Revelation

Vietnamese Organization for Rare Diseases

An estimated 11,000 rare diseases currently exist (according to Rare-X), impacting nearly 400 million individuals (also called people living with rare diseases, or PLWRD). PLWRD faces multiple hurdles, such as limited diagnostic tools and approved therapies. Nevertheless, the key issue remains the lack of access to accurate information about the disease, support communities, and the post-diagnosis experience.

According to Canadian Organization for Rare Disorders (CORD) statistics, around 62% of PLWRDs surveyed reported that their pediatricians did not provide enough information about rare diseases. Conversely, when given sufficient information, at least 40% of PLWRD have difficulty understanding their doctor's advice. Moreover, over two-thirds of patients are unaware of where to seek information about their disease, aside from relying on medical staff for support.

With these figures, it is evident that the absence of information and a defined patient pathway contribute to the numerous psychological challenges encountered by PLWRD. As Dr. Schildkrout, assistant clinical professor of psychiatry at Harvard Medical School, said, "they may also feel a sense of injustice, hopelessness, or even despair."

In Vietnam, our country is in the initial stages of addressing rare diseases, so comprehensive data is not fully accessible. An approximate estimation from our Ministry of Health indicates around 6 million people are affected by rare diseases, whereas there are just 125,000 physicians, most of whom are not specialized in treating these illnesses. As a result, over 20% of the interviewed patients who joined our mini-survey had knowledge about the necessary treatment or post-diagnosis care, mainly acquired from fellow patients. Assumption: more than 2.5 million children living with rare diseases and their parents in Vietnam do not have enough awareness about specific rare diseases (based on our survey and estimation based on the CORD statistic). This problem stems from a number of factors, including a lack of "easy-to-use" information resources, a shortage of doctors qualified to practice medicine in accordance with standards of care, poorly engaged doctors in their patient relationships, etc.

Thus, we observe that the issue of inadequate awareness and support for patients is a global phenomenon, prevalent not only in advanced countries like Canada but also notably challenging in developing countries with lower average incomes like Vietnam. To enhance the standard of patient care and the overall quality of life for patients, it is crucial for patients and their support network (as caregivers, physicians, social workers, etc.) to have a comprehensive understanding of the patient journey. This understanding should encompass information about the particular illness, where to access medical equipment and assistance, and how to reintegrate into society post-treatment.

Revelation is a mobile app platform designed to connect 3 main groups involved in rare diseases: (1) patients and families, (2) medical staff and researchers, and (3) pharmaceutical companies and medical equipment suppliers (referred to as companies). Its key features include: Updated health information, Survey and statistical platform, Donation transparency, and Information on community integration programs.

Features include:

1. Updated medical information will be available on Revelation platform for all parties. This information will include basic details about the disease, steps for diagnosis, available treatments (including clinical trials and options for participation), and information about medical professionals in various treatment areas. All the information provided will adhere to the following principles:
   1. Scientifically verified (for documents we use) and aligned with standards of care (for medical professionals we connect)
   2. Appropriate for different audiences
   3. Widely used and applicable worldwide.
2. Survey and statistical platform: Questionnaires from organizations and countries are used for the following purposes: (1) developing patient registration programs, (2) pre- and post-treatment assessments, (3) surveying patients based on health economic capabilities and unmet needs, and (4) assessing the feasibility and application of new medical equipment based on the needs of patients and doctors. All surveys are guaranteed
   1. Confidential and follow Vietnamese privacy rules,
   2. Questionnaires are adjusted to fit the Vietnamese context while maintaining international consistency, and 
   3. The survey targets an appropriate audience. 
   4. The survey results will be analyzed at a basic level so that all parties have a common understanding of the current issues.
3. Donation transparency: Revelation ensures transparency in donations and verifies the eligibility of vulnerable individuals based on factors such as genuine need, suitability for support solutions, and tracking the impact of support on the recipient's quality of life. Moreover, individuals also receive support from a team of lawyers to ensure that the entire process, from receipt to disbursement, complies with regulations in Vietnam."
4. Information on community integration programs: Revelation offers skills training programs tailored to each patient's unique abilities, including: (1) Courses for self-care at home, school, and work, (2) Computer skills training to improve job prospects, (3) Various other educational programs. These programs have some characteristics that are available to all patients free of charge, but they need to pay attention, and a willingness to actively engage and participate is necessary.

Revelation works on the principle of "to give and to take" based on core values of empathy, sharing, and transparency. Therefore, at the present time, it is more important to optimize and integrate available tools and resources than to rely too heavily on advanced technologies such as artificial intelligence or machine learning.

