Tiny Lives, Big Pharma: Analysing Patent Barriers to the Right to Access Essential Pediatric Medicines

Children’s Right to Access Essential Medicines: Real or Rhetoric?

“Pediatrics does not deal with miniature men and women, with reduced doses and the same class of diseases in smaller bodies, but (…) it has its own independent range and horizon.”

– Abraham Jacobi, 1889

Owing to their immature physiological systems, drug response in children differs from that of adults. This necessitates pediatric-specific formulations. According to the latest census, 24% of India’s population is aged 0–14, thus making pediatric medicines an essential component of public health. However, a medicine's purpose is realised not merely through its development, but through its availability, accessibility, and affordability. These three are mandates embedded as the ‘human right to health’ in the World Health Organisation (WHO) Constitution 1946, the Universal Declaration of Human Rights 1948, the International Covenant on Economic, Social and Cultural Rights (ICESCR) 1966, and Sustainable Development Goal-3. For children specifically, the Convention on the Rights of the Child 1989 enshrines the ‘best interests of the child’ principle in Article 3. It, thus, embodies Access to Essential Pediatric Medicines [“ATEPM”] by implication.

Domestically, this right is both constitutionalised and embedded in policy. In Mohd Ahmed v. Union of India (2014), the Delhi High Court (DHC), drawing on Article 21 of the Indian Constitution (1950), Article 12 ICESCR, and General Comment 14, directed the State Government to provide free Enzyme Replacement Therapy for Gaucher disease. It held that providing affordable essential medicines is a non-derogable obligation enforceable regardless of the State’s fiscal constraints, thus echoing ICESCR’s minimum core doctrine and incorporating progressive realisation standards into right-to-health jurisprudence. Citing Paramanand Katara v. Union of India (1989) and Paschimbangal Khetmazdoor Samiti v. State of West Bengal (1996), it held that poverty cannot justify denying life-saving treatment. Policy-wise, the National Health Policy 2017 aims to ensure accessibility to the highest attainable standard of health across all ages without financial hardship.

Yet a closer examination of ground realities indicates a yawning gap between extolled rights and their actual realisation. This article examines systemic barriers to pediatric medicine access, evaluates existing but flawed mechanisms, and proposes targeted reforms to strengthen and operationalise them.

A System in Crisis: Scrutinising Endemic Violations of the Right to ATEPM

“If access to health care is considered a human right, who is considered human enough to have that right?”

– Paul Farmer, 2013

Despite formal recognition of ATEPM, outcomes indicate a severe disconnect between policy and access. To illustrate, onasemnogene abeparvovec, a gene therapy for Spinal Muscular Atrophy (a rare disease primarily diagnosed in children), is priced around ₹16 crore in India. This renders it inaccessible to even most high-earning households. This scarcity extends beyond orphan drugs to essential pediatric supplements. A 2010 survey across 20 states reported an 80% median ATEPM for five medicines (vitamin A, cotrimoxazole, oral rehydration salt, paracetamol, zinc sulphate). In 2020, a broader survey found only 53% of pediatric formulations to be available in public facilities, forcing caregivers to either rely on costly private treatment, causing high out-of-pocket expenditure, or to forgo care, leading to avoidable pediatric deaths. The human and constitutional right to ATEPM is thus undermined in practice.

Policy obligations are also frequently breached. In 2017, the National AIDS Control Programme (NACO) faced stockouts of pediatric anti-retroviral syrups containing lopinavir and ritonavir due to the government’s non-payment to Cipla. NACO imprudently advised parents to divide adult tablets into eight parts for pediatric doses. Similar shortages owing to non-payments occurred with tuberculosis medicines in 2013, reflecting administrative apathy.

Over the past decade, while corrective steps have emerged, the legal and policy framework for ATEPM remains both inadequate and underutilised, thus warranting examination of these initiatives and devising mechanisms to strengthen their effectiveness.

