IndoUSrare Patient Concierge

Indo US Organization for Rare Diseases (DBA IndoUSrare)

Globally, an estimated 400 million people suffer from rare diseases, with around 350 million remaining undiagnosed. Of the 11,000 known rare diseases, less than 5% have approved treatments. Access to these existing therapies and ongoing research is heavily skewed towards Western countries, leaving large segments of the global population underserved. This disparity persists despite the significant progress made under the Orphan Drug Act in the USA, which has facilitated the development of over 1,000 orphan drugs in the past 41 years. Thus, global collaboration is essential to enhance accessibility and affordability for both current and future therapies.

The Indian subcontinent, home to approximately a quarter of the world’s population, illustrates the profound challenges faced in accessing diagnostics, therapies, and supportive care for rare diseases. India, with its 1.4 billion people, has an estimated 70 million rare disease patients. However, there are only 12 Rare Disease Centers of Excellence (COEs) in India, creating significant geographical and logistical barriers for patients seeking the latest diagnostics and specialized care. The situation is further complicated by the fact that nearly two-thirds of India’s population resides in rural areas, including tribal communities in remote locations. This rural demographic faces severe access issues to specialized care for rare diseases.

Language barriers compound these access issues, as many educational and medical resources are unavailable in local languages, further hindering patients from obtaining reliable information and expert advice. This exacerbates the challenges rare disease patients face in accessing accurate diagnoses, effective treatments, and comprehensive supportive care. In India, the average diagnostic delay for rare diseases can extend to several years, primarily due to these systemic challenges and the limited availability of trained healthcare professionals.

The global statistics on rare diseases highlight a stark need for improved infrastructure and more localized resources, particularly in regions like the Indian subcontinent. Advanced technological solutions such as telemedicine, which has gained prominence during the COVID-19 pandemic, could play a crucial role in bridging these gaps. By leveraging telemedicine, remote consultations with specialists become feasible, providing rare disease patients in rural and underserved areas with access to expert care without the need to travel long distances.

Furthermore, enhancing the training of local healthcare providers in rare disease diagnosis and management is essential. Establishing more Centers of Excellence, especially in rural and underserved areas, would ensure that patients have better access to specialized care. Policy changes are also needed to prioritize rare disease care and facilitate the availability of treatments and diagnostics.

Addressing these combined factors is imperative for ensuring equitable access to rare disease care. This includes the development of infrastructure, the provision of resources in local languages, and the integration of technological advancements. By doing so, we can significantly improve the healthcare outcomes and quality of life for rare disease patients globally. Ensuring that all patients, regardless of their geographic location or socioeconomic status, have access to accurate diagnoses, effective treatments, and comprehensive support is a critical goal that requires concerted global efforts.

Our tech-enabled Patient Concierge is designed to provide essential information and support to rare disease patients and their caregivers, including those who are still undiagnosed. This innovative service ensures that patients receive comprehensive, personalized assistance, leveraging advanced technology and a robust network of rare disease stakeholders. The IndoUSrare Patient Concierge offers personalized assistance to rare disease patients by:

• Providing credible information about rare diseases, treatments, support groups, and clinical trials.
• Connecting patients with expert advice and facilitating virtual consultations.
• Offering continuous support through various communication channels like email, SMS, audio, and video calls.

How it works

• Request Support: Patients or caregivers fill out a simple request form on our website, sharing details about their condition and giving consent to share information with our partners. This form is now available in over 100 languages to support patients from all parts of the globe
• Contact and Collect Details: Our team contacts the patient through their preferred communication method (email, SMS, audio, or video call) to gather more details about their needs.
• Gather Information: We tap into the expertise of our network of doctors and experts and trusted partners to find accurate and up-to-date information relevant to the patient's condition.
• Provide Personalized Assistance: We refer patients to expert doctors, arrange virtual consultations, and offer ongoing support until the patient’s needs are met.

• Multi-Channel Communication: Patients can choose how they want to communicate with us, making our service accessible to everyone, regardless of location or technological ability.
• Personalized and Continuous Support: We ensure that each patient receives ongoing, tailored assistance, enhancing their access to critical healthcare resources and expert guidance.
• Strong Network of experts and resources

The IndoUSrare Patient Concierge is dedicated to serving rare disease patients and their families, focusing particularly on those in low- and middle-income countries (LMICs), where access to healthcare resources is often severely limited. Globally, it is estimated that 300 million people suffer from rare diseases, with nearly 70 million in India alone. These patients frequently face numerous challenges, including delayed or incorrect diagnoses, a lack of available treatments, and inadequate support systems. For instance, in India, the average diagnostic delay for rare diseases can extend up to seven years, often due to a shortage of trained healthcare professionals and insufficient healthcare infrastructure.

