Points of View Consideration of Outcome Indicators for DTx Reimbursement - Toward Diverse Value Assessment Based on the Latest Trends in the U.S. and Europe

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Junya Tsujii, Senior Researcher, National Institute of Biomedical Innovation Policy

1. Introduction

Since Welldoc's "BlueStar," a blood glucose management application for diabetics, received regulatory approval from the U.S. Food and Drug Administration (FDA) in 2010, multiple digital therapeutics ("DTx") have appeared around the world. In the U.S., for example, more than 10 DTx have already received FDA clearance/approval1), and in Germany, 28 products/indications have been approved for clinical use, including provisional registration for continued review2). Meanwhile, in Japan, CureApp's nicotine dependence treatment application received regulatory approval as the first DTx in Japan in 2020 and was included in the insurance coverage3). 3 ) In May 2021, CureApp also filed an application for a hypertension treatment application4), and DTx development is becoming more active in Japan. 4) In addition, more and more pharmaceutical companies are entering the market, and various products are under development (Table 1).

As digital technology continues to advance, DTx products are appearing one after another, and Japan urgently needs to develop and establish various systems to support their medical development. In FY2021, the Central Social Insurance Medical Council (hereinafter referred to as "Chuikyo") held discussions on the reform of the insured medical material system in FY2022, and included "Evaluation of Programmed Medical Devices" in the insured medical material system. The discussion included the inclusion of "Evaluation of Programmed Medical Devices" in the medical materials system. The committee clearly stated that the axis of evaluation in the insurance treatment should take into account values other than efficacy and safety that reflect the characteristics of programmed medical devices5).

Therefore, in order to examine the evaluation axis for reimbursement of DTx in Japan, this paper focuses on the United States and Germany, which have a large number of DTx approved/approved items, as well as the United Kingdom, where various value evaluations of pharmaceuticals and other products are in progress, and investigates the current status of reimbursement and outcome indicators for DTx in each country.

Table 1 Trends of pharmaceutical companies in DTx development in Japan

2. Summary of this survey

According to the definition of the U.S. Digital Medicine Society, health-related digital solutions are broadly referred to as "digital health," and are further subdivided into "digital medicine" and "DTx" according to the purpose of use and level of evidence6). Of these, DTx, which provides therapeutic interventions for the prevention, management, and treatment of medical disorders and diseases, has the highest level of evidence and requires clinical effectiveness and data collected in actual clinical practice.

In addition, the "Evidence standards framework for digital health technologies" issued by the National Institute for Health and Clinical Excellence (NICE) in the U.K. classifies digital health according to the purpose of use and impact on the human body (risk class). The framework of evidence required for each of these categories is shown7). The evidence required for Tiers A through C is cumulative, and meeting the requirements of Tier C necessarily means meeting the requirements of Tiers A and B as well.

Based on the above, we believe that the consideration of outcome measures to build evidence for DTx will be useful not only for DTx, but also for future consideration of evaluation axes for digital medicine and digital health. It should be noted that since this survey collected a broad range of indicators related to insurance reimbursement, product quality, such as software quality, is also included in the survey results.

Figure 1 Functional classification in NICE Evidence standards framework for digital health technologies

3. Current status of DTx reimbursement in Japan

Before moving on to the results of the survey on reimbursement in each country, we would like to review the current status of reimbursement for DTx in Japan.

In Japan, DTx is organized as one of the components of "Software as a Medical Device (SaMD)," and discussions on reimbursement and a system for practical application are underway for programmed medical devices as a whole. CureApp's nicotine dependence treatment application, the first DTx in Japan, including a CO checker for measuring exhaled carbon monoxide concentration, was evaluated as a "new technology fee " 8). However, in the discussion at the Chuikyo (November 11, 2020) regarding insurance coverage of this application, it was mentioned that "the concept and mechanism of insurance coverage of programmed medical devices need to be organized at an early stage " 9), indicating that a gap existed between the advancement of digital technology and the system at that time.

