for evaluation and judgment from a broader perspective, such as the impact on national finances and the social economy, rather than from the perspective of medical costs alone. The results of future domestic and international evaluation methods and results will be closely watched.

Finally, with the experience of COVID-19 and the development of digital health including DTx, the value perspective is expected to expand further. The pharmaceutical industry will first engage in discussions with each stakeholder to broaden the perspective of value, as well as work on data collection and evidence building necessary for value assessment, and further promote consideration of appropriate value assessment according to disease or innovation characteristics.

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