ORPHAN DRUGS – SCIENCE & TECHNOLOGY

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.

Source: https://www.thehindu.com/sci-tech/health/understanding-orphan-drugs-what-are-they-how-are-they-classified-and-what-are-their-challenges-in-the-indian-context/article68806270.ece