India Raring to Make its Mark in the Rare Disease Market

With one-third of all new approved drugs over the past five years in the US addressing rare diseases, the last decade is said to have been the most productive period in the history of orphan drug development. Rare diseases are often referred to as orphan diseases, and a drug that has been developed specifically to treat a rare medical condition is designated as an orphan drug.

For a long time now, pharmaceutical companies have been staying away from developing orphan drugs because of spiralling costs of development and stringent regulations, and that orphan drugs offer limited market size. Rare Disease India (RDI), a volunteer-driven online entity, describes a rare disease as one that has fewer than 100 patients per 1,00,000 population. Today, approximately 7,000 rare diseases have been estimated across the globe. One in every 10 Americans is affected with a rare disease. In India, 72 lakh people are estimated to be affected by rare diseases, as per the RDI data.

The US in 1983 became the first country to propose a legal framework to encourage the development of orphan drugs. The US, the EU, and Japan are said to have the most well-defined frameworks for the development of orphan drugs. While the assignment of orphan status to a disease or drug is a matter of public policy of any country, overall the framework allows for incentivising pharmaceutical companies. This has resulted in medical breakthroughs that may not have otherwise been achieved. In the US, when a drug is given the orphan status, it enjoys market exclusivity for seven years, and in the EU and Japan it is 10 years.

“There are basically two strategies to encourage orphan drug development. The push strategy and the pull strategy. Under the push strategy, pharma companies are given grants to design and conduct trials, and the pull strategy involves tax credits and other financial incentives,” said Christopher Milne, Director of Research and Research Associate Professor at the Tufts Center for the Study of Drug Development.

Moreover, “orphan drugs typically take fewer resources to develop”, he notes. He adds that for orphan drugs, the sample size required for trials is generally a third of that of non-orphan drugs.

“For orphan drugs, the sample size would probably be a 100, whereas for non-orphan drugs, it will be around 500,” he says. Another thing is that for rare diseases, marketing expenses are less. It is said that half of the orphan drugs that were approved over the last 10 years have been from Big Pharma, and a quarter of the biologics approves have been orphan drugs. Despite the limited market, Big Pharma and biologics are becoming very active in the space. Biologics account for a major share of the orphan drugs market (over 50 per cent).

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