India’s clinical research industry is staging a comeback after a decade of mistrust, regulatory crackdowns and “guinea pig” fears drove global drugmakers away from the country.
But even as India emerges as the world’s third-largest destination for clinical trials, a key gap remains: first-in-human (FIH) studies, the earliest and most critical stage of testing new drugs on people.
The hesitation is rooted in a painful past.
Allegations of unethical trials, weak oversight and inadequate patient protections during the late 2000s and early 2010s, including trials of a vaccine against cervical cancer and cancer drugs, triggered public outrage and judicial scrutiny.
India responded with sweeping safeguards, but the tightening also made regulators more cautious, especially around Phase 1 and FIH studies where experimental molecules are tested in humans for the first time.
The impact was stark. Globally, more than 51,000 Phase 1 trials were conducted between 1999 and 2022, but India accounted for just 2,260 — barely 1.5 percebt despite being home to nearly 20% of the world’s population.
Today, industry leaders say the country has rebuilt credibility in later-stage research, but remains restrained in early-stage innovation where discretionary approvals under India’s New Drugs and Clinical Trials (NDCT) Rules, 2019 still shape the landscape.
These rules were enforced to regulate the approval and conduct of clinical trials and new drugs in India and key features include fast-track approval timelines, provisions for post-trial access, strict ethical conduct rules, and standardized guidelines for serious adverse event reporting and financial compensation.
ETHICS LEGACY PERSISTS
“While the country has emerged as an important destination for Phase 2 and 3 studies, participation in First-in-Human trials remains relatively limited,” said Dr Priya Kapoor G. Hingorani, managing director at Miltenyi Biotec India and vice president for APAC.
“These studies represent the earliest stage of translating scientific innovation into potential patient impact and are critical for building a strong innovation-led healthcare ecosystem,” she said.
Experts say the caution is understandable. Phase 1 studies involve healthy volunteers or small patient groups and are designed to establish safety, dosage and side-effect profiles before larger efficacy trials begin. Any failure at this stage can have serious implications.
“Following concerns around ethics and oversight more than a decade ago, India adopted a more cautious regulatory approach toward early-phase research,” Hingorani said.
“These reforms were important in strengthening patient protection and compliance standards, but they also contributed to longer approval timelines and operational uncertainty for global innovators.”
The reforms transformed the regulatory ecosystem.
Under the NDCT Rules, 2019, approval timelines for clinical trials were capped at 90 working days.
Compensation norms for trial-related injury or death were formalised, ethics committee oversight was strengthened and informed consent requirements became stricter. Participants now have mandatory rights to full risk disclosure, constant medical monitoring and withdrawal from studies at any stage.
Industry executives argue that these protections are often overlooked in public perception.
“The ‘guinea pig’ label ignores the reality that no first-in-human study begins until extensive laboratory and pre-clinical testing establishes safety thresholds,” said one senior clinical research executive.
Protocols in regulated trials, experts add, frequently involve tighter monitoring than routine healthcare settings.
Dr Santanu Tripathi, veteran pharmacologist and clinical trial expert, said public participation in research should be viewed as a social contribution rather than exploitation.
“If access to evidence-based treatment is a right of the citizens, participation in clinical research to generate such evidence is their responsibility and social obligation,” he said.
“Clinical research is the only way to evaluate and validate new interventions. Without voluntary participation in clinical research, the development of life-saving drugs, medical devices, and diagnostics is impossible.”
DISCRETIONARY APPROVAL BARRIER
Despite reforms, India’s approach to imported molecules in Phase 1 research remains conservative compared with competing markets such as Australia.
Sanjay Vyas, managing director for India at Parexel, said the NDCT framework still creates uncertainty because Phase 1 trials for drugs developed outside India generally require special regulatory consideration.
“The challenge in our regulations is the fact that if it is a molecule that is not developed in India, our NDCT regulations say that you cannot do a Phase 1 trial here,” Vyas said. “You first need enough data and then come back for a Phase 2 trial or a bridging study.”
He pointed out that regulators do grant exemptions in cases of unmet medical need, as seen during the COVID-19 pandemic, but approvals remain discretionary rather than automatic.
“Every time you have to go for an approval, it has to be an exceptional approval to do a Phase 1 trial,” he said. “Unlike in Australia, where all Phase 1 studies are welcome as long as safety is proven.”
That difference has reshaped global clinical trial geography.
Australia, Japan and China have rapidly expanded their share of early-stage drug research over the past decade by streamlining approvals and investing in specialised Phase 1 infrastructure, following countries like the US where many these trials have been conducted for decades.
India, by contrast, became heavily concentrated in Phase 2, 3 and 4 trials, where drugs already have preliminary safety data.
Yet momentum is returning.
Between 2019 and 2024, India recorded 1,641 Phase 1 trials, with around 60 percent involving first-in-human studies. Phase 2 and 3 trials are growing at roughly 20 percent annually.
India also registered nearly 18,000 new clinical trials in 2024, marking a 50 percent year-on-year increase and an estimated 80 percent compound annual growth rate since 2019.
The country’s clinical trial market is projected to expand from $1.68 billion in 2025 to $3.62 billion by 2035.
HIGH-STAKES OPPORTUNITY
Industry experts say the stakes go far beyond investment and outsourcing contracts. Limited participation in early-stage research delays patient access to breakthrough therapies in disease areas where India faces a heavy burden
Cancer, rare genetic disorders, neurological diseases, autoimmune conditions and advanced infectious diseases are among the areas most affected by the shortage of cutting-edge clinical research infrastructure.
Emerging fields such as cell and gene therapy are particularly dependent on early-phase trials because treatments are often highly personalised and require sophisticated biomarker analysis and intensive patient monitoring.
“Greater participation in early-phase research can help improve access to cutting-edge therapies for patients, particularly in areas with high unmet medical need,” Hingorani said.
She added that India possesses several natural advantages: one of the world’s most diverse patient populations, growing scientific expertise, expanding digital health infrastructure and increasingly mature regulatory systems.
But scaling FIH capability requires more than policy reform. Dedicated Phase 1 hospital units, real-time pharmacovigilance systems, translational research platforms and investigators trained in early-phase risk management remain limited.
Vyas said expanding Phase 1 studies would also strengthen hospitals and research institutions financially and scientifically.
“Phase 1 studies always happen in a hospital environment, in a controlled environment,” he said. “Once you do the Phase 1 and efficacy is proven, access to the medication becomes much easier for patients as well.”
India’s balancing act now lies in preserving hard-won ethical safeguards while avoiding over-correction that pushes innovation elsewhere. For regulators, the memory of past controversies continues to influence decision-making.
For industry, the challenge is building trust that clinical trials are no longer synonymous with exploitation. And for patients with life-threatening illnesses, the outcome could determine how quickly they gain access to the next generation of therapies.
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