Precision Medicine to Smart Regulation: India's New Health Frontier

Most of India's health story is about fighting disease after it strikes. But a quieter revolution is working from the other end: reading a patient's genes before prescribing, storing biological samples in national biobanks, giving patients with ultra-rare diseases a fighting chance, and policing the pills, vapes and fats that quietly harm millions. This article covers that new frontier: precision medicine and biobanks, India's rare-disease policy, the long battle over fixed dose combinations, and the regulation of e-cigarettes and trans fats. For the bigger disease-management story, antimicrobial resistance, vaccines, TB, drug quality and NCDs, see our companion article on health and biotechnology.

Medicine tailored to your genes: precision medicine

Precision medicine is an approach that tailors healthcare interventions based on an individual's genetic makeup, environment, and lifestyle. It marks a decisive shift from the one-size-fits-all model of medicine to a personalised healthcare model, where the right drug reaches the right patient at the right dose. In India, government initiatives are now harnessing genetic data to tackle non-communicable diseases (NCDs), rare disorders and pandemic threats through personalised therapies.

Three national programmes anchor this push. The Genome India Project, which sequenced 10,000 genomes from 99 ethnic groups to discover genetic causes of disease, is covered in detail in our biotechnology article. The Phenome India Project has collected 10,000 samples to build better predictive models for cardiometabolic diseases, linking measured traits like blood pressure and blood sugar to underlying genetic risk. The PRaGeD Mission, short for Paediatric Rare Genetic Disorders, maps the genes and variants behind rare childhood diseases so that targeted therapies can be designed for children who today have almost no treatment options.

Why this matters for India is simple: India is among the most genetically diverse populations on earth, and drug responses discovered in European or East Asian genomes do not always transfer. Population-specific data reduces adverse drug reactions, uncovers disease genes unique to Indian groups, and makes screening programmes far more accurate.

The libraries of life: biobanks

A biobank is a structured repository that collects, processes, and stores biological samples such as DNA, blood, tissues, cells, and associated health data. Think of it as a library where the books are human samples, carefully catalogued with the donor's health records, so researchers can check out precisely the right ones for their study.

Biobanks are the backbone of precision medicine because they enable four things at population scale:

• Disease diagnosis and prognosis: comparing a patient's sample against thousands of stored samples to spot disease earlier.
• Development of targeted drugs: finding the genetic variants a new drug must act on.
• Population-based studies: understanding how diseases distribute across ethnic groups.
• Predictive health modelling: forecasting who is at risk before symptoms appear.

India currently has 19 recognised biobanks supported by institutions like the ICMR, the Department of Biotechnology and AIIMS (status as reported in 2025). But the ecosystem has serious gaps: there is no dedicated legal framework, which leaves ownership of samples, usage rights and ethical accountability ambiguous; informed-consent mechanisms are weak, with participants often unaware of how their samples will be used in the future; genetic data faces privacy and security risks including misuse, discrimination and leaks; there is no central oversight body and no penalties for unethical practices; and the system is vulnerable to bio-piracy, where external pharmaceutical firms use Indian samples without fair compensation, eroding national bio-sovereignty.

The way forward has five parts: a comprehensive biobanking law guaranteeing participant rights, transparency and informed consent, aligned with global standards such as the EU's GDPR and the US Biobank Act; a central regulatory authority under the Health Ministry or DBT to monitor operations, data sharing and compliance; bringing genetic data explicitly under the Digital Personal Data Protection Act with stringent penalties for breach or misuse; standardised, multilingual, easily understood consent forms with continuous updates to participants about sample usage; and strategic diplomacy through BRICS, the Quad and the WHO to advocate global ethical standards and project India as a responsible bio-research hub.

When the disease is rare: India's rare-disease policy

A rare disease, according to the WHO, is a condition that affects 1 or fewer individuals per 1000 population. These are often chronic, life-threatening conditions with very few treatment options; examples include Fanconi Anemia and Osteopetrosis. Because patient numbers are tiny, drug companies have little commercial incentive to develop cures, which is why treatments are called orphan drugs.