Revelation is currently being used in the neuromuscular disease community and has shown promising initial results.

Our solution primarily focuses on aiding those lacking access to accurate information or sufficient awareness regarding a specific rare disease. This demographic comprises (I) patients and families (approximately 75% lack comprehensive information) and (ii) healthcare professionals and researchers. In addition, as an accompanying synergistic effect, we also support "companies" through Revelation's features.

Details impact of our solutions

• For patients and families: they can now access medical information and support faster, more accurately, and more confidently through Revelation. This platform provides verified and tested information sources, legal support, and evaluation of appropriate medical equipment to avoid misuse and illegal fundraising.
• For medical professionals and researchers: In Vietnam, there is a significant knowledge gap between doctors and patients, and communication time is very short. This results in a lack of coordination and poor understanding, reducing care efficiency. With Revelation, healthcare workers can communicate with patients using a pre-calculated patient journey, leading to active engagement and better understanding of patients' needs. This allows for the development of new ways to enhance the current care process and generate research ideas.
• For companies: With Revelation's support, companies gain insights into customer needs and spending capacity, leading to more tailored product designs and reduced research and promotion costs. 

Revelation continuously updates and prioritizes the needs of the target population for timely support.

Our solution is applied for Neuromuscular Disorder Communities and medical professionals with in this area. At least 35 patients and more than 10 healthcare provider received benefits from our platform.

Our application platform may seem similar to a social network, a telemedicine application, an information portal, and an e-commerce platform at first glance. However, we have significant differences based on the following criteria: (1) Privacy security, (2) Scientificity; and (3) Authenticity.

Compared to social networks or information portals: The information posted on Revelation has been evaluated and approved by medical organizations and experts to ensure accuracy and scientific validity (Scientificity). We also ensure the authenticity of medical information and other supporting details (Authenticity). Additionally, patient and medical staff information is encrypted according to Vietnam's information security law to maintain confidentiality (Privacy security).

In comparison to telemedicine applications: Revelation not only connects patients with medical experts but also facilitates connections between experts to create a virtual multidisciplinary treatment board (MDT board) to enhance the quality of medical care for patients (Scientificity). Moreover, Revelation provides access to systematically arranged resources for doctors, raising the level of medical staff to meet international standards of care (Authenticity).

In contrast to an e-commerce platform, with Revelation, suppliers' products are evaluated for medical suitability by experts, particularly those in the field of physical therapy and rehabilitation.

With Revelation, patients and physicians's habits will be changed, especially their knowledge of how to engage and connect with each other. On the other hand, the misleading information will reduce its impact, so as a result, the quality of healthcare can be better.

We are presenting for The Amgen Prize with a product that has been tested on a small community and has shown positive initial results. However, we know that there are still many challenges ahead and many improvements needed to enhance our application. Participating in this competition will hopefully help us better understand and address the three key challenges that await us:

• Financial: While Revelation is free for individual users, the current budget to maintain its operations mainly comes from initial capital sources and angel sponsors. In order to expand to other community groups or target B2B customers in the future, we need more financial support to add additional features. If we succeed in convincing the judging panel, we plan to allocate the reward in the following manner: 50% towards building a better data storage and retrieval system to meet future user needs, 30% for direct patient support activities through matching grants (or co-payment methods) that contribute to improving diagnosis, treatment, or integration capabilities, 10% reserved for activities to enhance the capacity of healthcare providers; and 10% for general operations.
• Technical: Revelation is a real-time patient registration tool that contains a wide range of medical and social information. With a large amount of data, we hope to have the support of experienced experts in the field of rare diseases to better understand ways to overcome the limits of science, technology, and construct a database robust enough to support research, inform policies, and contribute to enriching knowledge about rare diseases.
• Affiliate Network: Recognizing that combating rare diseases necessitates collective efforts transcending individual countries or organizations, our presence at The Amgen Prize is meant to foster connections with diverse entities, individuals, and organizations. We aspire for Revelation to assist organizations, solicit feedback for enhancing existing features, and gain fresh perspectives our team may have overlooked.