The IndoUSrare Patient Concierge addresses these critical issues by fostering cross-border collaborations between rare disease stakeholders worldwide, including healthcare providers, researchers, and patient advocacy groups. Our goal is to reduce healthcare disparities by accelerating the diagnostic journey and improving access to therapies and standard care, irrespective of geographic location, language, or socioeconomic status. This is crucial as more than 70% of rare diseases are genetic in origin, and early diagnosis can significantly impact the effectiveness of treatment and the overall quality of life for patients.

One of our key objectives is to ensure equitable access to verified and validated information on rare diseases, clinical trials, and care navigation. Currently, more than 90% of rare diseases lack an FDA-approved treatment, highlighting the urgent need for increased awareness and accessibility to clinical trials that could offer potential treatments. By providing reliable information and connecting patients with experts worldwide, we aim to empower patients and their families with the knowledge and resources they need to manage their conditions effectively.

Moreover, the IndoUSrare Patient Concierge aims to build a sustainable ecosystem for supporting rare disease patients and their families in LMICs. Through the use of a tech-enabled platform, we aim to bring a paradigm shift in access to diagnostics and care for rare disease families. Earlier, the expanded use of telemedicine has proven invaluable during the COVID-19 pandemic, offering a model for extending specialist care to remote and underserved areas. This includes creating an expanding network of healthcare providers trained in rare disease diagnosis and management, industry representatives with a focus on rare diseases, and disease-specific support groups for patients and families. Together, these can lead to lasting improvements in healthcare outcomes and quality of life for rare disease patients in these regions.

In conclusion, the IndoUSrare Patient Concierge is committed to bridging the gap in rare disease care for patients in LMICs. By accelerating diagnosis, improving access to treatment, and establishing sustainable support systems, we aim to transform the landscape of rare disease care globally, ensuring that every patient, regardless of their geographic location or socioeconomic status, has the opportunity to receive the best possible care and support.

Our solution has already progressed beyond the prototype and pilot phases. We have built and tested our tech-enabled Patient Concierge platform through which we have successfully served 120 rare disease patients and their caregivers. The requests have come from 15 different countries. While most requests have come from patients and families of Indian origin residing in the Indian subcontinent or other countries, we are now getting more requests from Africa and the Middle East. We have also supported families in North America, Europe, and Australia.

We have provided verified information, expert referrals, and support to a growing user base. Our accomplishments include establishing a robust network of partners and experts in various regions, enhancing our platform’s capabilities, and receiving positive feedback from beneficiaries, indicating a strong impact and demand for our services.

Our solution, the tech-enabled IndoUSrare Patient Concierge, combines advanced technology with a robust network of rare disease experts to provide personalized, comprehensive assistance to patients and caregivers in a fully unified user-friendly package.

Existing concierge services are limited by their ability to cater to diverse populations with language, communication, and cultural differences being major barriers. Our solution works to enable a seamless platform with a patient-friendly interface that minimizes these barriers for underrepresented groups. This approach has the potential to catalyze broader positive impacts in the rare disease space by setting a new standard for patient support and care coordination. This can drive systemic changes, encouraging cross-border collaborations, data sharing, and innovation in the development of rare disease care. Our solution addresses the immediate needs of rare disease patients while also having the potential to transform the space by fostering a more inclusive, patient-centered approach.

We have partnered with Jeeva Clinical Trials and use their technical expertise and computational resources to successfully implement and scale the Patient Concierge program. Jeeva brings its expertise in executing modern clinical trials with significant efficiency using their innovative one-login unified software platform. Our patient Concierge platform built on Jeeva is driven by AI and tech innovation to automate manual repetitive tasks and has built -n features for eConsent, electronic data capture including patient-reported data, televisits or video visits, with a patient engagement portal, appointment booking, and rescheduling, and much more under a single login

We are applying to the Amgen Prize to overcome several key barriers that hinder our ability to support rare disease patients effectively. 

1. The Prize would enable us to expand our tech-enabled Patient Concierge platform, enhancing its capabilities and reach. We will be able to improve the patient-centric features offered by the platform and upgrade the DEIA capabilities through innovations such as:
   1. Language translation closed captioning, and subtitles using two-way audio-to-text, text-to-text, and text-to-audio between English and all supported languages.
   2. A seamless interface for providing translated content between the concierge staff and patients based on the participant’s native or preferred language.
   3. Integration of patient-friendly educational materials to reduce knowledge, fear, and mistrust barriers.
2. The recognition from the Amgen Prize would build trust and engagement within diverse communities, helping us address the unique needs of patients from various backgrounds.