Based on this background, the Regulatory Reform Implementation Plan (approved by the Cabinet in June 2021) included "clarification of the concept of evaluation for ensuring and improving the quality of medical care provided to patients using programmed medical devices" as a priority regulatory reform implementation item10). Furthermore, the insurance reimbursement system is being discussed mainly by the Chuikyo Expert Committee on Insurance Medical Supplies. In this context, the medical device industry is calling for evaluation based on outcomes in principle, and has proposed that values specific to programmed medical devices, such as equalization of medical care quality and improvement of medical care productivity, be subject to evaluation based on "patient efficacy and safety," "social necessity," and "economic efficiency/efficiency " 11). The draft revision of the system for insured medical materials5), which also takes into account industry opinions, has been approved by the Chuikyo, clarifying that the equalization of medical technology and support for medical treatment will be evaluated from the perspective of reforming the way physicians work. As the implementation of this system progresses, further study is required with reference to reimbursement systems in other countries, in addition to opinions from related industries and others.

Reimbursement pathways and outcome indicators in each country

4-1. United States

The U.S. medical insurance system is essentially different from the Japanese system. There are two major types of public medical insurance: "Medicare" for the elderly over 65 years old and the disabled, and "Medicaid" for low-income people. However, most US citizens are not eligible for public medical insurance and use private medical insurance12). Therefore, DTx reimbursement in the U.S. is characterized by the existence of a pathway through private medical insurance as well as public medical insurance. On the other hand, this makes business predictability more complicated than in Japan, and it is important to envision from the development stage what kind of commercial pathway to pursue and what outcome measures are necessary to achieve this.

Reimbursement by public medical insurance

The FDA is vigorously promoting the design of a new system for SaMD, including DTx, and several products have already received 510(k) and De novo approval/authorization13). On the other hand, the system has not been fully linked to public health insurance, and currently DTx used directly by patients is not covered under the benefit category14). However, against the backdrop of the rapid penetration of digital technology, discussions of reimbursement of DTx by public medical insurance are accelerating.

For example, in March 2020, a bill (Prescription Digital Therapeutics to Support Recovery Act) was introduced in the U.S. Senate of Congress to make DTx for mental health and substance use disorders covered by the Medicare and Medicaid programs15) In addition, the Centers for Medicare & Medicaid Services ("CMS") has proposed a rule in January 2021 that would provide four years of Medicare coverage for innovative medical technologies with FDA BreakThrough Device designation (The Medicare Coverage of Innovative Technology and Definition of 'Reasonable and Necessary': MCIT/ R&N) for innovative medical technologies with FDA BreakThrough Device designation. 16) However, none of these have been enacted, and the reimbursement system for DTx in public health insurance is still under discussion.

Industry has also made recommendations regarding reimbursement of DTx by public health insurance. In September 2020, the Advanced Medical Technology Association, a US medical device association, issued a white paper titled "Modernizing the Medicare Coverage of Digital Health Technologies," which outlines the following recommendations 17), urging the U.S. government, the U.S. Department of Health and Human Services, and CMS to consider improvements, including the establishment of benefit categories for DTx (either modifying or creating new categories already in place) and improving accessibility through improved reimbursement pathways. It also calls for the establishment of a public-private consortium, led by CMS and FDA, to collaborate with government agencies, private health plans, providers, patients, and others to establish best practices for evaluation of digital health (including DTx) outcome measures related to insurance reimbursement. Specific measures include "quality, health outcomes (clinical effectiveness), patient safety, and cost," and the report recommends the establishment of an evidence framework that developers should submit to CMS.

Reimbursement by private medical insurance

While an increasing number of insurers are separately making DTx eligible for reimbursement, several Pharmaceutical Benefits Managers (PBMs) have created their own "Digital Health Formulary" to systematically advance reimbursement for digital health, including DTx. This is not limited to SaMDs but also includes Non-SaMDs in the formulary if default evidence is accepted, thereby increasing accessibility to digital health broadly. For example, Express Scripts has developed the Evernorth Digital Health Formulary, which includes 15 mobile applications targeting diabetes, hypertension, and other conditions and caregiver care18). CVS Health has also developed the "CVS Health Point Solutions Management" and has selected 12 digital solutions19).

