Evolving landscape of haemophilia care in India: Balancing innovation, access, and sustainability

Epidemiology and underdiagnosis

Although the estimated number of persons with haemophilia in India approaches ∼140,000, only about 26,352 patients are currently diagnosed and registered, according to the World Federation of Haemophilia Annual Global Survey (WFH-AGS 2024), highlighting a substantial diagnostic gap in the country.²

From episodic care to prophylaxis: a slow but steady shift

Historically, 96% of patients relied on episodic treatment therapy, resulting in high annual bleed rates, progressive arthropathy, and preventable disability. The introduction of low-dose prophylaxis, validated by Indian studies and later reinforced by the Jawaharlal Institute of Postgraduate Medical Education and Research (JIPMER) randomised trial, marked a turning point.³ Early Indian cohorts demonstrated dramatic reductions in annual bleed rates (from ∼37–40 to 1–4 per yr), improved school attendance, and better functional outcomes.^3,4 However, adoption remains inconsistent. Of the many States implementing low-dose prophylaxis, a few such as Kerala, Karnataka, Tamil Nadu, and Maharashtra have adopted State-funded programmes, whereas many other States continue to rely predominantly on episodic care.^5,6 However, translating these recommendations into practice will require sustained political commitment, reliable supply chains, and continued advocacy. While humanitarian aid programmes currently help bridge a portion of this gap, long-term access must ultimately be secured through strengthened government-supported programmes.

Therapeutic innovations: promise with practical caveats for India

Extended half-life (EHL) products and non-factor therapies are altering the treatment paradigm by reducing infusion frequency and potentially improving adherence, particularly given India’s access constraints and travel burdens. In India, real-world data from observational studies on glycopegylated EHL Factor VIII and Factor IX strategies suggest clinical benefits in reducing bleeding and improving joint status, with a note on gains in adherence and QoL, while also highlighting the need for individualised prophylaxis and laboratory testing (e.g., pharmacokinetic profiling).⁷ These findings support the potential value of EHL strategies in resource-limited settings, provided costs and monitoring are managed.⁷

Emicizumab: A catalyst for equity and innovation

So far, only a few developments have reshaped India’s haemophilia landscape as profoundly as emicizumab. Within the limitations of funding, it was initially prioritised for inhibitor-positive patients. However, its low-dose, low-frequency regimen has proven transformative, particularly in resource-constrained settings.^8,9 Studies from King Edward Memorial (KEM) Hospital, JIPMER, and other centres have demonstrated its cost-effectiveness and real-world impact, prompting several States, most notably Kerala, to incorporate it into public programmes.^10-12 Beyond clinical outcomes, emicizumab has helped address sociocultural barriers, such as enabling safer care for infants and young children, reducing caregiver burden, and improving adherence in families previously overwhelmed by frequent intravenous infusions.^13,14

The role of patient organisations, especially the Hemophilia Federation of India (HFI), has been pivotal in advocating for equitable access and influencing State-level policy decisions.

Many States lack budgetary provisions and continue to face gaps in prophylaxis availability, inhibitor management, and access to bypassing agents. Addressing these disparities is essential to ensure that innovation does not widen inequity.

Decentralisation: A quiet revolution

One of the most encouraging shifts is the decentralisation of haemophilia care. Several States now provide factor concentrates at district and taluka hospitals, reducing travel burdens and improving timely access. Kerala’s model, where prophylaxis continues beyond the age of 18 for those already initiated, demonstrates how policy innovation can directly improve long-term outcomes.¹⁵ However, home therapy, a global standard of care, remains largely unavailable in India, with only a few anecdotal reports. Without home-based infusion capability, even patients on prophylaxis remain vulnerable to delays in bleed management, particularly in rural areas.¹⁵

Gene therapy: Promise tempered by reality

The landmark lentiviral autologous transplant conducted at Christian Medical College (CMC) Vellore represents a milestone, and ongoing work on non-viral vector approaches reflects the country’s growing gene therapy research capacity.¹⁶ Yet, the global landscape has become more cautious. The discontinuation of certain Adeno-associated virus (AAV)-based programmes due to declining factor levels underscores the uncertainties surrounding durability and long-term safety. India faces additional challenges, including a high prevalence of anti-AAV antibodies, limited laboratory infrastructure for long-term monitoring, and the absence of a regulatory framework for gene therapy follow up.^17,18 Gene therapy remains aspirational but not out of reach. Moreover, emerging non-viral and CRISPR-based approaches (LNP-CRISPR) are being explored, though they remain at the preclinical/early clinical stages and require substantial validation and safety data before widespread adoption in India.¹⁹

Rebalancing therapies and the need for laboratory readiness

Rebalancing therapies are expected to enter the Indian market by 2026 and represent the next frontier. However, their safe deployment requires upgraded laboratory capabilities, including validated assays for novel agents, harmonised One-Stage Assay/Chromogenic Substrate Assay (OSA/CSA) protocols, and robust pharmacovigilance systems.²⁰ Many Indian laboratories, especially in district hospitals, which already lack basic haemophilia identification and inhibitor testing, are not geared to monitor these therapies. Without investment in laboratory modernisation, the benefits of innovation will remain confined to a few tertiary centres.

The road ahead: A framework for equitable progress

To translate innovation into impact, India must pursue a coordinated, equity-focused strategy under the following steps: