Accessing Rare Disease Medications in India: What Patients Should Know
05:04:53 April 3, 2025
For patients living with rare diseases, the journey to diagnosis is often long and complex—but the search for appropriate treatment can be even more challenging. In India, where many rare disease treatments are not readily available, patients and caregivers often find themselves navigating a maze of uncertainty, regulations, and limited resources.
However, there is hope. Thanks to global partnerships, compassionate-use frameworks, and access management solutions like those offered by Global Rare Meds, patients can obtain life-saving therapies that are not approved or available locally. This article aims to shed light on how patients in India can access rare disease medications, and what they need to know about the process.
The Challenge of Rare Diseases in India
A rare disease is defined as a condition that affects a small percentage of the population—typically fewer than 1 in 10,000 people. However, collectively, rare diseases affect over 70 million people in India, according to estimates from the Ministry of Health.
Some of the most common rare diseases seen in India include:
- Duchenne Muscular Dystrophy (DMD)
- Gaucher Disease
- Pompe Disease
- Fabry Disease
- Spinal Muscular Atrophy (SMA)
- Mucopolysaccharidosis (MPS)
- Primary Immunodeficiencies
- Batten Disease
Most of these conditions are genetic, chronic, and often life-threatening. Unfortunately, due to limited commercial viability and regulatory hurdles, many treatments approved abroad are not yet available in India through regular channels.
Why Aren’t These Medications Easily Available?
There are several reasons why rare disease drugs (also known as orphan drugs) may not be accessible in India:
- High R&D costs and low demand discourage pharmaceutical companies from launching these drugs in developing markets.
- Lengthy approval timelines delay local availability, even when drugs are approved by agencies like the US FDA or EMA.
- Limited reimbursement or funding options make the drugs unaffordable for most families.
- Lack of awareness among healthcare providers and patients further complicates access.
Despite these challenges, patients can still legally access such drugs through specific regulatory provisions designed for personal, non-commercial use.
The Legal Pathway: Named Patient Import in India
In India, the Central Drugs Standard Control Organization (CDSCO) permits the import of unapproved or unavailable medicines for individual patients under a provision known as the Named Patient Programme (NPP).
This allows patients with life-threatening or seriously debilitating conditions to access medications that are:
- Approved in other countries but not yet in India
- Unavailable due to commercial or logistical reasons
- Prescribed by a qualified doctor as a last resort
Global Rare Meds facilitates this process by working with international distributors, licensed suppliers, and regulatory authorities to ensure safe and timely access to rare disease medications for Indian patients.
How the Process Works
Here’s a step-by-step overview of how patients can access rare disease drugs through legal channels in India:
1. Medical Prescription & Justification
The patient’s doctor must issue a prescription and a justification letter confirming the diagnosis, the necessity of the medication, and the lack of alternatives in India.
2. Consent & Documentation
The patient or caregiver provides consent along with relevant medical documents, identity proof, and application forms required by CDSCO.
3. Regulatory Approval
An import license (usually under Form 12B) is applied for and granted by CDSCO, specifically for personal use.
4. Drug Sourcing & Import
Global Rare Meds sources the medication from approved global suppliers and manages the import process, including cold-chain logistics if needed.
5. Delivery
Once cleared through customs, the medicine is delivered to the treating doctor or hospital for administration.
Why Choose Global Rare Meds?
We are not a pharmaceutical manufacturer—we act as a specialized access partner who collaborates with global medicine distributors and healthcare providers to ensure that patients in India receive the drugs they need.
Our Strengths Include:
- Access to hard-to-find rare disease medications
- Partnerships with reliable, licensed international suppliers
- Expertise in regulatory compliance and documentation
- End-to-end cold chain and logistics management
- Deep understanding of therapy areas like Neurology, Immunology, Hematology, and Lysosomal Storage Disorders
Our goal is to bridge the access gap and give patients a real chance at a better quality of life.
Important Considerations for Patients
- These medications are not for resale or general distribution—they are imported for specific patients with valid prescriptions.
- The process can take 2–4 weeks, depending on drug availability and regulatory approvals.
- Some rare disease drugs are extremely expensive. Patients may explore government subsidies, NGOs, or crowdfunding for financial support.
- Medical supervision is essential. These drugs must be administered or monitored by a qualified healthcare provider.
Real Lives, Real Impact
Through our support, patients across India have gained access to medications for DMD, Batten Disease, Gaucher Disease, and more. Every case is different—but our commitment remains the same: to help every eligible patient access the treatment they need with dignity, speed, and compliance.
Final Thoughts
Living with a rare disease shouldn’t mean living without options. While there’s still a long way to go in terms of systemic support for rare diseases in India, Named Patient Access offers a legal, ethical, and life-saving solution for those who can’t afford to wait.
At Global Rare Meds, we’re proud to walk this path with patients, doctors, and caregivers—turning global innovation into local access, one patient at a time.