The issue entered the national spotlight when the Delhi High Court issued strong directions to ensure treatment access for rare-disease patients, reading the Right to Health into Article 21. Then, in January 2025, AstraZeneca's Eculizumab received CDSCO permission to be imported, sold and distributed in India, becoming the country's first anti-complement therapy for rare diseases, indicated for Paroxysmal Nocturnal Hemoglobinuria (PNH) and atypical Hemolytic Uremic Syndrome (aHUS) in both children and adults.

The policy backbone is the National Policy for Rare Diseases (NPRD), 2021, approved by the Union Health Ministry in 2021. It lists 63 rare diseases in three groups:

On financing, the 2021 policy proposed support of up to Rs 20 lakh under the Umbrella Scheme of Rashtriya Arogya Nidhi for Group 1 diseases. By November 2024 the government told the Lok Sabha that patients registered with any of the 12 Centres of Excellence receive financial assistance of up to Rs 50 lakh across all three groups, and that 876 patients had received treatment under the policy. Imported rare-disease drugs also get tax exemptions.

Challenges remain steep: diagnosis is often delayed by years, treatment options are limited, R&D is thin, clinical-trial representation of Indian patients is below 0.1 per cent of global trials, there is no standard national definition or robust epidemiological data, and orphan drugs impose crushing financial strain on families. The way forward is to strengthen the National Rare Diseases Committee and a central information portal, build a national registry, fast-track approvals, expand Centres of Excellence into underserved regions, encourage CSR contributions, allow flexibility in funding caps for Group 3 cases, and incentivise domestic manufacturing of therapies, including through the PLI scheme.

One pill, many drugs: the FDC controversy

A fixed dose combination (FDC) is a pharmaceutical product that contains two or more active ingredients combined in a fixed ratio in a single dosage form. The definition comes from Rule 122-E of the Drugs and Cosmetics Rules, 1945. Familiar examples include Atripla and Vicks Action 500. FDCs are widely used in infectious diseases, cardiovascular disorders, diabetes and asthma.

Well-designed FDCs have real merits. They simplify dosing regimens and improve patient adherence by reducing the pill burden; they minimise medication errors since patients do not juggle multiple drugs; and they can be cost-effective. The problem is the irrational ones: combinations with no therapeutic justification, sometimes mixing a dozen or more ingredients, which expose patients to side effects and drug interactions without added benefit.

India's regulatory record on FDCs has been troubled. State drug controllers have openly licensed FDCs never cleared by the Drugs Controller General of India (DCGI); courts have interpreted the Centre's banning power under Section 26A of the Drugs and Cosmetics Act, 1940 inconsistently; and the widespread use of unapproved, irrational antibiotic FDCs has accelerated antimicrobial resistance, a crisis covered in our companion health article.

The decisive moment came in August 2024, when the government banned 156 fixed dose combinations with immediate effect under Section 26A. The gazette notification of 21 August 2024, based on orders dated 12 August 2024, followed an expert committee and the Drugs Technical Advisory Board (DTAB), which found the combinations irrational, with no therapeutic justification for their ingredients and likely to involve risk to human beings, while safer alternatives existed. The banned list spanned painkillers, antibiotics, multivitamins and cold-and-fever drugs. This is a favourite UPSC theme: the 2013 mains paper asked candidates to discuss the merits and demerits of FDCs.

The long-term fix has three planks: establish a National Drug Authority through an Act of Parliament, as proposed by the Hathi Committee and outlined in the 1994 Drug Policy, to end fragmented Centre-State regulation; conduct periodic surveys of manufacturers and distribution outlets; and enforce severe legal deterrents, on the lines recommended by the Mashelkar Committee, while engaging all stakeholders, consumers, physicians, regulators, industry and academia, against irrational use.

Regulating what we inhale and eat: ENDS and trans fats

Electronic Nicotine Delivery Systems (ENDS) are devices that deliver nicotine via vaporised solutions, including e-cigarettes, vape pens and e-hookahs. E-cigarettes are battery-powered devices that heat e-liquids containing nicotine and flavourings to produce an inhalable vapour, while heat-not-burn devices heat tobacco instead of burning it, releasing a nicotine-containing aerosol. Once marketed as safer alternatives to smoking, these new-age gateway devices have triggered a public-health crisis, especially among youth.

