‘There are plenty of problems begging to be solved in rare diseases’

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Harsha Rajasimha is the Founder Chairperson of the Indo-US Organization for Rare Diseases, a non-profit organization dedicated to tackling rare diseases through international collaboration, research, clinical trials, and data sharing. This year, the organization is hosting its second annual flagship event, the Indo-US Bridging RARE Summit 2024, which will take place in person from November 16-18 in New Delhi, India.

In an exclusive interview with South Asian Herald, Rajasimha, a Virginia resident and Founder and CEO of Jeeva Clinical Trials, discussed the landscape of rare diseases in India, and the United States and highlighted the significance of the upcoming summit in addressing the unique challenges these diseases pose.

What can you tell us about the 2024 RARE Summit and its mission?

The Indo US bridging RARE Summit 2024 is a flagship event where we bring key stakeholders of rare diseases, orphan drug development, and policy makers from both United States and India together under one roof. The three-day Summit will feature stimulating conversations through panel discussions, and working groups to essentially address key challenges, such as policies for newborn screening, diagnostics, orphan drug development and clinical trials, and how to make them more accessible for all people in both US and India and globally.

Who should attend this Summit, what message do you have for them?

I take this opportunity to invite all people involved in rare diseases; from a research point of view, patients, families, advocacy groups and nonprofit organizations, or if you are an innovator, startup entrepreneur looking for problems to solve. There are plenty of problems begging to be solved in rare diseases. And so, I invite all of you to join us at the Summit in New Delhi.

Can you please share some insights into rare diseases?

A rare disease is any disease that affects less than 200,000 individuals. In India, there is a definition for orphan drug, which is meant to treat a condition that affects fewer than 500,000 people. So, every country has adopted its own country definition of what a rare disease is, primarily based on the prevalence of diseases, such as muscular dystrophies. There are many types of muscular dystrophies, and each type might be rare.

For example, like Duchenne muscular dystrophy and limb girdle muscular dystrophy, spinal muscular atrophy is another neurological condition which is considered rare. And in India, the there is a national policy for rare diseases now which recognizes much smaller numbers of diseases as rare, mainly focusing on treatable rare diseases. The challenge is 95 per cent of rare diseases are not treatable, and most of them are still in research and development phase, in animal models, in pre-clinical and data, and patient registries. So, it’s very important to have digital access to these data sets of patients with rare disease so that investments can be made in research and development of treatments and diagnostics.

Given the existing complexities associated with rare diseases, how does your upcoming Summit plan on addressing these challenges?

There are complementary strengths, and complementarity between what United States offers and what India offers. The first country in the world to adopt an Orphan Drug Act was the United States in 1983, when President Ronald Reagan signed that into Act. That has resulted in a patient led revolution for developing therapies in the US, where they receive seven years of market exclusivity and tax credits for investments that go into developing treatments for rare diseases in the US. We now have 1100 drugs that are approved by the Food and Drug Administration (FDA) that target specific rare diseases, whereas very few of these are accessible and affordable anywhere else in the world, other than European Union and Japan. But, there is a lot of scientific talent, technology, AI, and digital health talent, coming out of India. There is also a massive complementarity in what United States offers in terms of investments and funding in biomedical research in terms of scientific advances and biomedical technology innovation and new drug development, with an ecosystem built in the US over last 30 years for rare diseases,

India has demonstrated tremendous progress in the last five years with a national policy for rare disease. A recent 2024 court order by the Delhi High Court directed implementation of the national policy expeditiously and make these treatments available to patients in India. This is very encouraging as it provides an opportunity to bring US and India closer together and piggyback on the progress that has already been made for cancer. In fact,  the White House’s Cancer Moonshot Initiative opened doors to Indian clinical trials for cancer with the FDA’s Oncology Center of Excellence Project Asha initiative.

Does India have access to these medications produced in the United States?

Typically, the approvals within a country need to be provided by regulators in that country, and so FDA-approved drugs are mainly meant for marketing within the United States. Now, drug Controller General of India recently passed a circular in August 2024, which is also super positive and encouraging. Indian regulators will consider a waiver of new clinical trials in India for drugs that are already approved by the FDA and or UK, European Medicines Agency, Canada, Australia, and Japan.

What are the lessons learned from last year’s Summit, and how does that translate to identifying solutions at the 2024 Summit?

Last year, we held the Summit at the George Mason University. The idea of alternating the Summit in India and US is to increase cross border collaborations, experience culture, an understand on ground challenges and opportunities. Last year we had a great participation from US based companies that are dedicated to developing treatments for rare diseases. There are investments going on for these drug development efforts, which can take 5-10 years of high-risk investments. But, when they come through, they are lifesaving. And we have seen that happen with more than 17 gene therapies, including two gene therapies that were approved on the same day in December 2023 for sickle cell disease. This disease affects 1 million people in India, but it’s rare in the United States. In Africa, there are 5 million people affected by sickle cell disease.

Can you please talk about your other venture Jeeva Clinical Trials? 

I became the dad to a child with rare disease in 2012, as I was coming out of this post-doctoral research experience at NIH. Eventually, I got exposed to this revolution led by patients and families with rare disease in the US, starting with the Orphan Drug Act of 1983. As such, I started getting more involved in patient advocacy, in addition to science and technology. So, I founded Jeeva Clinical Trials as a software platform which is truly patient centric, that can make automated user-friendly software for patients to participate in clinical trials and creation of rare disease patient registries and natural history studies.

Can you please tell us more about your child’s condition?

My late daughter Khushi was diagnosed with Edwards syndrome, which is a Trisomy of chromosome 18. She only lived for four days in the NICU, and we had to make the difficult choice of pulling the plug on ventilators after four days. And that’s why we have instituted an award, Abbey Meyers Khushi Bridging RARE Award, which we award each year to at least one individual in the US, and one in India, who have made significant contributions to cross border collaborations. Abbey Meyers represents the US orphan drug revolution, and Khushi represents the Indian child that suffered with a rare disease.

(This interview has been edited for length, brevity and clarity)

(Used under special arrangement with South Asian Herald)