Ensuring Access to Life-Saving Medicines: The Case for Compulsory Licensing in Rare Diseases

Rare diseases pose some of the gravest challenges in healthcare. Though uncommon individually, they collectively affect millions of people worldwide. Often these diseases require specialized, lifelong treatments that come with exorbitant price tags. In India, patients suffering from conditions such as spinal muscular atrophy (SMA), haemophilia, thalassemia, and others face tremendous hurdles obtaining affordable medicines.

This has placed compulsory licensing, a legal provision allowing production of generic versions of patented drugs without the consent of patent holders, firmly in the spotlight as a tool necessary to safeguard public health.​

Understanding Compulsory Licensing and Its Legal Framework in India

Compulsory licensing is embedded in the Indian Patents Act of 1970, under Section 84. It grants the government or third parties the authority to manufacture or import patented drugs without the patent owner’s permission under specific circumstances. One essential condition is that a compulsory license may be issued only after three years from the date of the patent grant. The grounds for granting such a license typically include the drug being unaffordable, insufficiently available to meet public demand, or not adequately worked (produced or imported) within India.

India’s compulsory licensing framework aligns with the Trade-Related Aspects of Intellectual Property Rights (TRIPS) Agreement under the World Trade Organization. The Doha Declaration of 2001 further affirmed the sovereign rights of nations to grant compulsory licenses in the interest of public health, even if there is no declared health emergency.

For rare diseases, this tool is invaluable because many drugs are priced beyond the reach of most Indian patients. Pharmaceutical companies often justify high prices by citing research and development costs, but when those costs create barriers to access, the government can intervene to protect its population.​

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Why Rare Diseases Require Special Consideration

Rare diseases, sometimes called orphan diseases, affect small numbers of people, generally fewer than one in several thousand. Their rarity means drug developers often see little commercial incentive to develop treatments for these conditions. As a result, the few medicines that do exist for rare diseases tend to be extremely expensive and treatments may be unavailable or delayed in lower-income countries like India.

For patients, the stakes are life and death. Lifelong therapies are frequently required, meaning the cost burden multiplies over time. Diseases like SMA, a motor neuron disorder that can severely limit movement and lifespan, highlight the urgency. For such patients, access to drugs like risdiplam, often patented and costly, can determine survival.​

The Reality on the Ground in India

Despite having a compulsory licensing mechanism in place, India has made limited use of it for rare disease medicines. The country’s first compulsory license was issued in 2012 for a cancer drug, setting a precedent that many hope, will be extended to rare disease therapies.

Rare disease patients and advocacy groups have increasingly petitioned the Indian government and judiciary to invoke compulsory licensing provisions for life-saving drugs now priced unreachable for the majority. However, success has been scarce, as pharmaceutical companies vigorously oppose such measures, citing intellectual property rights and investment protection.

This reluctance points to a need for stronger political will and clearer policy frameworks addressing the unique challenges posed by rare diseases. Without such support, the hope of affordable treatments remains elusive for many.​

Public Health, Justice, and Economic Considerations

Compulsory licensing for rare diseases is not only a public health issue; it is deeply intertwined with questions of social justice and equity. Ensuring affordable access aligns with constitutional and human rights principles, safeguarding the right to health for every citizen regardless of economic status.

Economically, access to cheaper medicines through generic competition can reduce the burden on government health programs and families. It can also stimulate local pharmaceutical manufacturing, building domestic capacity and reducing reliance on costly imports.

However, compulsory licenses come with the requirement of adequate remuneration to patent holders, balancing incentives for innovation with public needs. Thoughtful calibration of this compensation, with transparent criteria, is vital to maintain industry confidence while prioritizing patient welfare.​

Complementary Strategies and the Way Forward

Compulsory licensing alone is not a silver bullet. It must be accompanied by policy reforms supporting rare disease research and development, funding mechanisms, and streamlined regulatory pathways. Expanding Centres of Excellence (CoEs), improving rare disease registries, and scaling up government assistance programs are equally important.

Recent court cases underscore the critical role of judiciary and government collaboration in pushing for patient rights and timely access. Combined efforts from government, industry, civil society, and international partners can craft a comprehensive ecosystem that balances innovation, affordability, and patient needs.

India’s example could inspire other countries grappling with rare disease access, showing that well-implemented compulsory licensing frameworks can be part of a compassionate, sustainable health policy.​

Conclusion

The need for compulsory licensing in rare diseases reflects broader tensions in global healthcare: how to reward innovation without compromising access? India’s existing legal provisions, international commitments, and court interventions offer a pathway to reconcile these demands.

For many rare disease patients, invoking compulsory licenses could mean the difference between hope and despair. As awareness and advocacy grow, India stands at a critical juncture, one that requires decisive action to prioritize lives over profits while keeping the wheels of innovation turning. The global community will watch closely as India balances these crucial considerations for its millions of patients in need.​

Author: Amrita Pradhan, in case of any queries please contact/write back to us via email to chhavi@khuranaandkhurana.com or at  Khurana & Khurana, Advocates and IP Attorney.

References

1.     The Patents Act, 1970 (Act No. 39 of 1970), § 84.

2.     The Patents Act, 1970 (Act No. 39 of 1970), § 92.

3.     Controller General of Patents, Designs and Trade Marks, Order Granting Compulsory Licence for Bayer’s Nexavar (Natco Pharma Ltd., 2012), Intellectual Property India, https://ipindia.gov.in/writereaddata/Portal/Images/pdf/Compulsory_License_Order_Nexavar.pdf.

4.     Ministry of Health and Family Welfare, Government of India, National Policy for Rare Diseases, 2021, https://main.mohfw.gov.in/sites/default/files/Final%20NPRD%2C%202021.pdf.

5.     Ministry of Health and Family Welfare, Government of India, Notification on Rare Diseases – List of Diseases and Centres of Excellence, Gazette of India, S.O. 1488(E), March 30, 2021, https://main.mohfw.gov.in/sites/default/files/Rare%20Diseases%20Policy%20Notification.pdf.

6.     World Health Organization (WHO), Rare Diseases and Orphan Medicines – Global Report on Access to Essential Medicines, 2022, https://www.who.int/publications/i/item/9789240052963.

7.     Organisation for Rare Diseases India (ORDI), India Rare Disease Report 2024 – Burden, Access Gaps and Policy Recommendations, https://www.ordindia.in/wp-content/uploads/2024/03/India-Rare-Disease-Report-2024.pdf.

8.     Indian Council of Medical Research (ICMR), Rare Diseases Registry of India – Current Status and Challenges, 2023, https://rarediseases.icmr.org.in/reports/Rare_Diseases_Registry_India_2023.pdf.

9.     Press Information Bureau (PIB), Government of India, National Policy for Rare Diseases 2021 – Provision of Financial Support up to ₹50 Lakh per Patient, March 30, 2021, https://pib.gov.in/PressReleasePage.aspx?PRID=1708213.

10.  Department of Pharmaceuticals, Government of India, Report of the Committee on High Trade Margin Drugs – Pricing of Orphan Drugs for Rare Diseases, 2022, https://pharmaceuticals.gov.in/sites/default/files/Report%20of%20Committee%20on%20High%20Trade%20Margins.pdf.

11.  United Nations Committee on Economic, Social and Cultural Rights, General Comment No. 14: The Right to the Highest Attainable Standard of Health (Article 12 of the ICESCR), E/C.12/2000/4, August 11, 2000, https://www.ohchr.org/en/documents/general-comments-and-recommendations/general-comment-no-14-right-highest-attainable.