The Basics
Name: Harsha Rajasimha

Title: Founder and CEO

Organization: IndoUSrare (Indo-US Organization for Rare Diseases)

Social Media Links:

Disease focus: General education, research, and support services for all rare diseases

Headquarters: Herndon, Virginia

How did you become involved in rare disease: My first encounter with a rare disease was about 10 years ago at the neurobiology, neurodegeneration, and repair laboratory (NNRL Chief: Anand Swaroop) at the National Eye Institute at the National Institutes of Health. I was a genomics data scientist (consulting) back then analyzing next-generation sequencing data sets from inherited retinal disease familial cases. As we were discovering novel rare and common mutations in FBN2 (an extracellular matrix gene associated with macular degeneration) in 2012 at NEI, my family and I were introduced to the sufferings and pain that more than 300 million patients and families with rare diseases deal with worldwide. I had become the father of a child with Edwards Syndrome, a rare congenital disease. The baby was diagnosed at full-term birth and did not make it back home from the neonatal intensive care unit. My family and I were devastated. As we went through several months of soul searching and education, my eyes were opened up to the other side of rare disease research—patient advocacy. I decided to dedicate a percentage of my time volunteering for the rare disease community. In the majority of my time, I decided to apply years of post-doctoral training in genomics and precision medicine towards accelerating the discovery and development of novel diagnostics and therapeutics for rare and orphan diseases.

Previous career: My involvement in rare diseases has been in the capacity of a genomics data scientist from 2003 onwards starting with the analysis of raw, next-generation DNA sequencing datasets for bacteria and virusus that could be used as objects of bioterrorism, rare retinal disease genomics data, and global commercial launch of gene panels for rare diseases and Cancer.

Education: BE in Computer Science & Engineering, Bangalore University, India; MS in Computer Science & Applications with Bioinformatics Option, Virginia Polytechnic Institute and State University; Ph.D. in the interdisciplinary program in Genetics, Bioinformatics and Computational Biology, Virginia Polytechnic Institute and State University

The Organization
Organization’s mission: IndoUSrare is an independent non-profit organization focused on helping patients with rare diseases of Indian origin in the United States, India, and globally with a vision to build collaborative bridges between the east and the west to accelerate research and development of diagnostics and therapies through education, advocacy, and research.

Organization’s strategy: Our strategy is to focus on accelerating novel diagnostics and therapeutics by bringing the East and the West together in R&D collaborations. We collaborate with Global Genes, RARE-X, IRDiRC, NORD, UDNI, RDI, ICORD, and other international consortia to fill the specific gaps between the United States and India for rare disease communities. We promote, encourage, and facilitate the inclusion of data from diverse patients, including those from the Indian subcontinent, in global research databases. In addition, we create pathways and a roadmap for ultra-rare patients including N-of-1 cases who contact IndoUSrare for assistance. Lastly, we engage with the governments and policymakers, which is crucial to bring about real sustaining change/impact.

Funding strategy: Our funding strategy in the first three years is to primarily fund our activities through corporate social responsibility, individual donations, crowd funding. The strategy is to create self-sustaining programs that can create measurable impact.

What’s changing at your organization in the next year:
The rare disease community of advocates are asking how they can engage with IndoUSrare. They are eager. We anticipate launching three programs to engage the rare community this year. We will announce them once we have defined the programs well enough. Meanwhile, we will continue the IndoUSrare newsletter series, public and community awareness, engagement events, scholarship grant programs in collaboration with Global Genes to educate and engage Indian American rare disease advocates; and speaking, participating, and collaborating at major conferences and events, such as the global rare disease day, NIH rare disease day, RDLA events, and other annual events.

Management Style: We have a board of directors with seven members that might expand to ten. The goal for the board is to raise funds and define programs (self-sustaining and measurable impact) in consultation with the advisory board members, which can enable the hiring of an executive team to run the programs.

Management philosophy: Do not duplicate efforts, collaborate with existing organizations and consortia to the maximum extent possible, create self-sustaining program models with measurable impacts.

Guiding principles for running an effective organization: Servant leadership, unwavering focus on patients, transparency, and accountability.

Best way to keep your organization relevant: Keep showing up at conferences and events, be a collaborator, be genuine, and focus on unmet needs of the neglected patient communities.

Why do people like working with you: Because we love working with them. When you demonstrate the commitment to do the right thing for patients, think win-win collaborations with mutual respect, and be sensitive to nomenclature and needs, it’s natural for an organization to attract like-minded people and collaborators. There will always be people who still don’t want to collaborate, or work in silos. We have to take as many people along as possible and keep moving.

Mentor: Frank Sasinowski (director at Hyman, Phelps & McNamara) and Stephen Groft (senior advisor to the director, NCATS, NIH) have been great mentors and I admire their leadership styles.

On the Job
What inspires you: The fact that the technologies currently exist (e.g., genome sequencing, gene editing, digital health) to enable the diagnosis and treatment of most rare diseases. Yet, 95 percent of rare diseases remain without FDA approved treatments.

What makes you hopeful: The U.S. FDA and NIH’s leadership in driving patient centricity and innovation. The recent programs like patient listening sessions and patient-focused drug development are a major step forward. They provide a framework for IndoUSrare to represent the minority communities’ needs.  

Hardest lesson learned: Documentation and paperwork (agreements) are very important and cannot be postponed even when acting in the best interest of the patients.

Toughest organization decision: The creation of the organization itself was the toughest decision. Most individuals involved in the organization are contributing to the rare disease communities in their own capacities already. It’s a lot of hard work to create and sustain a non-profit organization. I commend the collective decision to form IndoUSrare to fill specific unmet needs and gaps.

Biggest missed opportunity: The Indian Health Ministry released a national policy for the treatment of rare diseases in January 2020. The policy missed a lot of ground including adhering to the formal definition of a rare disease. IndoUSrare did not get a chance to engage with the policymakers before the official release of the policy. It’s a missed opportunity. However, we look forward to engaging with U.S. and Indian policymakers to see how we can influence the amendment of the policy.

Like best about the job: Every day is a new day with new opportunities to advance the mission. It’s a global village. Policy frameworks (e.g., ODA) are working and encouraging.

Like least about the job: It’s never ending. Sometimes its tiring and exhausting. I don’t like dealing with illogical behavior and politics.

Pet peeve: We all say the number of patients with rare diseases is more than 300 million globally, 30 million in the United States, 70 million in India.  However, a majority of these people remain undiagnosed or otherwise elusive from the healthcare systems.

First choice for a new career: Rock star

Personal Taste
Most influential book: The Emperor of All Maladies: A Biography of Cancer by Siddhartha Mukherjee. It reminds us about the nature of the common enemy.

Favorite movie: Seven or Lorenzo’s Oil

Favorite music: Jazz

Favorite food: Thin crust veggie pizza

Guilty pleasure: At the end of every holiday season in December, I end up with 10 extra pounds to lose in January.

Favorite way to spend free time: Do nothing vacation and travelling with kids

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