India's Rare Disease Shift Driven by PRIP and PLI Policies

Saransh Chaudhary, President, Global Critical Care, Venus Remedies Ltd & CEO, Venus Medicine Research Centre, engaged in a conversation with Thiruamuthan, Assistant Editor at India Pharma Outlook, discusses how India is working to improve rare disease treatment. It covers government policies, healthcare facilities, patient support, and industry efforts. He also discusses challenges in research, manufacturing, and diagnostics and the steps being taken to make therapies more available and affordable.

How are PRIP and PLI shifting India’s current rare disease landscape?

The PRIP and PLI schemes mark a turning point in how India approaches rare diseases, but they reveal a policy tension. PRIP focuses on value and innovation by explicitly including rare diseases, orphan drugs, gene therapies and precision medicines in a national mandate. This shifts rare diseases from CSR-type interventions to strategic areas for long-term growth. It recognizes the unsustainability of dependence on costly imported orphan therapies and prioritizes early research, platform technologies, collaborations and locally relevant pipelines.

PLI, in contrast, is about volume and scale. It offers performance-linked incentives for manufacturing high-value products, supporting capacity expansion and supply chain development. However, its volume-driven logic fits poorly with rare diseases, where low-volume, high-precision access defines the market.

PRIP pushes early innovation while PLI pulls toward high-output products. Without an incentive bridge tailored to rare diseases, many projects may fail between lab and market, despite both schemes’ potential to build strategic autonomy.

How prepared are Indian firms to build end-to-end capabilities for complex orphan molecules?

India is transitioning from being the world’s workbench for generics to steadily building capabilities in biologics and biosimilars. Indian companies hold a meaningful share of the global biosimilars market, and leading firms are investing in R&D pipelines for complex biologics, including those relevant to rare diseases.

However, there remains a gap in “end-to-end capability” for orphan molecules. Manufacturing strength is high—India runs large, efficient facilities, validates processes, and meets global quality standards, often excelling as contract development and manufacturing organizations. Where gaps exist is at the biology-first end: owning proprietary discovery platforms for target identification, validation, and screening. This requires deep translational biology, strong clinician-scientist collaboration, and a culture of early scientific risk.

Historically, Indian pharma focused on reverse engineering and incremental innovation, producing vectors while licensing the underlying genetic payload from abroad. This is especially evident in advanced modalities like gene and cell therapies.

Government and industry initiatives—National Biopharma Mission, BIRAC programs, PRIP, and PLI—are addressing these gaps through lab infrastructure, shared testing facilities, bio clusters, and early funding. Indian firms are reliable manufacturing partners for complex orphan molecules, but full in-house end-to-end capabilities remain a work in progress.

With newborn screening still limited, how is India addressing diagnostic delays for rare diseases?

India is working on reducing delays in diagnosing rare diseases, even though universal newborn screening is not yet in place. The National Policy for Rare Diseases and Centers of Excellence provides a clearer framework. These centers, including hospitals like AIIMS Bhopal, offer neonatal screening, genetic testing, counselling, and multidisciplinary care, helping patients reach specialists faster.

The government is also setting up Nidan Kendras and improving diagnostic labs with better molecular and metabolic testing. Gene-based diagnostics and advanced sequencing are being used in both academic and private labs. Training programs are helping doctors and pediatricians spot rare diseases earlier.

However, challenges remain. Many centers are overcrowded or poorly connected, and families face administrative hurdles, such as meeting eligibility rules, securing funding, and waiting for treatment slots. Digital tools like the Ayushman Bharat Digital Mission could help, but without a national registry linked to care and funding, their potential is limited.

Overall, India is making progress with better testing, more specialized centers, and improved referral systems. The next step is designing a system where once a child is diagnosed, treatment can begin quickly without unnecessary delays.

How significantly are patient organizations influencing trials, access and pricing for orphan therapies?

Patient organizations have become a powerful force in India’s rare disease space. Groups like the Organization for Rare Diseases India and condition-specific alliances are now actively shaping policy, research, and access strategies. They played a key role in pushing the government to announce the National Policy for Rare Diseases, set up dedicated funds, and establish Centers of Excellence.

In research, these organizations partner with clinicians and industry to design clinical trials, refine eligibility criteria, select meaningful endpoints, and support patient recruitment. They also help families access compassionate use programs, early access schemes, and crowdfunding options for costly or unavailable therapies.

However, challenges remain. Many Centers of Excellence underutilize allocated funds, and patient groups have limited influence in pricing and reimbursement discussions due to the absence of a fully empowered health technology assessment system.

Patient organizations have moved from observers to agenda setters, and their next goal is gaining structured influence to ensure timely, affordable therapies reach the patients they represent.

Which infrastructure gaps most constrain near-term scale-up of gene therapy platforms in India?

India’s gene therapy story is gaining momentum with early clinical efforts, indigenous CAR T programmes, and growing investor interest. Scaling domestic platforms faces key constraints.

The primary bottleneck is limited domestic capacity for GMP-grade viral vector manufacturing. High-quality vectors like AAV and lentivirus require specialized containment, validated processes, rigorous testing, and skilled staff. Currently, Indian developers often rely on overseas manufacturers, facing high costs and long delays that can hinder early-stage programmes.

Regulatory clarity is another challenge. Though steps like the New Drugs and Clinical Trials Rules recognize advanced therapies, developers face uncertainty on chemistry, manufacturing, controls, potency assays, bridging data, and post-marketing surveillance. Many regulators refer to US or European standards, which is less helpful for novel locally driven science.

Clinical infrastructure and trained human capital are also limited. Gene therapy requires centers that can manage complex procedures and multidisciplinary teams. Only a few tertiary hospitals in India can consistently provide this care.

Training clinicians, strengthening regulatory capacity, and building GMP facilities are ongoing solutions, but developing a mature vector manufacturing ecosystem will take time and investment.

Which factors will most determine whether India becomes a global hub for affordable rare disease innovation?

India has a real chance to become a global hub for affordable rare disease innovation, but success is not guaranteed. Cost engineering, shown by the indigenous CAR T therapy, is a strength, but regulatory sovereignty is crucial. India must independently evaluate therapies, maintain strong registries, and use data effectively. Patient organizations and clinicians shaping research and access are key. Sustained capital, clear reimbursement pathways, and innovative payment models are needed. If PRIP funds discovery, PLI builds manufacturing, regulators provide timely approvals, and payers ensure access, India can move from a low-cost producer to a leader in creating and delivering affordable, high-quality rare disease therapies.