Long the Path Behind, Longer Still Ahead: Evaluating What Exists, Envisioning What’s Next

Rethinking Reliance on a Failing Opposition Regime

Undoubtedly, several instances demonstrate the merit of the patent opposition regime in achieving ATEPM. In July 2023, the Indian Patent Office rejected Johnson & Johnson’s (J&J) bid to evergreen the anti-tuberculosis drug bedaquiline, after a pre-grant opposition by the Delhi Network of Positive People (DNPP) and a tuberculosis survivor backed by Médecins Sans Frontières (MSF), paving the way for generic medicine availability at far lower costs than the $400 per six-month course. It followed a March 2023 rejection of J&J’s attempt to patent a salt form of bedaquiline. Similarly, in March 2016, MSF had opposed Pfizer’s patent on its pneumonia vaccine, Prevnar 13 (priced at ₹3,900 per dose), leading to its revocation for lack of inventive step.

However, recent trends show generic companies entering licensing deals with originators, enabling the latter to control prices and limiting pharmacy stocks to costly versions, making generics nearly as unaffordable as the original drug. Such commercial dynamics will inevitably cause opposition to Big Pharma patents from the generic industry to wane, allowing many non-meritorious patents to proceed unless challenged by civil society actors such as MSF. The growing dependence on civil society reflects that patent opposition cannot be the sole mechanism to ensure ATEPM.

Compulsory Licensing as an Enfeebled Exception

A bypass to pharmaceutical patents, apart from or after failure of oppositions, is through the TRIPS flexibility of compulsory licensing [“CL”]. India has previously granted CL where public access outweighed the rights of monopoly holders. Mohd Ahmed also alluded to using CL to curb patent-driven price inflation and obtain ATEPM, given the state’s limited resources to fund treatment for all.

However, generics are not a panacea. Firstly, CL does require royalty to be paid against the license, and, as noted earlier, the government has a history of payment failure, which has caused drug shortages. Secondly, India’s generic sector faces serious quality lapses, even when catering to global demand. In 2013, Ranbaxy’s American subsidiary pleaded guilty to selling generics contaminated with glass shards. In 2023, contaminated Indian cough syrups killed dozens of Gambian children, tainted eye drops blinded numerous Americans, and faulty chemotherapy drugs were distributed internationally. These incidents evidence the necessity of a pharmaceutical industry built on transparent, evidence-based medicine. Stringent audits and globally shared safety data will prevent low-priced generics from compromising safety and quality.

The EMLc Discrepancy

One mechanism to ensure transparency in medicine access is the WHO’s Essential Medicines List (EML), which monitors procurement and supply after domestic adoption. However, all five versions of India’s EML have failed to specify pediatric formulations. To address ATEPM, WHO introduced the EML for Children (EMLc) in 2007, with the 9th version listing around 350 medicines. In 2011, the Indian Academy of Paediatrics (IAP), in collaboration with WHO, released India’s first EMLc with only 134 medicines.

Here, two major issues persist. First, entire categories are missing, including medicines for peritoneal dialysis, reproductive health, perinatal care, and mental and behavioural disorders, along with critical omissions of drugs like acyclovir, azathioprine, bedaquiline, cefixime, cisplatin, delamanid, filgrastim, piperacillin-tazobactam, vancomycin, and vaccines for rotavirus, cholera, hepatitis, and typhoid. Second, timely updates are lacking. India’s EMLc is 14 years old, while the EML was last updated 3 years ago. This lag exposes a disconnect between ATEPM commitments and their reflection in EMLs, demanding urgent correction.

Addressing Orphan Drug Access for Children

Rare diseases disproportionately affect children, with about 75% manifesting in childhood. In 2014, Mohd Ahmed noted India’s absence of an orphan drug policy like the United States’ Orphan Drug Act, 1983, or the European Union’s Regulation (EC) No 141/2000 (Orphan Regulation) and 1901/2006 (Paediatric Regulation). India’s legislative and judicial response was slow. Only in Master Arnesh Shaw v. Union of India (2023) did the DHC direct finalisation of the National Health Policy for Rare Diseases and the creation of a National Consortium for Research and Development on Therapeutics for Rare Diseases, a National Rare Diseases Committee, and a National Fund for Rare Diseases.