In order to carry out this project, we recognize that it must address the needs of the community. Understanding these needs requires a comprehensive study or spending significant time with the community. We have chosen to involve individuals from the community to conduct this project. Our process and organizational structure ensure that everyone's expertise and abilities are appropriately empowered. Our team includes:

• Project leader: Dr. Do Phuoc Huy, overseeing foreign affairs and operational management. As a PLWRD, Dr. Huy also empathizes with the challenges faced by patients and their families dealing with rare diseases.
• Professional Team: Consisting of 4 individuals from the Medical Board and the Scientific Advisory Council. Their role is to curate medical information tailored for diverse audiences and guarantee its accuracy. Additionally, they work with the community team-lead to develop relevant research campaigns that reflect the needs of patients and families while maintaining scientific rigor.
• Community team-lead: comprising 4 patients and families from the Duchenne muscular dystrophy and Spinal Muscular Atrophy communities, this group is tasked with surveying and providing a comprehensive understanding of the needs, characteristics, and appropriate approaches of neuromuscular diseases. This group plays a crucial role in bridging the gap between Revelation and the community, contributing to the practical implementation of solutions, and evaluating their effectiveness afterward.

We operate according to the following principles: evidence-based, democratic, and patient-centered. Specifically:

• Evidence-based: prioritizing community-sourced needs through surveys and basing solutions on statistical data or established scientific findings.
• Democracy: our core team of 9 includes 4 medical professionals, 5 patients, and caregivers. Decisions are made through voting, ensuring over 50% agreement from patients and families and emphasizing community-driven initiatives.
• Patient-centered: tailoring plans to community feedback while addressing individual needs, especially critical ones. From budgeting to operational processes, we always have contingency plans in place for emergencies.

We are a product of Vietnamese Organization for Rare Diseases (VORD) - a non profit social enterprise. All staff members, resources come from the VORD.

The Revelation's goal will align with the goal of the Vietnamese Organization for Rare Diseases, especially for these goal:

1. All stakeholders in Vietnam have good awareness about the life of a PLWRD.

2. Patients and physicians in Vietnam understand how to diagnose and manage a specific rare disease.

3. Connect patients not only in Vietnam but also around the world to share experiences about living with a specific rare disease.

That is the long-term vision of Revelation. But in the short- term, we have some key goal with impact metrics and impact evaluation metrics.

Impact metrics:

Revelation mainly works as a connection platform with "easy-to-use" information so "the number of patients approach the pre-defined patient's journey". 

Impact evaluation metrics:

Beside that, Revelation also have impact on the physicians's habit, so to enhance the quality of care for the patients, we also evaluate the affect of Revelation on the reduction of work load for healthcare provider in diagnose or manage the patients. Beside that we also estimate how our platform and data on its support researcher finding their research question. All metrics above can improve the quality of life for the patients in the future.

Our Theory of Change demonstrated as the diagram below

We have 9 full time staff members (5 are patients and families, 4 physicians and researchers). To build the platform, we have 4 part-time staff members (they are all patients)

For the researchers and physicians, we have 3 years working together.

For the patients and families, we worked with each other for around 3 years until now. 

For the solutions, we take around half year for the idea and turn it into reality product for 1 year and a half.

Our process and organizational structure ensure that everyone's skills and abilities are appropriately empowered. This means that regardless of their background, those willing to support Rare Diseases patients can join us. Depending on their capabilities and the current budget, they can engage with our solutions as full-time employees, part-time workers, or volunteers.

To date, over half of our full-time staff members are individuals from vulnerable backgrounds. We have established regulations and programs to ensure they can advocate for their safety, equity, and respect. Additionally, we foster collaboration among all employees based on their unique skills, ensuring that no one feels isolated within their respective teams.

Finally, we will carefully consider every idea to enhance the patient's quality of life. Monthly, our solutions team convenes an advisory board to generate fresh ideas for tackling emerging issues. All are encouraged to contribute insights from their experiences. These advisory meetings serve our goal of finding ways to enrich each person's life.

Our business model based on social business model canvas as picture below

Please take note of the following text:

Our users now include patients and their families, physicians, and researchers. Currently, they use Revelation for free. We do not receive money from these types of users, because:

• We want them to try our solutions and provide feedback or insights to help improve the solution over time. This is the most suitable method to encourage community participation in all our activities.
• We want stakeholders to understand the daily challenges faced by patients. This is the only way to ensure that the patient journey can be applied in their practice.

In the future, we anticipate having a budget to expand into other areas or specific diseases using the following resources:

1. Funding from pharmaceutical companies and other medical equipment vendors. These companies annually allocate budgets for market research or evidence-gathering to prove the health-economic effectiveness of their therapies. Some of them do not see a direct return on investment. Our solutions aim to support these companies in understanding more about their future customers or the barriers they need to overcome in establishing products. This is the first resource we are targeting in the near future.
2. Corporate Social Responsibility: This is the second resource we aim to tap into to increase our budget.
3. Revenue from patient products: This is the most valuable resource. Our "students" from the community integration feature will return and utilize their knowledge or abilities to support our system. This will help us reduce costs and demonstrate to society that patients with rare diseases are as useful as anyone else.