ORPHAN
DRUGS – SCIENCE & TECHNOLOGY
NEWS:
India
faces major hurdles in making orphan drugs accessible and affordable. Countries
like the U.S. and EU have more established frameworks to support orphan drug
development, whereas India’s progress is comparatively limited.
WHAT’S IN THE NEWS?
Understanding Orphan Drugs
- Definition:
Orphan drugs are pharmaceuticals specifically designed to treat rare or
"orphan" diseases.
- Disease
Impact: While these diseases affect a small
population, they often lead to severe, life-threatening, or chronic
conditions.
- Regional
Definitions:
·
United States:
Defines a rare disease as one affecting fewer than 200,000 people.
·
European Union:
Considers diseases affecting fewer than 1 in 10,000 people as rare.
·
India’s Approach:
India lacks a clear prevalence-based definition, but the National Policy for
Rare Diseases (NPRD) of 2021 provides a framework to identify and treat rare
diseases.
Challenges Due to Lack of Definition
- Identification
Difficulty: Absence of a formal definition in
India complicates identifying orphan drugs and addressing patient needs.
Classification of Orphan Drugs
- Disease-Based
Classification:
·
Genetic Disorders:
Conditions like cystic fibrosis and Duchenne muscular dystrophy, typically
requiring lifelong treatment.
·
Rare Cancers:
Includes cancers such as neuroblastoma and gliomas, which are uncommon and
often lack specific treatments.
·
Metabolic Disorders:
Diseases like Gaucher’s disease and Fabry disease, caused by enzyme
deficiencies.
·
Autoimmune Diseases:
Conditions like systemic sclerosis, where the immune system attacks the body's
tissues.
- Regulatory
Status Classification:
·
Approved Orphan Drugs:
Drugs that have passed regulatory approval from bodies like the FDA or EMA,
indicating proven safety and efficacy.
·
Orphan Drug Candidates:
Drugs still in clinical trials, undergoing tests to establish their safety and
effectiveness before approval.
Difficulties in developing drugs in India
·
Even though
India produces all 450 of the world’s orphan medications (APIs), the majority
of these medications are not readily available and are not reasonably priced
here.
·
Developing orphan
drugs is expensive, and the small number of patients makes it hard for
companies to profit.
·
Since rare diseases
affect few people, finding enough participants for clinical trials is
challenging.
·
Many treatments are
very expensive, such as enzyme replacement therapies (ERTs), costing crores of
rupees per year, making them unaffordable for most Indian families.
India’s National Policy for Rare Diseases
(NPRD) 2021 Classification
- Group
1: Diseases curable with one-time interventions,
such as Lysosomal Storage Disorders treatable via Hematopoietic Stem Cell
Transplantation.
- Group
2: Conditions requiring long-term or lifelong
management but are relatively less costly to treat, like Phenylketonuria
and Maple Syrup Urine Disease.
- Group
3: Diseases requiring expensive, lifelong
treatments, such as Gaucher Disease and Pompe Disease.
Criteria for Orphan Drug Designation
- Low
Prevalence: The disease must affect a small
portion of the population.
- Unmet
Medical Need: There must be no approved
treatments, or the orphan drug should provide significant advantages over
existing options.
- Evidence
of Benefit: Scientific evidence demonstrating
the drug’s potential to treat the disease, whether in preclinical or
clinical trial stages.
Incentives for Orphan Drug Development
- Market
Exclusivity: Grants a period where competitors
are barred from launching similar products, incentivizing innovation.
- Tax
Credits: Offered on R&D costs to lessen
financial risks for developers.
- Fee
Waivers: Regulatory application fees are
waived, reducing entry barriers for new orphan drugs.