Policy Research Institute page Consideration of Outcome Indicators for Reimbursement of Digital Therapeutics (DTx) -Toward a Diverse Value Assessment Based on the Latest Trends in Europe and the U.S.A.

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Advancing digital technologies are transforming the "treatment-centered" nature of medicine and healthcare into a more individualized and stratified involvement as well as expanding the "entire life course from prevention to prognosis". In the midst of these changes, digital therapies (DTx), which provide treatment using digital technology, are being deployed in countries around the world, and various products are being developed in Japan as well. Japan has only just begun to develop a system for this new technology, but discussions in various quarters have led to significant progress in the DTx insurance reimbursement system. In this issue of the Newsletter, we introduce the evaluation axes for DTx reimbursement in Japan based on the latest developments in Europe and the U.S., as discussed in Policy Research Institute News No. 65*1.

Introduction

Since the U.S. Food and Drug Administration (FDA) regulatory approval of a blood glucose management application (BlueStar: Welldoc Inc.) in 2010, several DTx have appeared in countries around the world. For example, in the U.S., more than 10 DTx products have already received FDA clearance/approval*2 and in Germany, 28 products/applications have been approved for clinical use, including provisional registration for continued review*3. In addition, more and more pharmaceutical companies are entering the market and various products are under development ( Table 1 ).

Table 1 Trends of pharmaceutical companies in DTx development in Japan
Source: Prepared by the author based on information in each company's press release (as of February 2022)

As DTx are being introduced one after another, there is an urgent need for Japan to develop and establish various systems to support the medical development of DTx. In FY2021, the Central Social Insurance Medical Council (Chuikyo) held discussions on the reform of the insured medical material system in FY2022, and included a section on "Evaluation of Programmed Medical Devices". The Council also clearly stated that the evaluation axis for reimbursement should take into account values other than efficacy and safety that reflect the characteristics of the device*5.

This paper therefore focuses on the United Kingdom, where a variety of values for pharmaceuticals are being evaluated, in addition to the United States and Germany, which have a large number of DTx approved/approved items, and examines the evaluation axis for DTx reimbursement in Japan based on the current status of reimbursement and outcome indicators for DTx in each country.

Reimbursement Pathways and Outcome Measures in Each Country

1. United States

There are two major types of public health insurance in the U.S., "Medicare" for the elderly over 65 years old and the disabled, and "Medicaid" for low-income people, but most U.S. citizens are not eligible for these benefits and use private health insurance*6. This makes business predictability more complicated compared to Japan. 6 This makes business predictability more complex than in Japan, and it is important to envision from the development stage what kind of business plan to pursue and what outcome measures will be necessary to achieve it.

＜Reimbursement by public health insurance

The FDA is vigorously designing a new system for programmed medical devices (SaMD), including DTx, but it is not fully linked to public health insurance, and DTx used directly by patients is not currently covered under the benefit category*7. 7 However, against the backdrop of the rapid penetration of digital technology, discussions on the reimbursement of DTx by public health insurance are accelerating.

For example, in March 2020, legislation was introduced in the U.S. Senate to make DTx for mental health and substance use disorders covered by the Medicare and Medicaid programs*8 and the Centers for Medicare & Medicaid Services (CMS), a government agency However, none of these have been enacted, and the DTx reimbursement system for public health insurance is still under discussion. Some drug benefit management companies are working on their own.

＜Reimbursement by private health insurance

Several drug benefit management companies have developed their own "Digital Health Formulary" to systematically advance reimbursement of digital health, including DTx. This is not limited to SaMDs, but also covers Non-SaMDs as long as 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 diseases such as diabetes and caregiver care.10 CVS Health has developed the "CVS Health Point Solutions Management" and has selected 12 digital solutions*11.

In determining whether or not to include a solution in the formulary, "clinical effectiveness," "security/privacy," "cost," and "user experience," which are all related to product efficacy and safety, are evaluated.

＜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 experts*12.

In addition, the Digital Health Scorecard*13 proposed by Johns Hopkins University to government agencies and others to establish a digital health framework in the U.S. defines usability criteria as "the ability to provide patients with a variety of requirements (e.g., visual impairment, motor impairment, cognitive impairment) with The Digital Health Scorecard*13 defines usability criteria as "ease of use, measurement effort, and input burden acceptable for patients with various requirements (e.g., 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.

