Named Patient Program in India: A Lifeline for Accessing Unavailable Medicines
With a population of 1.3 billion, India carries a significant burden of rare diseases, with estimates suggesting that 70%-96% people may be affected. These patients face multiple barriers to treatment. Most rare diseases – around 80% have a genetic basis – remain poorly recognized, and awareness among both healthcare providers and the public is limited.1
Unlike the US and EU, India still lacks a national definition of rare diseases1 and a dedicated orphan drug policy.2 Without such a framework, pharmaceutical companies have little incentive to develop or manufacture these treatments locally, even though India represents a large potential market.2 As a result, most of the orphan drugs are manufactured abroad and take years to become available in India. As a result, Indian patients must rely almost entirely on importing them, which are typically expensive. This situation drives up costs for families, leading to heavy out-of-pocket spending and significant emotional and financial stress.1
In this context, the Named Patient Program (NPP) provides a regulated pathway for patients to access lifesaving medicines that are otherwise unavailable in India.
What is the Named Patient Program?
The NPP allows patients, on the recommendation of a registered medical practitioner (RMP), to legally import small quantities of medicines not approved or marketed in India for personal use. This pathway operates under Rule 36 of the Drugs & Cosmetics Rules, 1945 through Form 12A (application) and Form 12B (permit) issued by the Central Drugs Standard Control Organisation (CDSCO).3,4
Legal Framework and Policy for Rare Diseases
In India, the National Policy for Rare Diseases, introduced by the Ministry of Health and Family Welfare, sets out both short- and long-term measures to address rare diseases in a comprehensive manner. The policy supports the creation of a National Rare Disease Registry to collect and maintain patient data, which will guide policy, planning, and delivery of care. It also encourages the establishment of National Centres of Excellence to serve as referral hubs for diagnosis, treatment, and research on rare diseases.5 In addition, the policy provides up to ₹50 lakhs per patient as financial assistance for the treatment of certain listed conditions. However, because only a limited set of rare diseases are covered, many patients are still unable to benefit from this support.5,6
In India, the existing provisions under the Drugs and Cosmetics Act, 1940, and the Drugs and Cosmetics Rules, 1945, allow for the regulated personal import of medicines that are not approved or marketed in India.3,4
- Rule 36 of the Drugs & Cosmetics Rules, 1945: Permits the import of small quantities of unapproved drugs for personal use3,4
- Rule 122-A: Pertains to new drugs and the requirement for approval before importation and use in India7
- Rule 123: Provides exemptions for certain drugs from the provisions of Chapter III of the Act, which deals with import requirements7
This framework provides patients with serious or life-threatening conditions a clear, legal channel to access essential medicines that aren’t yet available in India, while ensuring regulatory oversight for safety and accountability at every step.3,4
Benefits of the Program for Rare and Specialized Treatments
Importing drugs for personal use provides the following benefits:
- Timely access to global innovations otherwise delayed in India by regulatory and pricing processes1,3
- Full exemption from basic customs duty on all such drugs imported for treating rare diseases, providing substantial financial relief to patients and their families8
How to Avail the NPP Pathway?
The NPP is generally used when3,4
- The patient has a serious, rare, or life-threatening condition
- The medicine is not approved or unavailable in India or has been withdrawn from the market
This pathway has been especially critical for patients with rare genetic, neuromuscular and oncology conditions, where lack of drug availability hinders treatment.1
Step-by-Step: How to Apply for a NPP Import
The procedure for importing small quantities of drugs for personal use is as follows4:
- Prescription from the RMP: RMP prescribes and justifies medical necessity.
- Application: Submit Form 12A with supporting documents to CDSCO.
- Permit: CDSCO issues Form 12B, typically within 1-2 working days when documentation is complete.