Our team is uniquely positioned to deliver this solution because we are deeply embedded within the rare disease communities we serve. The core team handling the Patient Concierge is comprised of people of Indian origin who either lived a large part of their lives or are currently living in India. The Team Lead and other key members have strong personal and professional connections within these communities. The Team Lead, Dr. Harsha Rajasimha is a RARE dad, having lost his baby to a rare congenital disease, and is driven to use his years of postdoctoral research and industry experience to support rare disease patients and families from diverse underserved communities such as those of Indian origin. The team closely works with rare disease stakeholders in India and the USA, ensuring our initiatives are informed by diverse perspectives and needs. IndoUSrare collaborates closely with patients, caregivers, and local healthcare providers to incorporate their input and feedback into our solution design and implementation. This close engagement ensures that our efforts are relevant, culturally sensitive, and effectively address the issues faced by rare disease patients and their families.

Additionally, the Patient Concierge is a key program that is central to IndoUSrare's mission of accelerating access to life-saving therapies and research for rare diseases by fostering collaborations between stakeholders in the USA and India. Our programs bridge the critical gaps of the under-representation of the Indian diaspora in global clinical trials for rare disease treatment development and enable them access to critical life-saving therapies. With an initial focus on the Indian diaspora, our goal is to build cross-border collaborations to connect stakeholders of rare diseases in low- and middle-income regions—such as India—with their counterparts and clinical researchers in the US. This would improve the diversity of clinical trial participants, accelerate research and development, and improve equitable access to life-saving therapies for diverse populations of rare disease patients.

Our impact goals are centered around accelerating access and improving the inclusion in rare disease research of rare disease patients from diverse backgrounds with a particular focus on LMICs. Our platform aims to address the unique challenges these individuals face, including access to specialized healthcare, cultural and linguistic barriers, and the need for community support.

In the upcoming year, our primary focus is on laying a strong foundation and expanding our reach to effectively serve rare disease patients:

1. Increased Access and Equity: We aim to increase access to expert advice and validated information on diagnosis, care navigation, and clinical trials for rare disease patients from diverse backgrounds by at least 20%. This will be measured through the number of new requests supported via our platform.
2. Cultural and Language Support: We plan to enhance our platform's ability to provide culturally and linguistically appropriate resources and support. By the end of the year, we aim to see a 30% increase in the usage of language-specific resources and interpreter services, as well as positive feedback from users on the cultural sensitivity of our support.

Measurement and Achievement Strategy for Next Year:

• Data Tracking: We will track user demographics, and service utilization rates using our platform.
• User Feedback: Regular feedback mechanisms will be used to assess patient satisfaction, perceived accessibility, and the effectiveness of our support services.
• Partnerships: We will strengthen collaborations with healthcare providers, advocacy groups, and other organizations to enhance service quality and impact

Next Five Years Impact Goals:

1. Reduced time to diagnosis: We aim to demonstrate measurable improvements in time to diagnosis among rare disease patients from diverse backgrounds. This will be tracked through patient-reported feedback, to achieve a confirmed diagnosis within 2 years.
2. Research and Innovation: Promoting participation in clinical trials and advancing research in rare diseases affecting diverse populations is critical. Over the next five years, we aim to improve the number of patients of Indian origin enrolled in clinical trials through education and awareness and the connections built through our programs, thereby contributing to significant advancements in rare disease treatment options. We aim to double the number of patients reaching out to us being connected to ongoing and upcoming clinical trials.

Measurement and Achievement Strategy for the next five years:

• Long-term feedback collection to track sustained satisfaction rates - Rare disease patients navigate various phases of their journey from diagnosis to treatment and support and we will have regular feedback and update calls to ensure they are supported through these phases - This will be through follow-up and wellness checks through calls and emails once every 6 months for 3 years for each case.
• Strategic Partnerships: Forge strategic partnerships with research institutions, pharmaceutical companies, and policy advocacy groups to leverage resources and influence policy changes effectively.
• Continuous Improvement: Regularly review and refine our platform based on user feedback, technological advancements, and the evolving healthcare regulatory landscape to ensure ongoing relevance and impact.

The solution aims to accelerate equitable access to verified, validated information on experts, clinical trials, and care navigation for rare disease patients globally. The expected outcomes include:

1. Improved Access to diagnosis, treatment, and care for a larger population of patients and caregivers in LMICs, where resources are often limited.
2. Fostering Cross-Border Collaborations between rare disease stakeholders globally, bridging the gaps in rare disease treatment development.
3. Enabling Equitable Access to Diagnosis and Therapies and reducing disparities in healthcare access based on geographic location, language, or socioeconomic status.
4. Long-term Impact: Over time, the Concierge will help create a sustainable infrastructure for supporting rare disease patients and families in LMICs, leading to lasting improvements in healthcare outcomes and quality of life.