In addition, the Institute for Clinical and Economic Evaluation (ICER) has identified usability based on physician and patient experience, impact on physician productivity, and applicability to diverse patient populations and health care systems as indicators for evaluating the cost-effectiveness of digital health, and states that robust evidence for these factors should be prepared. 14.

2. Germany

In Germany, the Digital Supply Act (DVG) came into effect in December 2019, allowing DTx and other devices registered in the DiGA (German for digital health app) directory to be used through public health insurance ( Figure 1 ). Furthermore, the fast-track system*15 introduced under the DVG is attracting worldwide attention as a system with quick 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.

Figure 1 Germany: Pathway from regulatory application to reimbursement
Source: The Fast-Track Process for Digital Health Applications (DiGA) accounting to Section 139e SGB V*15 Figure added by author.

To be included in the DiGA Directory, a product must meet the following requirements: safety, functionality, quality (interoperability, etc.), data protection, and data security. After these requirements are met, the clinical efficacy (positive care effect) is verified. However, clinical efficacy is not required at the time of DiGA Directory application, and if sufficient evidence has not been obtained, reimbursement can 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 using real-world data and other data during a 12-month period (maximum 24 months).

＜Examples of Various Outcome Measures

Clinical effectiveness can be broadly classified into "medical effectiveness" and "patient-related structural and process improvements. Although demonstration of both is not required for DiGA registration, it is said to be sometimes advantageous in determining reimbursement prices*15. Although it is necessary to consider differences in indications, effectiveness, etc., a relatively higher set price was observed for products that actually verified "patient-related structural and process improvements" (Table 2). were found ( Table 2 ).

Table 2 Reimbursement prices for German DiGA
Source: prepared by the author based on DiGA Directory*3 (viewed February 3, 2022)
Note: Most products have a 90-day use period

(1) Medical benefits

The "medical benefits" are treatment effects demonstrated in clinical trials, such as improved health status and quality of life, shorter duration of illness, etc. Most of them have been validated through multiple randomized controlled trials, but the Federal Institute for Drugs and Medical Devices (BfArM) also allows validation through backward-looking studies using real-world data*15.

(2) Improvements in patient-related structures and processes

The endpoints for the effectiveness of DiGA use as it relates to patients are set out in Table 3, with the goal of evaluating a variety of patient and family perspectives. Outcomes for "adherence," "health literacy," "patient independence," "coping with disease-related difficulties in daily life," and "reduction of treatment-related labor and burden for patients and relatives" were already used as actual evaluation indicators. Specific examples include the following.

Table 3 Outcome examples for "patient-related structural and process improvements"
Source: prepared by the author with reference to DiGA Directory*3 (viewed February 3, 2022)

3. the United Kingdom

The United Kingdom has been ahead of other countries in the medical deployment of digital health with the release of the "Evidence standards framework for digital health technologies" by the National Institute for Health and Care Excellence (NICE) in 2018. In addition, the Ministry of Health, which is in charge of the National Health Service (NHS), published "A guide to good practice for digital and data-driven healthcare" in January 2021. The Department of Health, which is in charge of the National Health Service (NHS), issued "A guide to good practice for digital and data-driven health technologies" in January 2021, setting out the evidence requirements (effectiveness, clinical safety, ease of use, accessibility, etc.) for the NHS to introduce digital and data-driven technologies*20. developers are encouraged to incorporate these requirements into their development strategies if they seek reimbursement for DTx.

Thus, while documents on evaluation indicators are published by public agencies, there is currently no dedicated pathway for DTx to receive reimbursement at the national level, with the local NHS playing a major role in reimbursement*21.

＜Examples of Various Outcome Measures

NICE has an independent committee composed of citizens, clinical experts, and others that prepares a variety of evidence-based documents, and for medical devices, including digital health, the committee publishes views and advice on the technology, evidence, and anticipated costs in "Medtech innovation briefings. Although this document does not provide a recommendation for use by NICE, we would like to explore in depth the outcome measures from a user perspective in the UK through this document, as this information will assist the NHS and healthcare organizations in making decisions on the adoption of this technology*23.

