Risdiplam Patent Expiry and Affordable Treatment for Rare Diseases
Source:The Hindu ↗
Why It Matters
The case of Risdiplam perfectly illustrates the tension between Intellectual Property Rights (patents) and public healthcare access (GS2/GS3). It underscores the role of generic medicines in ensuring the affordability of life-saving treatments for rare diseases in developing countries like India.
Summary
The patent expiration for Risdiplam, a critical drug for Spinal Muscular Atrophy (SMA), paves the way for generic manufacturing in India. This development promises to dramatically lower the drug's previously exorbitant cost—around Rs 70 lakh annually—thereby improving accessibility for patients with rare genetic disorders and easing the financial constraints of public healthcare frameworks.
Key Points
- Risdiplam, a critical oral medication used to treat Spinal Muscular Atrophy (SMA), has gone off-patent, clearing the path for Indian generic manufacturers to produce more affordable versions.
- SMA is a rare, progressive genetic disorder that affects motor neurons and leads to muscle wasting, requiring lifelong and historically exorbitant treatments.
- Previously sold under the brand name Evrysdi by Roche, the drug cost nearly Rs 70 lakh annually, making it inaccessible to most patients without crowd-funding or government aid.
- The patent expiry intersects directly with public health policy, heavily reducing the financial burden on the National Policy for Rare Diseases, which caps financial assistance at Rs 50 lakh per patient.
Important Terms
National Policy for Rare Diseases (NPRD)
A 2021 framework categorizing rare diseases into three groups and providing financial support (up to ₹50 lakh) for treatment at designated Centres of Excellence.
UPSC context
GS Paper 2: Social Justice/Health; tests knowledge of government interventions for vulnerable sections and health infrastructure.
Spinal Muscular Atrophy (SMA)
A rare, life-threatening genetic disorder characterized by progressive muscle wasting and weakness due to the loss of motor neurons in the spinal cord.
UPSC context
GS Paper 3: Science & Tech; focuses on genetic disorders, biotechnology, and the ethics of high-cost life-saving treatments.
Indian Patent Act, 1970
Legislation governing patents in India, featuring unique provisions like Section 3(d) to prevent 'evergreening' and Section 84 for compulsory licensing to ensure public health.
UPSC context
GS Paper 3: IPR; critical for understanding the balance between innovation and drug affordability in India.
Compulsory Licensing
A legal mechanism allowing the government to authorize third parties to manufacture a patented drug without the owner's consent under specific public health emergencies.
UPSC context
GS Paper 3: IPR/Economy; tests understanding of TRIPS flexibilities and India's stance on global trade versus public welfare.
Intellectual Property Rights (IPR)
The legal rights granted to inventors to protect their creations; in pharma, it balances R&D incentives against the need for affordable access to medicine.
UPSC context
GS Paper 3: Issues related to IPR; a recurring theme in trade negotiations and healthcare policy.
Generic Medicines
Pharmaceutical equivalents to brand-name drugs that contain the same active ingredients and are typically sold at significantly lower prices after patent expiration.
UPSC context
GS Paper 2: Health; GS Paper 3: Economy; emphasizes India's role as the 'pharmacy of the world.'
Prelims Trap
NPRD 2021 provides uncapped, lifetime financial support for rare diseases like SMA.
The NPRD strictly caps financial assistance at Rs 50 lakh per patient at designated Centres of Excellence, regardless of whether the disease requires lifelong high-cost therapy.
Static Concepts Connected
How does autosomal recessive inheritance work in genetic disorders like SMA?
- Inheritance requirement: The patient must inherit two defective copies of the mutated gene (one from each parent) to manifest the disease.
- Carrier parents: If parents carry only one defective copy, they remain asymptomatic carriers but have a 25% chance of passing the disease to their offspring.
- Common examples: Besides Spinal Muscular Atrophy, other prominent examples include Sickle Cell Anemia, Cystic Fibrosis, and Thalassemia.
How does the National Policy for Rare Diseases (NPRD) 2021 categorize conditions?
- Group 1: Disorders amenable to one-time curative treatment, such as severe combined immunodeficiency (SCID).
- Group 2: Diseases requiring long-term or lifelong treatment but at a relatively lower cost, and which respond to dietary interventions.
- Group 3: Diseases requiring lifelong, exceptionally high-cost therapies, such as SMA and Pompe disease.
- Funding cap: Up to Rs 50 lakh per patient is provided for treatment at designated Centres of Excellence (CoEs), covering all groups.
Cross-Topic Patterns
Orphan drugs + Indian generic pharma
The transition of rare-disease drugs off-patent highlights India's potential to become the 'pharmacy of the world' for orphan drugs, significantly reducing import dependence for high-cost therapies.
Gene therapies vs. chemical treatments
While Risdiplam treats SMA chemically via lifelong doses, gene therapies (like Zolgensma) aim for one-time genetic cures, representing a shift from NPRD Group 3 (lifelong) to Group 1 (one-time curative) strategies.
Expect a multi-statement question merging NPRD 2021 disease categorizations (Groups 1-3) with genetic inheritance patterns (autosomal).
Linked PYQs · 4
Linked PYQs · 4
- 2024MainsGS3
What is the present world scenario of intellectual property rights with respect to life materials? Although, India is second in the world to file patents, still only a few have been commercialized. Explain the reasons behind this less commercialization.
- 2014MainsGS3
In a globalized world, Intellectual Property Rights assume significance and are a source of litigation. Broadly distinguish between the terms-Copyrights, Patents and Trade Secrets.
- 2024MainsGS2
In a crucial domain like the public healthcare system the Indian State should play a vital role to contain the adverse impact of marketisation of the system. Suggest some measures through which the State can enhance the reach of public healthcare at the grassroots level.
- 2021PrelimsGS1
In the context of hereditary diseases, consider the following statements : 1. Passing on mitochondrial diseases from parent to child can be prevented by mitochondrial replacement therapy either before or after in vitro fertilization of egg. 2. A child inherits mitochondrial diseases entirely from mother and not from father. Which of the statements given above is/are correct?
