Why are Some Life-Saving Medicines Unavailable in India?

India is often called the “pharmacy of the world.”¹ It supplies affordable generic medicines to over 200 countries and plays a crucial role in global healthcare.² Yet, paradoxically, many lifesaving and breakthrough medicines are not readily available to Indian patients.³

For families battling rare diseases, advanced cancers, autoimmune disorders, or genetic conditions, this gap can mean delayed treatment—or no treatment at all. Doctors, too, face frustration when evidence-based therapies exist globally but are inaccessible locally.⁴

So why does this happen? The answer lies at the intersection of regulation, economics, clinical data, and intellectual property.⁵

This blog explores why some critical medicines are unavailable in India, how this impacts patients and clinicians, and what pathways exist to bridge this gap.

The Paradox: Global Innovation vs Local Access

Medical innovation has accelerated rapidly in the last two decades. Gene therapies, monoclonal antibodies, targeted cancer drugs, and precision biologics are transforming outcomes worldwide.⁶

However, innovation alone does not guarantee access.⁶

A medicine approved by the US FDA or the European Medicines Agency (EMA) does not automatically become available in India. Each country has its own regulatory, economic, and public health considerations.⁷

In India, the challenge is not the lack of scientific capability—but the complex pathway from global approval to local availability.⁷

1. Regulatory Approval Takes Time—and Data

Before any medicine can be marketed in India, it must be approved by the Central Drugs Standard Control Organisation (CDSCO).⁸

Why this matters^8,9:

• CDSCO often requires India-specific clinical data
• Global trial populations may not include enough Indian patients
• Bridging studies or local trials add time and cost

Real-world impact: For very-rare diseases or niche cancer subtypes, conducting Indian trials may be scientifically impractical or commercially unviable. As a result, companies may delay or avoid filing for approval, even if the drug is lifesaving.⁹

2. Small Patient Populations, High Development Costs

Many unavailable medicines are designed for rare genetic disorders, specific cancer mutations, advanced or refractory disease stages. These drugs often cost hundreds of crores to develop but serve very small patient populations in India.¹⁰

From a manufacturer’s perspective, launching such drugs in India may not be financially sustainable—especially without pricing flexibility.¹¹

3. Intellectual Property and Patent Challenges

India’s patent laws prioritize public health and access. While this approach has enabled affordable generics, it also creates concerns for innovators.¹²

Common challenges include patent rejections or delays, risk of early generic competition and uncertainty around data exclusivity.¹²

4. Limited Awareness and Diagnostic Gaps

Sometimes, medicines are unavailable simply because patients are not being diagnosed.⁹

Examples:

• Rare diseases mistaken for common conditions
• Genetic testing not routinely performed
• Lack of referral pathways to specialists

Without reliable epidemiological data, pharmaceutical companies may underestimate disease burden in India, leading to low prioritization for launches.

5. The Role of Global Launch Strategies⁷

India is rarely a first-launch country.

Pharmaceutical companies typically prioritize: US, Europe, Japan, or select high-income markets

India often enters the picture later—sometimes years later—especially for specialized therapies.

This delay can be devastating for patients with progressive or life-threatening diseases.

How Does This Affect Patients?

For patients and families, the consequences are deeply personal¹³:

• Delayed or interrupted treatment opportunities
• Disease progression that could have been prevented
• Financial exhaustion from importing medicines
• Emotional distress from knowing a treatment exists—but is unreachable

Many patients turn to: Crowdfunding, Medical tourism, Legal petitions, Compassionate use requests

Each route is complex, uncertain, and emotionally draining.

How Does This Affect Doctors?

For clinicians, especially specialists, this gap creates ethical and professional dilemmas¹³:

• Knowing the global standard of care but unable to offer it
• Navigating off-label or suboptimal alternatives
• Managing patient expectations and distress

Existing Pathways to Access Unavailable Medicines¹⁴

Despite these challenges, access is not always impossible.

1. Named Patient Programs (NPPs)

These allow doctors to import medicines for individual patients when no approved alternative exists in India.

Commonly used for: Oncology, Rare diseases and Advanced biologics

2. Compassionate Use / Expanded Access

Manufacturers may provide drugs free or at reduced cost for critical cases.

3. Personal Import Licenses

Patients can import medicines with: Doctor’s prescription, CDSCO approval and Customs clearance

4. Early Access Programs

Some companies introduce medicines in controlled settings before full commercial launch.

What Is Changing for the Better?

India is gradually improving access through^15,16:

• Faster regulatory pathways for rare diseases
• Orphan drug policies
• Increased genetic testing and awareness
• More structured Named Patient Programs

The Way Forward: Bridging the Gap¹⁵

Improving access to life-saving medicines in India requires collaboration.