(1) Outcome measures from the perspective of patients and caregivers

KardiaMobile (AliveCor), which consists of a portable atrial fibrillation detection monitor and management app, has been cited as a positive effect in that it allows patients to quickly confirm whether or not an arrhythmia is due to atrial fibrillation and to share measurements with their doctors and other care teams*24 The system is also used to monitor the patient's blood glucose levels.

In addition, DexcomG6 (Dexcom), a real-time blood glucose monitoring system, has been cited by the British Diabetes Society as having potential benefits in reducing diabetes-related anxiety experienced by parents and caregivers of children with diabetes when they are away from their children*25.

In addition, Sleepio (Big Health), an app for improving insomnia, mentions reduced number of hospital visits and waiting time for treatment as a benefit*26.

(2) Outcome indicators from the perspective of healthcare professionals

For myCOPD (my mhealth), a chronic obstructive pulmonary disease (COPD) management app, clinical experts cite benefits such as reducing medical staff time spent educating patients and encouraging patient self-management, reducing the need for medical care and hospitalization*27. In addition, 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)*28*29.

Consideration of the DTx reimbursement system in Japan

In the examination of reimbursement prices in Japan, the medical device industry has proposed "social necessity" related to equalization of the quality of medical care and "economic efficiency and efficiency" related to the productivity of physicians and others as new outcome indicators for programmed medical devices, including DTx. After discussions at the Chuikyo, the position of equalization of medical technology and support for medical treatment in terms of reimbursement was clarified, but we believe 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 the utilization of accumulated patient information by medical professionals, but also through its use by patients themselves and the sharing and management of their own health status, including by their caregivers. Given these characteristics of DTx, we believe that "outcomes from diverse user perspectives" should be considered, not only from the perspective of medical professionals. In addition, we believe that the need for outcome indicators from diverse user perspectives to promote the new innovation of DTx is one aspect of the active efforts to incorporate such indicators in Europe and the United States.

While individual consideration of evaluation axes in Japan is necessary, taking into account the characteristics of DTx and indications, etc., it may be necessary to practice diverse value evaluation utilizing outcome indicators that have already been used and to promote prompt institutional review. For example, considering the domestic development status of DTx ( Table 1) and outcome indicators from the user perspective in each country ( Table 4), it may be possible to demonstrate values such as improved labor productivity for mental illness, etc., improved health literacy, and reduced anxiety of patients and families about cancer, etc. In addition, as for the burden on medical personnel, we believe that it can be quantitatively evaluated in terms of supplementing the shortage of specialized therapists in the treatment of mental disorders based on cognitive-behavioral therapy and reducing the time required to assess the condition of patients with lifestyle-related diseases.

Table 4 Examples of outcome indicators from a user perspective related to reimbursement in each country
Source: prepared by the author based on information from each country
Note: Bolded figures in the table are outcome indicators for which specific examples were recognized in this study.

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 is covered by insurance, applies to programmed medical devices as well. When evaluating the value from various user perspectives, it is desirable to use the framework of this system to re-evaluate not only the clinical usefulness but also the value from various perspectives accumulated in the real world over 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 and healthcare professionals by linking 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. For rapidly evolving digital technologies, it is important to quickly develop a system that utilizes current rules and frameworks.

Summary

The dramatic progress in discussions on DTx insurance reimbursement in Japan over the past year is the result of tireless efforts by all stakeholders. We believe that the most important thing in brushing up the insurance reimbursement system in the future is that appropriate outcome indicators based on the characteristics of DTx can be set as evaluation items, and that their value can be appropriately assessed by the stakeholders involved. To this end, it is important that the public and private sectors work together to examine specific outcome indicators and evaluation criteria for individual DTx, while taking into account domestic and international examples and trends in other countries. Furthermore, considering the future global expansion of DTx, efforts should also be made to establish internationally harmonized regulations regarding common criteria for outcome indicators and quantification methods in each country. In this regard, it is highly significant for the pharmaceutical industry, which is involved in user-oriented development and international institutional design of pharmaceuticals, as well as in the consideration of diverse values, to participate in these discussions.

We hope that the pharmaceutical industry and other stakeholders will cooperate in furthering these discussions.

( Junya Tsujii, Senior Researcher, Pharmaceutical and Industrial Policy Research Institute)

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