The harms are now well documented: lung injuries grouped as EVALI, short for e-cigarette or vaping product use-associated lung injury; youth addiction driven by candy and fruit flavourings; high addictiveness from nicotine salts, protonated nicotine that allows rapid absorption and stronger cravings; links to anxiety, depression, suicidal thoughts and social isolation; and a gateway effect, with users experimenting with cannabis and other illicit drugs.

India moved early and hard: the Prohibition of Electronic Cigarettes Act (PECA), 2019 banned the production, manufacture, import, export, transport, sale, distribution, storage and advertisement of e-cigarettes. The context is stark: India is the world's second-largest consumer and third-largest producer of tobacco, over 1.3 million deaths a year are attributed to tobacco use (Global Adult Tobacco Survey), and the economic cost is estimated at Rs 1.77 lakh crore, about 1 per cent of GDP (WHO). The remaining agenda is to amend COTPA, the Cigarettes and Other Tobacco Products Act, to ban surrogate advertising; enforce the National Tobacco Control Programme strictly; raise tobacco taxes in line with WHO-FCTC recommendations; support tobacco farmers with crop substitution such as jowar, validated by the Central Tobacco Research Institute; and build real-time, region-specific surveillance of tobacco-use trends.

Trans fats are unsaturated fats that have been artificially altered to make them solid at room temperature, created by adding hydrogen to liquid vegetable oils, a process known as hydrogenation. They raise LDL (bad) cholesterol and lower HDL (good) cholesterol, increasing the risk of heart disease, stroke and type 2 diabetes. Industrially produced trans fats, found in fried foods, baked goods, snacks and frozen dinners, are the most harmful kind; small amounts also occur naturally in beef, lamb and dairy.

India's regulator acted ahead of the global deadline. The Food Safety and Standards Authority of India (FSSAI) capped industrially produced trans fat at 2 per cent by mass of total fat in food products with effect from 1 January 2022, notified in February 2021, after capping oils and fats at 3 per cent in 2021. FSSAI's then-CEO noted India would hit the 2 per cent elimination-equivalent target a year before the WHO deadline. Awareness drives like the Heart Attack Rewind mass-media campaign and the Eat Right India movement backed the regulation.

Globally, the WHO's REPLACE action framework, launched in 2018, gives countries a six-step roadmap to eliminate industrially produced trans fats: review dietary sources, promote replacement with healthier fats, legislate elimination, assess and monitor trans-fat content, create awareness, and enforce compliance. The WHO had called for global elimination by 2023, with best-practice policies limiting industrial trans fat to 2g per 100g of total fat or banning partially hydrogenated oils outright.

The way forward: a smarter health state

Five threads tie this frontier together. First, regulatory coherence: drug regulation is split between the Centre's CDSCO and state drug regulatory authorities, and the FDC saga shows what fragmentation costs, so greater central coordination with empowered state bodies is essential. Second, digital surveillance of drugs, from licensing to adverse-event reporting on a centralised platform. Third, stronger pharmacovigilance, the science of detecting and assessing adverse drug reactions, with wider public and professional participation. Fourth, data infrastructure for precision health: biobanks, registries and genomic datasets governed by clear law. Fifth, fiscal and legal tools against harmful consumption, from tobacco taxes to trans-fat caps. Together they move India from a health system that reacts to disease toward one that predicts, prevents and polices.