Key steps forward:

• Regulatory agility without compromising safety
• Innovative pricing models (tiered pricing, outcome-based pricing)
• Stronger rare disease registries
• Physician education and advocacy
• Patient-centric policies

India has the scientific talent, manufacturing strength, and clinical expertise. What’s needed is a system that aligns innovation, affordability, and access.

Conclusion: Hope Beyond Barriers

Understanding these barriers empowers patients to ask the right questions and enables doctors to explore alternative pathways. Most importantly, it highlights the need for shared responsibility among regulators, industry, healthcare providers, and society.

For many patients, access delayed is access denied. Bridging this gap is not just a policy issue—it is a moral imperative.

Frequently Asked Questions (FAQs)

Q. Does “unavailable in India” mean the medicine is unsafe?

A: No. Unavailability does not mean unsafe. The reasons are usually related to regulatory requirements, high costs, small patient populations, or business challenges—not safety concerns.

Q. Can I still access a medicine that is not approved in India?

A: Yes, in many cases access is possible through special routes, such as: Named Patient Programs (NPPs)

Q. What is a Named Patient Program (NPP)?

A: A Named Patient Program allows a doctor to request import of a specific medicine for an individual patient when no suitable alternative is available in India.

Q. Is importing a medicine legal in India?

A: Yes. Importing medicines for personal use is legal when done as per the rules.

Reference:

- Subramaniam N, Osoro I, Rajanandh MG, MG R. Lessons pharmacy practice in India should adopt from advanced nations: a review. Cureus. 2024;16(8).

- Guerin PJ, Singh-Phulgenda S, Strub-Wourgaft N. The consequence of COVID-19 on the global supply of medical products: Why Indian generics matter for the world?. F1000Res. 2020;9:225.

- Sunyoto T, Potet J, Boelaert M. Why miltefosine—a life-saving drug for leishmaniasis—is unavailable to people who need it the most. BMJ Glob Health. 2018;3(3):e000709

- Gimenez-Lozano C, Páramo-Rodríguez L, Cavero-Carbonell C, et al. Rare diseases: needs and impact for patients and families: a cross-sectional study in the Valencian Region, Spain. Int J Environ Res Public Health. 2022;19(16):10366.

- Rajaram S, Medhi B. Orphan drugs: The final frontier in the fight against rare diseases. Indian J Pharmacol. 2025 Nov 1;57(6):367-70.

- Olaghere J, Williams DA, Farrar J, et al. Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence. Biomedicines. 2025;13(3):758.

- Konwar M, Maurya MR, Nishandar TB, Thatte UM, Gogtay NJ. An evaluation of drug lag for new drugs approved by the Indian regulator relative to the United States, European Union, and Japanese regulatory agencies: A 15-year analysis (2004–2018). Perspect Clin Res. 2021;12(3):159-64.

- Bhatt A. Assessing unmet medical need in India: A regulatory riddle?. Perspect Clin Res. 2015;6(1):1-3.

- Mishra S, Bhat D, Venkatesh MP. Navigating health policies and programs in India: exploring opportunities to improve rare disease management and orphan drug research. Orphanet J Rare Dis. 2024;19(1):446.

- Rajueni K, Chakraborty Choudhury M. Assessment of the availability of repurposed orphan drugs in India. PLOS Glob Public Health. 2023;3(9):e0001498.

- Simoens S. Pricing and reimbursement of orphan drugs: the need for more transparency. Orphanet J Rare Dis. 2011;6(1):42.

- Priyam S, Awasthi A. Pharmaceutical patenting in India-problem of public access to health. IJLRA. 2024;2(7).

- Shukar S, Zahoor F, Hayat K, Saeed A, Gillani AH, Omer S, Hu S, Babar ZU, Fang Y, Yang C. Drug shortage: causes, impact, and mitigation strategies. Front Pharmacol. 2021;12:693426.

- Patil S. Early access programs: Benefits, challenges, and key considerations for successful implementation. Perspect Clin Res. 2016;7(1):4-8.

- Chirmule N, Feng H, Cyril E, Ghalsasi VV, Choudhury MC. Orphan drug development: Challenges, regulation, and success stories. J Biosci. 2024;49(1):30.

- Sheth J, Nair A, Sheth F, et al. Burden of rare genetic disorders in India: twenty-two years’ experience of a tertiary centre. Orphanet J Rare Dis. 2024;19(1):295.

Note:

This information is provided for educational purposes only and is not a substitute for professional medical advice. Patients should always consult their doctor or pharmacist for advice on diagnosis, treatment, and medication use, and should not make changes to their prescribed therapy without medical supervision.

Disclaimer:

Rx4U procures prescribed medicines directly from manufacturers or authorized distributors. It does not claim ownership of any trademarks and complies with the provisions of the Trademark Act, 1999, particularly Sections 30 and 30(1) concerning ‘Fair Use’. It solely facilitates access to new launches through named patient import.