In addition to "clinical efficacy," "security/privacy," and "cost" related to product efficacy and safety, outcome measures unique to digital health that are closely related to daily life, such as "user experience," are evaluated in determining whether to include a solution in the formulary.

Outcome Indicators for Reimbursement in the U.S.

Based on the above, we believe that clinical effectiveness (health outcomes), quality (security/privacy, etc.), cost, and user experience indicators will be key for DTx reimbursement in the US.

Outcome information on clinical efficacy is important information that determines whether regulatory approval and reimbursement are granted20). Efficacy and safety are often verified through randomized controlled trials ("RCTs"), and in the product library published by the Digital Therapeutics Alliance, a trade association of DTx-related companies, many of them had at least one RCT1). In addition, CVS Health has indicated that the results of RCTs are used to evaluate clinical efficacy19).

Regarding security/privacy, the Digital Health Center of Excellence has also published a compilation of guidance and white papers issued by the FDA. For example, the 2018 "Content of Premarket Submissions for Management of Cybersecurity in Medical Devices21), which provides information on what to include in device design and application materials with cybersecurity in mind. It is assumed that these evidences are considered in the evaluation for insurance reimbursement.

In addition, in the U.S., where healthcare costs are high, economic indicators such as cost-effectiveness are important, and development companies are involved and verifying their work for digital health. For example, Freespira (Freespira, Inc., post-traumatic stress disorder, etc.), which received FDA 510(k) clearance, has been verified to reduce emergency treatment and medication costs by over 60% each (reducing total medical costs by $2,280 per year) 22). In addition, Livongo's diabetes management program, which will be included in Express Scripts' formulary, has been reported to reduce per capita monthly medical costs by approximately $88, as verified in a retrospective study23). In addition, Big Health's Digital Sleep Improvement Program, which will be included in the CVS Health formulary, has been shown to reduce annual medical costs per person by $1, 67724).

Thus, the health economic benefits of DTx compared to conventional therapies have been validated (Supplement 1).

Supplement 1: Cost-effectiveness evaluation by ICER

The Institute for Clinical and Economic Review (ICER: Institute for Clinical and Economic Review), which conducts cost-effectiveness evaluations in the United States, published its first cost-effectiveness evaluation for digital therapies in November 202025). The report covers digital health for three opioid use disorders, including DTx (obtaining 510(k) clearance), but for two of the three, the cost-effectiveness assessment itself could not be validated due to lack of evidence. Even for DTx, the only one for which cost-effectiveness could be modeled, it was concluded that long-term cost-effectiveness is low at current prices due to lack of evidence from long-term continuation, among other reasons. (It is classified as C+ (Comparable or Incremental: equal or small contribution to health) in ICER's evaluation matrix.

ICER also concludes that the cost-effectiveness of digital health should be evaluated not only based on short-term evidence of efficacy and safety, but also on 1) sustainability of beneficial clinical effects, 2) impact on healthcare utilization, 3) impact on physician productivity, 4) usability based on physician and patient experience, 5) security, and 6) applicability to diverse patient populations and healthcare systems. The report states that robust evidence should be prepared for: 1) sustainability of beneficial clinical effects, 2) impact on healthcare utilization, 3) impact on physician productivity, 4) usability based on physician and patient experience, 5) security, 6) application to diverse patient populations and healthcare systems, and 7) extension to larger populations.

As the number of DTx approvals increase in the future, cost-effectiveness evaluations by ICER may impact reimbursement prices.

U.S.: Examples of Various Outcome Measures

One outcome measure that is unique to DTx is usability based on user experience.

For example, Express Scripts lists usability as ease of use, tracking, data synchronization, and accessibility, which have been validated by experts26).

