Why Clinical Trials Must Include India

1.

RARE DISEASE CLINICAL TRIALS
· INDIA · GLOBAL EQUITY
Why Clinical
Trials Must
Include India
The Case for Diverse Rare Disease Research

2.

THE DIVERSITY PROBLEM IN RARE DISEASE TRIALS
Most rare disease drugs are tested on a narrow slice of the world — leaving billions of patients without answers.
1.4B
<5%
People in India
Trial inclusion
17.5% of the world's population
Orphan drug trials with Indian patients
450+
96M
No treatment
Patients India
Rare diseases in India without approved therapy
Indians living with a rare disease today
Sources: ICMR | PMC | EURORDIS

3.

Trials built for 10% of the
world cannot serve the
other 90%.
Global rare disease clinical trials are built almost entirely
around Western populations — creating a safety and
efficacy gap for billions.

4.

WHY EXCLUSION HAS REAL CONSEQUENCES
Excluding Indian patients isn't just a fairness issue — it's a scientific one.
Genetic Blind Spots
Drug metabolism varies across
populations. Without Indian data,
therapies may be dosed incorrectly or
cause adverse effects unseen in Western
cohorts.
Sources: EURORDIS | PMC | Lancet
Missed Disease Signals
Some rare diseases present differently in
Indian patients. Excluding this population
means unique symptoms and disease subtypes go undetected in research.
Access Denied
Drugs approved without Indian trial data
face additional regulatory hurdles in India
— delaying or blocking access for the
patients who need them most.

5.

THE INDIA OPPORTUNITY
Including India isn't just the right thing — it's
the smart scientific move.
Massive Patient Pool
1,000+ Trial Sites
96M+ genetically diverse rare
disease patients, many with
uncharacterized conditions
representing novel research
opportunities.
India has 1,000+ CDSCOapproved clinical sites and a
growing GCP-trained workforce
ready for international rare
disease research.
Unique Genetic Variants
Indo-US Collaboration
India's population harbors rare
variants not found elsewhere,
offering insights that could unlock
new therapeutic targets globally.
The Indo-US Bridging RARE
Summit and Pitch4RARE are
building the bridges needed to
make inclusive rare disease trials
the standard.

6.

WHAT NEEDS TO HAPPEN
Three actions rare disease organizations, regulators, and researchers can take right now.
Mandate Diversity in Trial
Protocols
Build India-Specific Research
Infrastructure
Scale Cross-Border Partnerships
Regulators and sponsors must set explicit
diversity targets — requiring a minimum
percentage of LMIC participants including
India in rare disease trial design.
Investment in genomic databases, patient
registries, and GCP-trained site networks
across India will accelerate rare disease
trial readiness nationwide.
India-US research collaborations —
modeled on IndoUSrare's Bridging RARE
Summit and Vision 2030 with GORD —
must become the global standard.
Sources: EURORDIS | WHO | IndoUSrare Vision 2030

7.

Science Works Better
When It Includes
Everyone.
Every rare disease patient — in Boston or Bengaluru — deserves
therapies tested on people like them. Inclusive rare disease clinical
trials are not a future aspiration. They are a present scientific
necessity.
In researching this topic, I came across the Indo-US Bridging RARE Summit and Vision
2030 — two initiatives that are already turning this vision into practice, and a
compelling example of what becomes possible when borders stop being barriers.