What are Orphan drugs and why they are so expensive drugs:-

Orphan drugs are medications designed to treat rare diseases or conditions, affecting a small percentage of the population (typically fewer than 200,000 people in the United States). These drugs are often expensive due to:

1. *Low demand*: Fewer patients mean lower sales, making it challenging for manufacturers to recoup development costs.

2. *High development costs*: Researching and developing orphan drugs can be costly due to:

- Limited patient populations, making clinical trials more difficult.

- Complexity of rare diseases, requiring specialized expertise.

- Higher costs for manufacturing and quality control.

3. *Regulatory incentives*: Governments offer incentives, such as tax credits, grants, and market exclusivity, to encourage orphan drug development. These incentives can increase costs.

4. *Lack of competition*: Orphan drugs often have limited competition, allowing manufacturers to set higher prices.

5. *High production costs*: Small-scale production, specialized equipment, and rigorous quality control measures drive up costs.

6. *Marketing and distribution*: Targeted marketing and distribution strategies for rare diseases can be more expensive.

7. *Profit margins*: Companies may set higher prices to ensure profitability, considering the significant investment in development and limited sales volume.

8. *Payer and reimbursement dynamics*: Insurers and governments may cover high costs due to the rarity and severity of the conditions, contributing to higher prices.

Examples of expensive orphan drugs include:

1. *Luxturna* (voretigene neparvovec): A gene therapy for inherited blindness, priced at $850,000 per eye.

2. *Zolgensma* (onasemnogene abeparvovec): A gene therapy for spinal muscular atrophy, priced at $2.1 million per treatment.

3. *Ravicti* (glycerol phenylbutyrate): A treatment for urea cycle disorders, priced at $250,000 per year.

Keep in mind that prices may vary depending on the country, insurance coverage, and other factors.