Based on our conviction that the only way to achieve equity in healthcare for all rare disease patients is to build ross-border collaborations to enable data sharing and clinical research, the Patient Concierge aims to address the unmet need for access to validated information and expert advice by leveraging the latest technology and the current increase in widespread access to basic internet at reasonable costs to the larger population even in rural areas to bring high-quality information support to this underserved population. Patient families are also more aware of the value and process of clinical research and there is increasing trust in the clinical trial enterprise. The COVID-19 pandemic has resulted in an increasing understanding of the capabilities of decentralized healthcare technologies and the importance of cross-border collaborations to drive research in an expedited way.

The IndoUSrare Patient Concierge now engages the following number of team members:

1. Full-time staff - 3
2. Part-time review committee members - 2
3. Contractors - Jeeva Clinical Trials, Inc software subscription as the technology powering the platform

We also have a team of volunteers and a growing network of rare disease experts who support the solution.

The Patient Concierge has been operational for 4 years - since December 2019. It has developed since then from a simple Google form to a fully functional HIPAA, GDPR-compliant platform with patient-friendly features including omnichannel communication and language support in over 100 languages.

The core mission of IndoUSrare is to address and alleviate inequities in access to life-saving therapies for rare diseases and all our programs are designed to support the inclusion of all individuals.

IndoUSrare leadership and advisory board include representation from Indian, Hispanic, African American, and White backgrounds. Our team is built entirely of women who work remotely with flexible timings to ensure an equitable work environment. The team also comprises individuals with close personal and professional ties to rare diseases as patients, caregivers, advocates, healthcare professionals, or researchers, establishing a high level of sensitivity and empathy for the patients we work to support.

Need and Impact

Our Tech-Enabled Patient Concierge offers a unique blend of advanced technology and a comprehensive network of rare disease experts to deliver personalized assistance to patients and caregivers bridging critical gaps in the current landscape, providing seamless omni-channel communication and information exchange through a centralized platform to ensure that patients can access support in a way most convenient for them, regardless of their geographic location or language they speak. Our platform not only addresses the immediate needs of rare disease patients but also sets a new standard for patient support and care coordination, driving systemic changes in the way rare disease patients in underserved communities can access information support.

Opportunity and Key beneficiaries
Cumulatively the global rare disease burden is huge, with approximately 400 million people affected worldwide, of which 350 million remain undiagnosed. Less than 5% of the 11,000 known rare diseases have approved treatments, highlighting a significant unmet need. Most of these patients live in LMICs such as the Indian subcontinent. Our solution specifically targets these gaps, aiming to enhance diagnosis, treatment accessibility, and overall patient support. India, with an estimated 70 million rare disease patients, exemplifies the pressing need for our services, as many patients face geographical, logistical, and language barriers in accessing care. By targeting these underserved populations, we aim to address significant disparities in access to rare disease diagnoses and therapies, providing critical support to those who need it most.

Services Provided:

1. Verified Information:
Providing accurate and up-to-date information on rare diseases, available treatments, and ongoing clinical trials. This information is crucial for patients and caregivers who often struggle to find reliable resources.
2. User friendly platform offering Personalized Support:
Offering tailored assistance through an omni-channel communication approach, including email, SMS, audio, and video calls. This ensures that patients can access support in the manner most convenient and accessible to them, regardless of their location or language
3. Referrals and Connections to experts and support groups:
Facilitating connections to expert opinions and arranging e-visits with specialists. This service bridges the gap between patients and healthcare providers, ensuring that patients receive expert advice and care recommendations.
4. Long-term follow ups to ensure continued support and access to latest information

Revenue Streams:

The Concierge is a free service that is provided at NO cost to the patient's family. Possible revenue streams that are being planned are:
Subscription Fees: Healthcare providers, hospitals, and clinics can subscribe to our platform to offer enhanced services to their patients. These subscriptions could be tiered based on the level of service and support provided.
Partnerships and Sponsorships: Collaborations with pharmaceutical companies, healthcare providers, and patient advocacy groups can generate additional revenue streams through sponsorships and joint ventures focused on rare disease management and awareness.

IndoUSrare is a non-profit that is primarily funded by grants and donations from individuals, companies, and organizations including other non-profits.

For example:

• We have been recipients of the Horizon #RAREIs Global Advocate Grant and grants for specific projects from Rare-X, and the Rare Disease Diversity Coalition.
• We also receive funding as membership fees from our Corporate Alliance members which include biopharma companies and tech and service providers.
• We have also applied and are being considered for various federal grants such as from NIH and CDC.

Other possible ways to increase revenue that we are considering are:

• Subscription Fees: Healthcare providers, hospitals, and clinics can subscribe to our platform to offer enhanced services to their patients. These subscriptions could be tiered based on the level of service and support provided.
• Partnerships and Sponsorships: Collaborations with pharmaceutical companies, healthcare providers, and patient advocacy groups can generate additional revenue streams through sponsorships and joint ventures focused on rare disease management and awareness.

Our fundraising will ensure that the Patient Concierge is a free service provided at no cost to the patient's family and can be scaled to serve a larger population of underserved communities.