In addition, the Digital Health Scorecard27) proposed by Johns Hopkins University to government agencies and others to build a digital health framework in the U.S. defines usability criteria as "the ability to provide patients with various requirements (visual impairment, motor impairment, cognitive impairment, etc.) with In addition, the "Digital Health Scorecard27)" defines usability criteria as "ease of use, measurement effort, and input burden acceptable for patients with various requirements (visual impairment, motor impairment, cognitive impairment, etc.). In addition, it also mentions that expectations from the perspective of healthcare professionals, such as data linkage and burden in clinical settings, should also be fully reflected.

Usability evaluation in the U.S. is required to demonstrate that the solution improves user (patients, healthcare professionals, etc.) engagement and enables continued use from a variety of perspectives.

4-2. Germany

In Germany, the Digital Supply Act (DVG) came into effect in December 2019, allowing DTx, etc. registered in the DiGA (German for digital health app) directory to be used through public health insurance (Figure 2, about 7, 3 million people (about 90%) with public health insurance are eligible for use. The number of users is approximately 73 million (about 90%) of the citizens enrolled in public health insurance). In addition, the fast-track system28) introduced under the DVG is attracting worldwide attention as a system with rapid market access and high business predictability due to the speed from application to decision on registration (within 3 months of application) and the involvement of manufacturers in setting prices in the first year of reimbursement. In addition, various systems such as the Digital Health Applications Ordinance (DiGAV: regulates application procedures and evidence requirements) and the Digital Care and Nursing Modernisation Act (DVPMG: regulates data protection, security, etc.) have been established. The DTx medical development is being actively promoted under these systems29).

Figure 2 Pathway from regulatory application to reimbursement in Germany

As of February 3, 2022, there were 28 products/applications registered in the DiGA Directory under these systems (full registration: 8, provisional registration: 20) .30) To be registered in the DiGA Directory, a product must first meet the requirements for "safety, functionality, quality (interoperability, etc.), data protection and data security. The first step is to comply with the requirements for "safety, functionality, quality (interoperability, etc.), data protection and data security. For example, for equipment safety, acquisition of the European CE Mark is mandatory, and for data protection, compliance with the European General Data Protection Regulation (GDPR) and the Federal Data Protection Act (BDSG) is required. Regarding quality, compliance with the required standards is verified with respect to data interoperability with other digital applications, robustness, user protection, ease of use, healthcare professional support, quality of medical content, and patient safety.

Once the above requirements have been met, verification of clinical effectiveness (positive care effect) will be conducted. However, clinical efficacy is not required at the time of DiGA directory application. If sufficient evidence has not been obtained, reimbursement will be made in the form of provisional registration if an evaluation in the literature or a trial plan for clinical efficacy is approved. In this case, the efficacy must be demonstrated by utilizing real-world data and other data during a 12-month period (maximum 24 months).

Meanwhile, interestingly, the National Association of Statutory Health Insurance Funds (GKV-SV), which is involved in determining reimbursement prices, issued a position paper on DiGA requirements and criteria in January 202131). In it, the GKV-SV expresses concern that the legal framework for regulatory approval and reimbursement of DiGA is not designed to adequately guarantee the requirements of benefit, quality, and economic efficiency. In particular, with regard to a policy unique to Germany whereby manufacturers set the reimbursement price for the first year of provisionally registered products, the committee sees the economic rationality of reimbursing at a high price for products for which sufficient clinical efficacy and utilization have not been verified as an issue, and calls for a review of the free price system32). Table 2 shows the reimbursement prices of products registered in the DiGA Directory for reference. Although it is a basic premise that prices vary depending on the indicated disease, demonstrated efficacy, and other factors, many products were priced at around 500 euros.

Currently, health economic evaluation is not a DiGA registration requirement, but it has been indicated that it may be taken into account during price negotiations28). In order to reach a consensus among citizens/patients and the GKV-SV, outcome measures related to health economics, such as cost-effectiveness, may have an even greater impact on reimbursement pricing decisions in the future33).