However, without prioritising orphan drug development, regulation alone is insufficient. Firstly, the patent regime inherently overlooks small patient populations, skewing research toward high-revenue, ‘me-too’, and lifestyle drugs. Pediatric orphan drugs are particularly unattractive economically, as children make up only a quarter of the population and under 10% of the market, resulting in poor drug development from the outset. Secondly, even if developed, high prices make generic distribution of such drugs unfeasible, as Section 84 of the Patents Act, 1970, in practice interprets “public requirement” for CL as requiring mass demand, a condition nearly impossible to be met by pediatric orphan drugs. Even if granted, a generic entry could split the already tiny patient base, undermining incentives for innovation and follow-on manufacturing.

Public R&D under a cost-plus model (where the price is set as production cost plus a fixed profit margin) to incentivise local manufacturers may be more sustainable than a purely private-sector approach. Given that over 61% of orphan drugs originate from small and medium research centres or public institutions and not Big Pharma, cost-effective collaborative initiatives, such as the Council of Scientific and Industrial Research’s (CSIR) Open Source Drug Discovery (OSDD) programme, which crowdsources research to develop affordable drugs, can leverage global partnerships between academia, public institutions, and private actors. With adequate funding and political will, such models can spur innovation, lower costs, and improve ATEPM.

Prize-Based Innovation as a Supplement to the Patent Regime

Prize-based mechanisms have long been proposed to spur pharmaceutical innovation while ensuring affordable access. In 1943, early advocate Michael Polanyi suggested government patent buyouts to reward innovators and place products in the public domain through “Advanced Market Commitments” (AMCs). In 1998, Michael Kremer expanded this, arguing that patents often fail to capture full social value and proposing monetary prizes followed by public-domain release. Unlike Polanyi, Kremer favoured open auctions that incorporated market knowledge in patent valuation rather than relying solely on government assessment. He later combined both approaches for neglected diseases by setting price floors in bidding processes to guarantee minimum incentives and providing additional incentives based on market valuations. This model has seen application, albeit with criticism, in vaccine development.

In 2009, James Love and Tim Hubbard proposed linking remuneration to measurable global health impact and delinking R&D incentives from drug prices, giving innovators a greater incentive to pursue high-social-impact technologies. In 2015, Senator Sanders offered a more practical solution, the “Medical Innovation Prize Fund,” which allocates a fixed share of national income since quantifying health impact is practically challenging. However, Love-Hubbard’s model is fairer long-term as it fosters ongoing competition by basing awards on the relative social value of innovations rather than fixed sums.

For India, the AMC framework could offer significant advantages over CL by, firstly, avoiding ongoing government liability for royalty payments, which are always at risk of default, and secondly, strengthening the state’s capacity to independently uphold ATEPM by placing the invention in public ownership. A practical route would be piloting a lump-sum prize fund while simultaneously creating a formula to measure health impact against benchmarks to enable a gradual shift from Sanders’ to Love-Hubbard’s model.

Conclusion

“Medicine should be viewed as social justice work in a world that is so sick and so riven by inequities."

– Paul Farmer, 2022

The constitutional and human right to ATEPM demands urgent operationalisation for India. Despite jurisprudential advances, India’s record is marked by procurement failures, contamination scandals, and narrow patent flexibilities. EMLc gaps and structural neglect of orphan drugs illustrate a chronic policy deficit. Incremental fixes cannot ensure ATEPM. The government must address regulatory loopholes through a systemic overhaul, shifting toward innovation-linked prize regimes drawn on Polanyi’s patent buyouts, Kremer’s market-valued prize funds, and Love-Hubbard’s health-impact remuneration models, which delink R&D incentives from drug prices, offset sunk costs, and place resulting patents in the public domain. Coupled with R&D collaborations, such a framework would realign pharmaceutical innovation with public health priorities, transforming ATEPM from a piecemeal entitlement into a realised constitutional guarantee.

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This article has been authored by Tejaswini Kaushal, a student at the Dr. Ram Manohar Lohiya National Law University, Lucknow. It is a part of the RSRR's Blog Series on 'Ideas in Motion: Contemporary Frontiers in Intellectual Property Law’, in collaboration with Ahlawat & Associates.