Political Squabbles Slow Drug Development for Rare Diseases

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“The current process for developing new treatments for rare diseases leaves India and the Indian diaspora on the outside looking in.”

Political conflicts have stagnated the FDA’s attempts to modernize clinical trials to effectively reflect the global population. Research for rare disease treatments lacks racial and ethnic diversity, while trials remain inaccessible for millions of people worldwide. IndoUSrare advocates for engaging stakeholders of rare diseases in India and those in diaspora from India in the US to broaden the diversity, equity, and access of participants in research and clinical trials.


The U.S. Food and Drug Administration, the key regulatory agency governing clinical trials in the US, has been slow to address the known lack of diversity in drug research & development. It has also been an agency beset by external political forces as different factions angled for control of the COVID-19 pandemic response. The agency lacked a full-time chief commissioner for over a year, slowing reform efforts. Recent controversies over approved drugs that lack verifiable efficacy have further muddled attempts to diversify evidence-gathering systems.

FDA-approved clinical trials have historically lacked diversity among the participants. Research shows that as recently as 2021, including during the COVID vaccine clinical trials, about 80% of clinical trial participants were white adults, mostly males, from affluent socioeconomic backgrounds. Trials are often based in majority-white regions of the world. This denies people of color, non-English speaking, underrepresented minorities, and people in low-and-middle-income countries (LMICs), such as India, access to critical life-saving therapies and clinical trials related to rare and common diseases.(1)

The Indian subcontinent is home to about a quarter of the world’s population. The Indian diaspora (the dispersion of any people from their original homeland) is the largest in the world, comprising more than 4.2 million people in the US alone. Yet less than 2% of global clinical trials have any study sites in India. Moreover, clinical trial participants do not reflect the racial and ethnic makeup of the global population affected by the targeted disease. Drug development is meant to provide treatments for the general population, but participants in drug trials lack representative diversity.

This lack of diversity in patient clinical trials is counter-productive to research goals, particularly in studies involving ultra-rare diseases where a small number of affected patients are sparsely distributed geographically within the US and globally. Without a diverse base of study participants, researchers lack accurate data on comparisons of disease progression and treatment efficacy. The US Biopharma industry struggles to enroll the required number of patients in orphan drugs (a pharmaceutical drug that, for the most part, has not been developed commercially due to limited potential for profitability) clinical trials within the US, requiring a more global enrollment strategy. From the perspective of rare diseases patients located in LMICs, without local access to clinical trials, remain unable to access novel treatment options. Neither science nor patients are well-served, causing an increased burden on the orphan drug development process.

“The current process for developing new treatments for rare diseases leaves India and the Indian diaspora on the outside looking in,” says Harsha Rajasimha, Ph.D., Founder & Executive Chairman of the Indo-US Organization for Rare Diseases (IndoUSrare). “Indian patients miss out on opportunities to avail clinical research as a care option (CRAACO) or join a clinical trial that offers the best hope for timely care management. Then when drugs are eventually approved, they are based on safety and efficacy data from mostly non-Indian patients. It can be a long wait involving additional costs for a treatment that comes with a question mark about its safety and efficacy in the target Indian patient population.”

IndoUSrare leaders advocate for the inclusion of people of Indian origin in global clinical trials. This is especially true in certain rare genetic diseases, such as multiple sclerosis, muscular dystrophies, GNE myopathy, and blood-related disorders, such as hemophilia, thalassemia, and sickle cell disease, that disproportionately affect the Indian population.

Recent efforts by the Ministry of Health and Family Welfare (MoHFW) in India and the Indian regulatory agencies have ensured that research facilities meet international standards. Even before the COVID-19 pandemic, international pharmaceutical companies had already established research sites in India. The infrastructure is in place for India to be a productive site for global orphan drug clinical trials.(2)

In 2021, the Indian government revived the National Policy for Rare Diseases (NPRD) to assist people with rare diseases. In August 2022, the MoHFW issued a memorandum announcing financial support of up to INR (Indian Rupee) 5 million per patient with a rare disease falling into any of the three groups referenced in the NPRD towards the cost of treatment(s) and disease management. The eight centers of excellence (CoEs) for rare diseases recognized by the NPRD are already working with patients and medical geneticists to identify individuals needing this help. Researchers could build on these frameworks and develop trials that reflect the unmet needs of people in the subcontinent.(3)

“Given the massive number of persons of Indian origin living worldwide and the consanguinity [common ancestry] among various Indian ethnic groups resulting in a disproportionate prevalence of certain genetic diseases, there is an urgent need to develop strategies for inclusion of the diaspora in global research, development, and orphan drug launch planning,” says Dr. Rajasimha. “The access to a wider participant pool promises to speed up trial enrollment, particularly in one-and-done cell/gene therapy trials where sponsors are competing to enroll small patient populations in the US/EU. This will likely accelerate the time to market for the Sponsors and be likely supported through patient grants provided by the Indian Government of up to INR 5 million per patient. Certain public sector units in India also cover 100% of the healthcare costs for over 120 million people including their costs of treatments with no pre-set limits.

“I encourage the new leadership at the FDA to do more to quickly address this unmet need for diversity in clinical trials. We have submitted these perspectives and other ideas in response to FDA’s recent requests for public comments on their guidelines for DEI (6) and towards the development of global clinical trial networks.”

1) https://health.economictimes.indiatimes.com/news/industry/addressing-massive-health-inequities-on-rare-disease-day/89736862
2) https://journals.plos.org/globalpublichealth/article?id=10.1371/journal.pgph.0000890
3) Nature. “When will clinical trials finally reflect diversity?” May 2018.
4) Perspectives in Clinical Research. “Why are clinical trials necessary in India?” April 2014.
5) Ministry of Health and Family Welfare. “Union Health Minister approves National Policy for Rare Diseases.” April 2021.
6) Comments to Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials; Draft Guidance for Industry; Availability. https://www.regulations.gov/docket/FDA-2021-D-0789

About IndoUSrare
IndoUSrare is a humanitarian non-profit 501(c)(3) tax-exempt public charity organization based in the U.S. Founder and Chairman, Dr. Harsha Rajasimha, who lost a child to a rare disease in 2012, has been a rare disease advocate for more than ten years. To address the unmet needs of diverse patients with rare diseases globally, the leadership team comprised of experienced professionals from research, advocacy, regulatory, and drug development seeks to build cross-border collaborations connecting stakeholders of rare diseases in low- and middle-income regions such as India, with their counterparts and clinical researchers in the U.S. to improve the diversity of clinical trial participants, accelerate research and development, and improve equitable access to life-saving therapies to diverse populations of rare disease patients. Visit https://indousrare.org

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