Table 2 (Reference) Reimbursement price of German DiGA

Germany: Examples of Various Outcome Measures

In order to examine a variety of outcome measures, individual cases were investigated for clinical effectiveness (positive care effect). Clinical effectiveness can be broadly categorized into "medical effectiveness" and "patient-related structural and process improvements." Demonstration of both items is not required for DiGA registration, but may be advantageous in determining reimbursement prices28). Although differences in indications, effectiveness, etc. need to be taken into account, a relatively higher set price was observed for products that actually validated outcome cases for "improvement in patient-related structures and processes" (Table 2).

  1. (1)
    Medical Effectiveness.

    Medical effectiveness is a treatment effect demonstrated in clinical trials, such as improved health status and quality of life, reduced duration of illness, etc. Based on the information in the DiGA Directory, many of these effects have been verified in multiple RCTs. However, in its guidelines, the Federal Institute for Drugs and Medical Devices (BfArM) allows retrospective studies using real-world data28), and it is expected that more non-RCT studies will be conducted to generate evidence in the short 12-month period of provisional enrollment. 34).

  2. (2)
    Improved patient-related structures and processes

    In the case of the DiGA directory, the following evaluation items (9 items) have been established for the effectiveness of DiGA use related to patients, aiming to evaluate not only the medical effects but also various aspects from the perspective of patients and their families: "adherence", "health literacy", "patient independence", "patient's "health literacy", "patient's "health literacy", and "patient's "health literacy". Outcomes for "adherence," "health literacy," "patient independence," "coping with disease-related difficulties in daily life," and "reduction of treatment-related labor and burden on patients and relatives" were already being used as actual evaluation indicators.

    As a specific example, health literacy (indicator: HLS-EU-Q16) and labor productivity (indicator: WPAI: IBS) are used as outcome indicators in Cara Care (HiDoc Technologies), a treatment application for irritable bowel syndrome35). In addition, vorvida (GAIA), a treatment support application for alcoholism, has been validated to improve self-efficacy (independence) for alcohol consumption (indicator: Alcohol Abstinence Self-Efficacy Scale36). velibra (GAIA), a treatment support application for piafrophobia and other disorders, has shown that it can effectively reduce psychological stress in patients as "reducing treatment-related labor and burden on patients and relatives" (indicator: Brief Symptom Inventory) 37).

    As a national initiative, the Zukunftsregion Digitale Gesundheit (ZDG) project, an initiative of the German Federal Ministry of Health, has targeted several DiGAs, including M-sense Migraine (Newsenselab, migraine), German 38), which is being validated to accelerate the spread of digitization in healthcare.

In this context, the application of digital care services in chronic diseases such as diabetes, migraine, and back pain, as well as in daily care, was examined, with extensive treatment outcomes and opinions collected from patients, healthcare professionals, and caregiving families. Interviews with health care professionals (physical therapists) participating in the project have shown that continuous medical intervention in digital health improves patient accessibility to treatment39). The project is expected to continue until the end of 2022, and the patient and other outcomes collected may be used to consider insurance reimbursement for each product.

Table 3 German DiGA: Outcome examples for

4-3. United Kingdom

In general, in order for a medical device to be reimbursed in the UK, it must obtain the UKCA Mark40), be registered with the MHRA (Medicines and Healthcare products Regulatory Agency), and be recommended for use by the NHS (National Health Service). For DTx, NICE published the Evidence standards framework for digital health technologies in 2018, which is the first to widely promote the medical deployment of digital health technologies in other countries. health medical deployment ahead of other countries. However, there is currently no dedicated pathway for DTx to be reimbursed at the national level, with the regional NHS playing a key role in reimbursement41). The reimbursement system for DTx in the UK is still under review.

Against this backdrop, NICE published the "NICE strategy 2021 to 2026" in April 202142) to address the challenges of rapidly changing health and care, including advances in digital technology. One of the pillars of the strategy is "rapid, robust, and responsive technology assessment," and digital health is positioned as a focus area for the next five years. Specifically, the government is working to improve the accessibility of digital health by launching a Digital Health Office (established in May 2021) to support the development of regulations and evidence standards, and by developing access pathways to new medical technologies.

In addition, several documents on digital health evaluation indicators have been released by public agencies.

