RecupeRare (TM)

India does not have a definition for Rare Disease yet. In some instances, it is referred to as 1 per 2500 people, however a definition is required for budgeting exercises.  If we apply the international estimate of 6% - 8% of the population being affected by Rare Diseases, we will have approximately 72 million to 96 million Indians living with Rare Diseases. Now applying the international percentages for the various areas of Rare Diseases, we have:

1. 72% Rare Diseases are genetic – approximately 50 million – 67 million Indians having genetic form of Rare Disease
2. 70% of these rare genetic diseases start in childhood.
3. Many rare diseases, including infections, some rare cancers and some autoimmune diseases are not inherited.
4. In the United States, only a few types of rare diseases are tracked when a person is diagnosed. These include certain infectious diseases, birth defects, and cancers. It also includes the diseases on state new-born screening tests. Because most rare diseases are not tracked, it is hard to determine the exact number of rare diseases or how many people are affected. The same holds true for India.
5. Ministry of Health and family Welfare, Government of India formulated a National Policy for Treatment of Rare Diseases (NPTRD) in July, 2017. It saw the light of day in 2021.
6. Early diagnosis of rare diseases is a challenge because of multiple factors that include lack of awareness among primary care physicians, lack of adequate screening and diagnostic facilities. Delay in diagnosis or a wrong diagnosis increases the suffering of the patients. 
7. About 95% of rare diseases have no approved treatment even when a correct diagnosis is made.
8. Where drugs are available, they are quite expensive as very few pharmaceutical companies globally manufacture drugs for rare diseases. For this reason, rare diseases are also called ‘orphan diseases’ and drugs to treat them are called “orphan drugs.
9. In India, rare diseases are categorized under the following groups.

Group 1: Disorders amenable to one-time curative treatment:

• Disorders amenable to treatment with Hematopoietic Stem Cell Transplantation (HSCT)
• Disorders amenable to organ transplantation           

 Group 2: Diseases requiring long term / lifelong treatment having relatively lower cost of treatment and benefit has been documented in the literature and annual or more frequent surveillance is required:

• Disorders managed with special dietary formulae or Food for special medical purposes (FSMP)
• Disorders that are amenable to other forms of therapy (hormone/ specific drugs)

Group 3: Diseases for which definitive treatment is available but challenges are to make the optimal patient selection for benefit, very high cost and lifelong therapy.

• Based on the literature sufficient evidence for good long-term outcomes exists for the given disorders
• For the following disorders for which the cost of treatment is very high and either long term follow up literature is awaited or has been done on a small number of patients

RecupeRareTM – The Central Idea & what it does

• Most patients with Rare Diseases do not have access to therapies – either due to funding constraints or due to unavailability of medications within the country.
• As the name suggests – the RecupeRareTM platform will seek to address the needs of patients with Rare Diseases and provide them options and opportunities to avail therapy (long term or short term). This will enable such patients to recuperate from these diseases.
• Education - RecupeRareTM will help in educating the society on Rare Diseases
• Advocacy - RecupeRareTM will advocate the need for Rare Disease therapy
• Source therapies - RecupeRareTM will help patients with sourcing the therapy directly from the manufacturer or their authorised sales channel
• Import, Customs clear, Supply & Deliver (24*7) - RecupeRareTM will undertake all the regulatory clearances to ensure that the therapy is available to the patient.
• Clinical Trials - RecupeRareTM will support organizations, manufacturers and the government in enabling more clinical trials within the geography of operations. We will also support the development of the Clinical Trials infrastructure within the geography of operation.   

Impact that RecupeRareTM will have

RecupeRareTM will be able to impact healthcare in the following ways:

• Enable Access – There will be the possibility of a patient getting the therapy directly from the manufacturer.
• Be the bridge between manufacturer and the country’s requirements – It will help manufacturers form the supply arm for therapies even without their physical presence in the geography.
• Become an Advocacy platform – for policy and processes that enable a supportive environment around Rare Diseases in the geography.

Addressing the complete Rare Disease landscape – Advocacy, Clinical Trials and Product procurement

• Become the knowledge bank for Rare Disease in the geography.
• Support Clinical Studies on Rare Diseases – by linking researchers with the clinical study platform and enabling development of research papers on the subject.
• Enable the regulatory clearances and supply chain domains for organizations and manufacturers to enter the geography.  
• Data generation for government, organizations, manufacturers and those related to Rare Diseases – that can help enable better support to the patients with Rare Diseases.
• Drive the agenda of Clinical Trials through linking study requirements with investigators with CROs.
• Making Products available – through addressing the regulatory and legal requirements of the geography.

