"Rare-Disease Drugs Emerge as India's Pharma Opportunity"
Orphan drug
a medicine developed for a rare disease, historically unprofitable due to small patient numbers
Rare disease
a condition affecting a small share of the population, often genetic
Rare diseases affect only a small number of people each. But together there are about 8,000 such diseases worldwide. They affect hundreds of millions of patients. India has reported 1,004 rare genetic disorders so far. These conditions are also called 'orphan diseases'. The medicines made for them are called orphan drugs. Drug companies traditionally avoided orphan drugs. Treating very few patients did not bring enough profit. To fix this, the United States passed the Orphan Drug Act, 1983. It gave tax credits, grants, and fee waivers to companies. It also gave seven years of market exclusivity to the first approved treatment for a disease. Market exclusivity means the sole right to sell the approved drug. This made the field profitable. Before the Act, only 38 such drugs had been approved in the US. By 2022, this number had grown to 1,122. Japan, Australia and the European Union later passed similar laws.
In India, the National Policy for Rare Diseases gives financial help. It provides up to Rs 50 lakh per patient for treatment. This is often not enough. Many rare-disease drugs cost about $100,000 (roughly Rs 1 crore) a year. Families often turn to crowdfunding to cover the gap. Only about 5% of rare diseases currently have an approved drug. This funding gap and treatment gap show why India needs a clearer strategy for this sector.
Developing an orphan drug has two main hurdles. First, companies need enough patients with the same rare disease. They must run a clinical trial and prove the drug works. Second, once approved, the drug must be manufactured and distributed affordably. India has real strengths for the first hurdle. The population is above 1.4 billion. Even a rare condition can have a sizeable patient group in absolute numbers. High endogamy raises the chance of certain genetic disorders. Endogamy means marriage within the same community. For example, beta-thalassemia affects about 3.7% of India's general population. But it affects up to 10.5% in some specific ethnic groups. …
Consider the following statements regarding the U.S. Orphan Drug Act, 1983:
1.It provided tax incentives and regulatory fee waivers to companies developing drugs for rare diseases.
2.It granted seven years of market exclusivity to the first approved drug for a given rare disease.
3.It resulted in a decline in the number of rare-disease drug approvals in the United States.
Which of the statements given above is/are correct?
Which of the following factors make India an attractive location for conducting clinical trials for rare-disease drugs?
1.A large overall population providing bigger absolute patient numbers for any given rare condition.
2.High rates of endogamy in some communities raising the prevalence of certain genetic disorders.
3.The presence of organised patient advocacy groups and disease registries.
Select the correct answer using the code given below:
With reference to India's National Policy for Rare Diseases, which of the following statements is correct?
Consider the following pairs of terms related to rare-disease drug policy:
Term — Meaning
1.Orphan drug — A medicine developed for a disease affecting very few patients
2.Market exclusivity — A fixed period of sole right to sell an approved drug
3.Benefit-sharing — A principle that contributors to drug development receive a fair share of resulting benefits
4.Endogamy — A practice of marrying outside one's own community
How many of the above pairs are correctly matched?
The concept of 'benefit-sharing' discussed in the context of rare-disease drug development is most closely associated with which of the following?
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