The Department of Health, which is in charge of the NHS, issued "A guide to good practice for digital and data-driven health technologies" in January 202143). In it, the evidence requirements for the NHS to adopt digital and data-driven technologies (1) ethics of data use, 2) effectiveness, 3) ease of use and accessibility, 4) technical assurance, 5) clinical safety, 6) data protection, 7) data transparency, 8) cybersecurity, and 9) interoperability) are presented, and development companies are It is recommended that development companies incorporate these into their development strategies.

According to the "Evidence standards framework for digital health technologies" issued by NICE, DTx falls under Tier C (Interventions), which states "(1) Effectiveness (in the case of DTx, RCTs are recommended), (2) Appropriate behavior change In addition to "validity criteria" such as (1) use of appropriate technology, (2) appropriate behavior change, (3) reliability of data and information, (4) continuous data collection showing usage and value, (5) expert review, (6) quality and security, (7) relevance to the current care system, (8) user satisfaction, and (9) equality (accessibility to care), "economic effectiveness criteria" are also presented7) . 7).

It can be said that evidence that satisfies these criteria is required if DTx is to be reimbursed by insurance in the UK.

United Kingdom: Examples of Various Outcome Measures

At NICE, an independent committee composed of citizens, clinical experts, and others prepares various evidence-based documents. For medical devices, including digital health, the committee publishes its views and advice on the technology, evidence, and expected costs as "Medtech innovation briefings.) Although this document does not provide a recommendation for use by NICE, we would like to dig deeper into outcome indicators from the user perspective (patients/caregivers and healthcare professionals) in the UK through this document45), as this information will support the adoption decisions of such technologies in the NHS and healthcare organizations.

  1. (1)
    Outcome measures from patient/caregiver perspective

    KardiaMobile (AliveCor), consisting of a portable atrial fibrillation detection monitor and management app, aims to identify atrial fibrillation from routine ECG recordings. Patients cite the ability to quickly confirm whether arrhythmias are due to atrial fibrillation and the peace of mind of sharing measurements with physicians and other care teams (having a reassuring ally at hand for the patient) as positive benefits46).

    In addition, with DexcomG6 (Dexcom), a real-time blood glucose monitoring system, the British Diabetes Society has noted the potential benefits in reducing diabetes-related anxiety experienced by parents and caregivers of children with diabetes when they are away from their children47).

    Furthermore, Sleepio (Big Health), an app for improving insomnia, mentions reduction in number of hospital visits and waiting time to treatment (improved accessibility) 48) as a benefit.

  2. (2)
    Outcome measures from the healthcare professional's perspective

    Outcome measures for healthcare providers have primarily examined the impact on healthcare resources. Clinical experts commented on myCOPD (my mhealth), a chronic obstructive pulmonary disease (COPD) management app, noting that use of the app reduces medical staff time spent on patient education and encourages patient self-management, thereby reducing the need for medical care and hospitalization49) 49). In addition, as reported by Nakano in Policy Research Institute News No. 64, in Space from Depression (Silvercloud Health), a treatment app for depression, one of the outcome measures is a significant reduction in therapist response time to treatment (about 137 minutes on average)50) 50), 51).

5. Summary and Discussion

5-1. Summary

The following is a summary of the current status of outcome measures related to reimbursement in the U.S., Germany, and the U.K. collected in this survey.

Outcomes related to the essence of DTx, such as usefulness (efficacy and safety) and software quality (data protection, cyber security, etc.), are generally the same in each country, and unique indicators are incorporated on this foundation.

For example, health economic indicators such as cost-effectiveness are set as one of the important outcome indicators in the U.S. and the U.K. In Germany, however, they are not included in the DiGA registration requirements and are only indicated that they may be considered during price negotiations, showing differences in utilization.

What also showed strong characteristics of each country were outcome indicators related to usability/user experience. Especially in Germany and the U.K., various indicators are already being used to consider reimbursement in accordance with the characteristics of DTx (Table 4). For example, Germany emphasizes "patient-centered (including family) values" such as labor productivity, health literacy, and psychological stress of patients, while the UK is unique in that it considers outcome indicators (e.g., response time to treatment) not only from patients and caregivers but also from healthcare professionals' perspective.