Dr. Bhaskar Jyoti Sonowal has been instrumental in conceptualizing and developing the National Rare Disease Registry, by aligning and motivating stakeholders to initiate the project. It has since registered more than 15,000 patients with Rare Diseases across 12 Centers of Excellence. 

The National Initiative for Rare Diseases is a project initiated by Dr. Bhaskar Jyoti Sonowal, which ensured that a policy platform is created whereby policy makers interact with Academia, Industry and Patient Bodies. This initiative led to the formation and drafting of India's National Policy on Rare Diseases in 2017. 

This program was created by Dr. Bhaskar Jyoti Sonowal to ensure that a Rare disease like Hemophilia is brought to the limelight. This marketing program reached more than 300 million people across India. 

The above examples are proof of the possibility that Dr. Bhaskar Jyoti Sonowal, as the Managing Director of HED Healthcare can create. With adequate support from Academia and Industry to Dr. Bhaskar Jyoti Sonowal, the next big hurdle of funding Rare Disease therapies can be created. 

Dr. Bhaskar is supported by his team led by several stalwarts from the Medical Devices Industry. We have the Ex-Director of Medtronic supporting us on the Supply Chain front. We have a Senior Software professional that leads our team of software development. We also have our CEO, Ms. Bhairavi Sonowal leading the efforts on the patient bodies front. She is an ardent worker who has spent almost all her working career trying to bring about upliftment and justice to the people suffering from social inequalities or diseases. 

Dr. Bhaskar is also the lead for IndoUSrare, a NGO based out of the US for all Rare Disease related activities that would be done in India.  

HED Healthcare also represents the software solutions provider Jeeva Informatics Inc. (based in US) in Asia. Through this platform we seek to enhance the Clinical Trials ease for all diseases including Rare Diseases.

Our credentials are amongst the strongest in the field of Rare Diseases. 

The major issues that patients face can be broadly divided into 9 areas.

1. Delay in Recognition & Diagnosis

In the more developed economies, a patient usually goes through 5-7 consultations before a diagnosis is made. The number of such visits is unknown, but must be a higher number.

Moreover, the physicians are not trained to recognize the Rare Diseases, and therefore most diseases are missed.

2. Delay in seeking medical help

India is a diverse country with the majority of the population living outside the metropolitan cities. Almost all the Rare Disease healthcare providers live in one of the metropolitan cities, and therefore, invariably all patients have to come to such cities to be diagnosed and treated. These hindrances therefore cause the delay in seeking medical help.

3. Social discrimination

Many Rare Diseases are accompanied by either a prolonged sickness or disabilities or deformities. The lack of awareness amongst the general public as well as the larger medical fraternity, creates many social taboos. Many face discrimination and lack acceptance in society.

4. Economical crisis

Due to the lack of insurance and reimbursement for most of the people of India, many people are forced to sell their valuables, take loans and sometimes even their property to ensure that the expensive medications can be procured for their family members. This puts a huge burden on the family, pushing them into further economical crisis. 

5. Expensive and mostly unavailable therapies

Many of the therapies are not available in India commercially. Some patients have been provided the Charitable Access, however a large number of them go without treatment since they are either not available or just too expensive to be procured by an individual. The lack of Insurance cover or reimbursement schemes, makes such therapies unviable for most patients.

6. Lack of freely available Rehabilitation, Exercises and Physiotherapy facilities

The Rare Disease patients find it difficult to access facilities where rehabilitation, exercises and physiotherapy could be available. Such centers are far and few, and therefore the essential requirements are most often unaddressed.

7. Patient support groups not strong enough

The Patient support groups are active in a few geographies only, and therefore most patients find it difficult to have a voice that can speak for them. There is an urgent need for a unform and larger voice

8. Long term funding for therapy

The Government of India’s National Policy for Rare Diseases holds great promise for the patients with Rare Diseases. Now, the funding has to be channelized to ensure that the patients start benefitting. State governments need to start asking for the central funds as well as generate their own funds. Having a state nodal center for such rare disease patients that can fund them would be critical. The generation and usage of CSR funds would also go a long way to help patients.

9. Quality of Life

The patients of India are currently deprived of a good quality of life. There is minimal awareness in both physicians and society, very little access, practically non-existent funding for therapies and very few rehabilitation, exercise and physiotherapy facilities. A lot of effort is required to help improve the quality of life for patients living with Rare Diseases in India.  

We would face the following hurdles, which we plan to overcome:

1. Direct relationship with ever manufacturer - this is critical since we plan to source directly from the manufacturer. 

2. Regulatory challenges - Every country exporting the medication would have regulatory processes, and so does the importing country. A team to ensure that there is a smooth functioning of this aspect would require an in depth understanding.

3. Legal challenges - Every patient would have to be evaluated with the legal lens, which would consider whether the therapy would be the right one, adequate in quantity and whether it would make an improvement in their lives. 