Key Terms

• Precision medicine: an approach that tailors healthcare interventions to an individual's genetic makeup, environment and lifestyle, shifting medicine from one-size-fits-all to personalised care; India's programmes target NCDs, rare disorders and pandemics.
• Biobank: a structured repository that collects, processes and stores biological samples such as DNA, blood, tissues and cells along with associated health data; it powers diagnosis, targeted-drug development, population studies and predictive health modelling.
• Phenome India Project: a national project that collected 10,000 samples to build better predictive models for cardiometabolic diseases by linking measured traits to genetic risk.
• PRaGeD Mission: the Paediatric Rare Genetic Disorders mission, which maps genes and variants behind rare childhood diseases to enable targeted therapies.
• Rare disease: a condition affecting 1 or fewer individuals per 1000 population (WHO definition); usually chronic and life-threatening, with examples like Fanconi Anemia and Osteopetrosis.
• National Policy for Rare Diseases (NPRD), 2021: India's rare-disease policy listing 63 diseases in three groups by treatability and cost; it provides financial support through Centres of Excellence and tax exemptions on imported drugs.
• Orphan drug: a medicine developed to treat a rare disease; called 'orphan' because tiny patient numbers give companies little commercial incentive to develop it.
• Anti-complement therapy: a treatment that blocks the complement system, a part of the immune response that attacks the body's own cells in some rare diseases; Eculizumab, approved by CDSCO in January 2025, was India's first.
• Fixed dose combination (FDC): a pharmaceutical product containing two or more active ingredients in a fixed ratio in a single dosage form, defined under Rule 122-E of the Drugs and Cosmetics Rules, 1945.
• Section 26A: the provision of the Drugs and Cosmetics Act, 1940 empowering the Central Government to prohibit the manufacture, sale and distribution of a drug in public interest; used for the August 2024 ban of 156 FDCs.
• Drugs Technical Advisory Board (DTAB): the statutory expert board advising the Centre on drug regulation; its finding that 156 FDCs were 'irrational' with no therapeutic justification led to their ban.
• ENDS: Electronic Nicotine Delivery Systems, devices such as e-cigarettes, vape pens and e-hookahs that deliver nicotine via vaporised solutions.
• EVALI: e-cigarette or vaping product use-associated lung injury, the lung damage linked to vaping that helped establish ENDS as a public-health threat.
• PECA, 2019: the Prohibition of Electronic Cigarettes Act, 2019, which banned the production, import, sale, distribution, storage and advertisement of e-cigarettes in India.
• Trans fat: unsaturated fat artificially solidified by hydrogenation of liquid vegetable oils; it raises LDL and lowers HDL cholesterol, increasing heart-disease risk.
• REPLACE: the WHO's 2018 six-step action framework (review, promote replacement, legislate, assess, create awareness, enforce) for eliminating industrially produced trans fats.
• Pharmacovigilance: the science of detecting, assessing and preventing adverse effects of medicines; India's Pharmacovigilance Programme suffers from low public and professional awareness.
• Bio-piracy: the unauthorised commercial use of a country's biological samples or genetic resources by external firms without fair compensation; a key risk cited against unregulated biobanking.

Prelims practice

1. Consider the following statements about the National Policy for Rare Diseases, 2021: 1. It lists 63 rare diseases in three groups. 2. It was originally proposed to give up to Rs 20 lakh under the Umbrella Scheme of Rashtriya Arogya Nidhi. 3. Group 1 covers diseases amenable to one-time curative treatment. Which of the statements given above are correct? (a) 1 and 2 only (b) 2 and 3 only (c) 1 and 3 only (d) 1, 2 and 3
1. A fixed dose combination (FDC), as defined under Rule 122-E of the Drugs and Cosmetics Rules, 1945, refers to: (a) A drug sold with a fixed maximum retail price (b) A product containing two or more active ingredients in a fixed ratio in a single dosage form (c) A generic drug manufactured after patent expiry (d) A drug approved only for hospital use
1. With reference to trans-fat regulation in India, consider the following statements: 1. FSSAI capped industrially produced trans fat at 2 per cent of total fat in food products from 1 January 2022. 2. The WHO's REPLACE framework was launched in 2018 to eliminate industrially produced trans fats. Which of the statements given above is/are correct? (a) 1 only (b) 2 only (c) Both 1 and 2 (d) Neither 1 nor 2
1. Eculizumab, in the news in January 2025, is significant because it: (a) Is India's first approved mRNA vaccine for adults (b) Became the first CDSCO-approved anti-complement therapy for rare diseases in India (c) Is a biosimilar for diabetes management (d) Is the first Indian drug to receive USFDA fast-track status
1. Which of the following best describes a biobank? (a) A seed vault for endangered plant species (b) A structured repository that collects, processes and stores biological samples such as DNA, blood and tissues with associated health data (c) A digital locker for health insurance records (d) A blood bank network for emergency transfusions