On the other hand, in the U.S., outcome indicators (ease of use, accessibility, etc.) that are assumed to be from the perspective of patients and caregivers are incorporated in private medical insurance, but there were not as many examples of specific outcome indicators as in Germany and the U.K. In addition, evaluation indicators for public medical insurance are still under discussion, and proposals are being made by several organizations.

In the future, it is expected that outcome indicators from the user's perspective, which are subject to reimbursement evaluation in each country, will be further developed based on issues arising from the operation of the current system and the evolution of DTx.

Table 4 Examples of outcome indicators from the user's perspective related to insurance reimbursement in each country

5-2. Consideration of the DTx reimbursement system in Japan

Based on the survey results, we will consider the evaluation axis of the Japanese insurance reimbursement system for DTx.

While efficacy and safety play a major role in the consideration of reimbursement prices in Japan, the medical device industry has proposed a new outcome indicator for programmed medical devices including DTx: in addition to efficacy and safety, "social necessity" related to equalization of medical quality and "economic efficiency and efficiency" related to productivity of physicians and others. The proposal includes "social necessity" for equalization of medical care quality and "economic efficiency" for productivity of physicians and others. After discussions at the Chuikyo, the position of equalization of medical technology and support for medical treatment was clarified in terms of reimbursement, and it can be said that these outcome indicators are mainly from the perspective of medical professionals.

DTx is a tool that is expected to improve the quality of medical care not only through its use by medical professionals, but also through its use by patients themselves and the sharing and management of their own health status, including that of their caregivers. Given these characteristics of DTx, we believe that "outcomes from various users' viewpoints" should be considered, not only from the viewpoints of medical professionals. In addition, because outcome indicators from diverse user perspectives are needed to promote the new innovation of DTx, there may be an aspect of actively trying to incorporate such indicators in the U.S. and Europe.

For example, in the U.S., the medical device industry is requesting that a public-private consortium including patients consider outcome indicators for reimbursement of DTx and other digital health products by public health insurance, and in Germany, patient-related outcome indicators are already being used in reimbursement studies. In the U.K., in addition to the "Medtech innovation briefings" described in section 4-3, NICE has also developed "medical technologies guidance," which provides recommendations for the adoption of medical technologies in the NHS, with diverse opinions from patients, caregivers, healthcare professionals, and medical technology experts. In addition to the "medical technology innovation briefings," NICE is required to collect diverse opinions from patients, caregivers, healthcare professionals, medical technology experts, and others in developing the "medical technologies guidance" that recommends adoption of medical technologies in the NHS .) Thus, attempts to incorporate outcomes and opinions from diverse user perspectives are underway in various countries.

Although specific outcome indicators need to be considered individually, taking into account the characteristics of DTx, indications, purpose of use, etc., it may be necessary for Japan to practice diverse value evaluation using outcome measurement tools that have already been used, and to promote prompt institutional review.

Considering the development status of DTx in Japan (Table 1) and the user-oriented outcome measures utilized in various countries (Table 4), it is possible to demonstrate values such as improved labor productivity and health literacy for mental illness, etc. and reduced anxiety of patients and family caregivers for cancer, etc. In addition, as for the reduction of the burden on medical personnel, it would be possible to quantitatively evaluate the supplementation of the shortage of specialized therapists for mental disorders that require treatment based on cognitive-behavioral therapy, and the reduction of time required to understand patient conditions in the case of lifestyle-related diseases.

On the other hand, in order to promote the above, it is necessary to establish a system that reflects the opinions of a wide range of users, including patients, families, and caregivers, in addition to medical professionals, in the evaluation of insurance reimbursement.