4. Supply Chain challenges - Many of these medications would be requiring cold chain, and therefore we need to ensure that there is no break this the cold chain.

5. Funding - We will need to arrive at funding models and be innovative at them by using not just the social knowledge but also the commercial justifications. 

We do not have a procurement methodology in India at this stage. This is also not available in many Asian countries that I have worked in. This application would therefore, through a thorough marketing approach, become a useful link for many patient bodies, Doctors and patient relatives. 

Since international transactions would have legal and regulatory aspects, not many people have joined the race. Moreover this would also require a tremendous supply chain capability, besides the software and technological interventions. 

We plan to become and remain the only provider of such solutions over the next 5 years.

• Become the knowledge bank for Rare Disease in the geography.
• Support Clinical Studies on Rare Diseases – by linking researchers with the clinical study platform and enabling development of research papers on the subject.
• Enable the regulatory clearances and supply chain domains for organizations and manufacturers to enter the geography.  
• Data generation for government, organizations, manufacturers and those related to Rare Diseases – that can help enable better support to the patients with Rare Diseases.
• Drive the agenda of Clinical Trials through linking study requirements with investigators with CROs.
• Making Products available – through addressing the regulatory and legal requirements of the geography.

We will also address multiple areas through the Recuperare platform. 

Our company, HED (Healthcare, Education, Environment & Development) Healthcare Private Limited, was set up with creating business solutions around the SDGs. We have focused our businesses upon:

1. SDG 3 - Good Health and Wellbeing

2. SDG 4 - Quality Education

3. SDG 5 - Gender Equality (we promote Diversity through a sister organization, where our CEO, Ms. Bhairavi Sonowal is a partner)

4. SDG 6 - Clean Water & Sanitation

5. SDG 7 - Affordable and Clean Energy

6. SDG 10 - Reduced Inequalities

7. SDG 17 - Partnerships 

Our solution would therefore impact SDG 3 and 10. 

We will measure progress by the following parameters:

1. Number of Users

2. Number of Enquiries

3. Number of Patients supported through us

Our desired long term change: Rare Disease patients get easy access to their medications, irrespective of where they are located geographically, and receive the same treatment across the globe.

The change envisaged: Over the next 10 years, if we are adequately supported, we will work on the multiple problems or middle points that we have identified. Some of them include:

1. If a company is not registered in a country, their medications are usually not available.

2. The regulatory processes are different, vast, complicated and not easy to understand.

3. Funding mechanisms are state / country based. A more uniform ability to address this area would help millions of people.

We are using existing tools to make our business model more effective and have a larger reach. We also plan to use AI to drive patient interactions and help them choose their responses. This will enable us to understand our customers (manufacturers responses, timeframe of response, location of medical solutions and regulatory mechanisms) better, so that the response time is shortened and better customer relationships can be cultivated. 

Our mission in the area of women’s and societal development is to create a more inclusive environment where differences are recognized, understood, appreciated and utilized to its full potential. In the mission to spread awareness on various aspects and best practices of Inclusion, we are working with organizations and institutions to sensitize, build awareness and equip them with tools to build a more inclusive world. 

Under our education wing, we have developed an agreement with BD Foundation to carry out our work in Diversity and Women’s development in the social sector. BD Management Advisory (a.k.a BD Foundation) is a global think tank focusing on Diversity and Inclusion. The organization is providing a platform for creating awareness on inclusive practices, conducting research & building advocacy platform to promote inclusive leadership. As a Global Top 10 Diversity Consulting firm, the foundation recognizes that every organization has unique talent management needs, needs that cannot be addressed with a simple "one-size-fits-all"providing. That’s why we take the time to understand its client's business objectives and provide solutions tailored to meet those head-on.

We are an extremely passionate team in the area of women’s development and societal development – differentiated by our commitment to making a difference and harnessing the power of business to promote diversity and inclusion. It is our endeavor to provide end to end value added services across functionalities and domains to our clients and assist them to create a robust culture by making Diversity and inclusion a vibrant reality in the workplace. We work across all facets of diversity, women entrepreneurship and economic development– Gender, LGBT, disability, age, culture, and so on. This is where our multi-disciplinary team of practitioners with their diverse skill sets, competencies and varied experience bring their expertise to each of the projects in hand.

Our Business Model is very straightforward. We have 4 business units, which operate under the overall umbrella of the organization. Each of the units will be considered a P&L unit. 

Recuperare would similarly have a separate P&L account. The funding would be self generated by patients, hospitals and doctors joining the platform. 

On the conservative front, revenues would grow to 38.08mn USD by 2031. Detailed working is as under:

Still in Pilot