Documents Required from Patients and Physicians
- From the physician4: Prescription containing MCI registration number of the physician and the name, quantity and duration of treatment
- From the patient4: Clinical records, ID/address proof
- From the supplier4: An invoice or other statement showing the name and address of the manufacturer and the names and quantities of the drugs
Common Challenges
Personal importation involves extensive paperwork and legal complexity. While the Drugs and Cosmetics Act and Rules lay down the procedures, complying with them can be burdensome. The CDSCO regulates these processes, but requirements differ depending on whether the drug is approved or unapproved – the latter facing stricter scrutiny. This variability and procedural rigidity underline the constant need for regulatory updates that balance ease of access with transparency and safety.9
Medications Accessed via NPP
Some of the medicines that can be accessed via the NPP include Patisiran for hereditary amyloidosis related to transthyretin, Alprolix for haemophilia B and Bosulif for chronic myeloid leukaemia.10 Other rare diseases for which medications can be imported include thalassemia, sickle cell anaemia, primary immunodeficiencies and haemangiomas.2
Role of Rx4U in Facilitating NPP Access
Organizations like Rx4U support patients and physicians by:
- Preparing Form 12A/12B documentation
- Liaising with CDSCO and Customs
- Ensuring compliant, temperature-controlled shipping
- Tracking regulatory updates and approval timelines
- This makes the pathway smoother for families already burdened with high disease-related stress.
Final Takeaway
- Rare disease patients in India often struggle with limited treatment options and high costs.
- The Named Patient Program offers a legal, regulated route to access life-saving medicines not available locally.
- With the right guidance, families can navigate the process more smoothly and get timely access to essential care.
Frequently Asked Questions
1. Is the Named Patient Program legal?
Yes, under Rule 36 of the Drugs & Cosmetics Rules, 1945, with CDSCO permits.
2. How quickly is the permit issued?
CDSCO indicates priority processing, often 1-2 days if papers are complete.
3. Are duties applicable?
They are generally exempt for life-saving medicines.
Note:
The information provided is for education purpose only and is subjected to prescribing information of the drug and the guidance of your treating physician. Always consult your health care provider before making any medical decision for starting your treatment.
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.
References:
1. Rajasimha H. Tackling rare disease in India: each stakeholder of the healthcare and life sciences ecosystem should take responsibility for the millions suffering from rare diseases worldwide. Inside Precision Med. 2023;10(3):32‑35.
2. Chaudhary N, Shukla VK. Challenges in orphan drug development and regulatory requirements in India. Int J Drug Regul Aff. 2020;8(1):31-5.
3. Patil S. Early access programs: Benefits, challenges, and key considerations for successful implementation. Perspect Clin Res. 2016;7(1):4-8. doi:10.4103/2229-3485.173779
4. Central Drugs Standard Control Organisation. Procedures To Import Small Quantities of Drugs for Personal Use: Notice. Ministry of Health & Family Welfare, Government of India. February 19, 2015. Accessed August 25, 2025. https://cdsco.gov.in/opencms/resources/UploadCDSCOWeb/2018/UploadConsumer/ProcedureforpermissiontoImportSmallQuantitiesofDrugsForPersonalUse_link.pdf
5. Kar A, Sundaravadivel P, Dalal A. Rare genetic diseases in India: steps toward a nationwide mission program. J Biosci. 2024;49:34.
6. Venugopal N, Naik G, Jayanna K, et al. Review of methods for estimating the prevalence of rare diseases. Rare Dis Orphan Drugs J. 2024;3:5. doi:10.20517/rdodj.2023.39
7. Ministry of Health and Family Welfare, Government of India. The Drugs and Cosmetics Act, 1940 and the Drugs and Cosmetics Rules, 1945. December 31, 2016. Accessed August 25, 2025. https://www.nitiforstates.gov.in/public-assets/Policy/policy_files/ANC509Q000002.pdf
8. Customs duty full exemption for all imported drugs & food for special medical purposes for personal use for treatment of all rare diseases. News release. Press Information Bureau – Delhi; March 30, 2023. Accessed August 25, 2025. https://www.pib.gov.in/PressReleasePage.aspx?PRID=1912095
9. Budhwar V, Rohilla Y, Yadav V, Choudhary M. Drugs import procedure in India: a comprehensive review. J Pharm Tech. 2019;12(2):869-72. doi:10.5958/0974-360X.2019.00148.3
10. Gahilod RS, Veeranna B, Thakre S. Orphan drug development policies in India and the United States: a comparison. Ind J Pharm Edu Res. 2023;57(2):329-336.