In Japan, the "challenge application system," in which the clinical usefulness of a product is reevaluated based on its actual use after it has been covered by insurance, is applied to programmed medical devices. When evaluating the value from various user perspectives, it is desirable to utilize the framework of this system to re-evaluate not only the clinical usefulness but also the value from various perspectives obtained from a wide range of outcomes accumulated in the real world during a certain period of time after the product is listed. Especially in the case of DTx, it is possible to collect a wide range of outcomes for patients, healthcare professionals, etc., in collaboration with other digital tools. However, since patient groups, etc. are not currently included in the preliminary reassessment plan or in discussions on the validity of the collected evidence, it is necessary to establish a system and system that reflects the opinions of patients, etc. in order to appropriately set and assess the "value from the user's perspective.

In response to rapidly evolving digital technology, it is important to promptly establish a system that utilizes current rules and frameworks.

So far we have looked at outcome measures related to DTx reimbursement, but there are some unclear differences in measures in each country as to which outcome measures will be used and how they will be used to determine whether or not reimbursement is possible, including price setting. However, in terms of "user perspective outcomes," for example, it is inferred that patient perspective outcome measures in Germany may have a significant impact on pricing, as they are said to be sometimes favorable for determining reimbursement prices. On the other hand, in the U.K., NICE's guidance giving recommendations for the adoption of medical technology in the NHS stipulates that diverse opinions, including those of patients and caregivers, be taken into account, which may also have an impact on the decision on reimbursement.

As mentioned above, it would be desirable for Japan to re-evaluate insurance reimbursement prices, etc. through a challenge application system, utilizing outcomes from diverse user perspectives accumulated in the real world, but discussions are needed on which process these outcomes should be evaluated in the future.

On the other hand, the application of health economic indicators (cost-effectiveness) to reimbursement prices in Japan is currently limited to products with a large market size or significantly high unit costs. For DTx, which does not meet these criteria, it is not realistic for development companies to independently calculate and present health economic efficiency as a requirement for insurance reimbursement. However, since DTx is expected to curb the risk of developing serious diseases and increasing the efficiency of medical care by improving treatment outcomes, demonstrating the impact of DTx on overall medical resources in Japan, such as social security costs, will be one key to promoting the medical deployment of DTx in Japan. In Germany, studies are being conducted to evaluate the medical economic efficiency of DTx using public funds33). In Japan, it may be necessary for the public and private sectors to collaborate to evaluate the medical economic efficiency of DTx for psychiatric and lifestyle-related diseases, for which DTx development is already underway, in order to promote the medical development of DTx. This paper focuses on DTx.

6. Summary

This paper focuses on DTx and discusses the evaluation axes for DTx reimbursement in Japan, based on the current status of reimbursement and outcome indicators in the United States, Germany, and the United Kingdom.

The dramatic progress in the discussion of DTx reimbursement in Japan over the past year can be attributed to the tireless efforts of various stakeholders. The most important thing in brushing up the reimbursement system in the future is to be able to set appropriate outcome indicators based on the characteristics of DTx as evaluation items, and to be able to evaluate their value appropriately by the stakeholders concerned. Within this framework, it is important that industry, government, academia, and the private sector work together to carefully examine specific outcome indicators and evaluation criteria for individual DTx, taking into account examples accumulated in Japan and overseas and trends in other countries. Furthermore, considering the future global development of DTx, we should also take into consideration the development of internationally harmonized regulations regarding the criteria for outcome indicators and quantification methods common to each country. In such a case, it is highly significant for the pharmaceutical industry, which is involved in the development of user-oriented development and international institutional design of pharmaceuticals, and which also promotes the consideration of various values, to participate in these discussions.

In the future, digital technology will evolve at a speed far beyond our imagination. However, no matter how much digital technology advances, it will be people who use the technology, and the technology must be beneficial to users. To realize this, we believe that one of the important perspectives in the development of DTx and other technologies will be to pursue not only efficacy and safety, but also user-centered value and to build evidence from highly reliable outcomes. Furthermore, the "sense of security" that the "value" embodied in such a perspective will be appropriately evaluated under an appropriate system will lead to the promotion of the DTx industry in Japan.

In order to realize such a future, we hope that various stakeholders in industry, government, academia, and the private sector, including the pharmaceutical industry, will cooperate in discussions to foster a new form of medicine and healthcare that utilizes digital technology.

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