India Key Market For APIs, But Patients Forced To Cough Up Crores To Buy Orphan Drugs

HYDERABAD: Though India manufactures most of the key active pharmaceutical ingredients (APIs) for over 400 FDA-approved orphan drugs for rare diseases, patients back home are forced to cough up crores to procure them.

Primary reason being reluctance among Indian pharma companies to develop the drugs at home owing to policy hurdles. Meant to treat rare medical conditions, orphan drugs would not be profitable to produce without govt assistance due to the small population of patients affected by the conditions.

The APIs are hence exported to foreign companies which are sold back to India as tablets, capsules, injections etc., at exorbitant prices.

For instance, an annual dose of Trientine, imported for treatment of Wilson’s disease (genetic disorder that causes build-up of copper) costs Rs 1.6 crore, making it out of reach of patients.Drugs for rare diseases burn pockets, patients pay price for policy hurdles Patients suffering from rare disease in the city are being forced to cough up huge amounts for key drugs. For instance, the Eliglustat drug administered to patients with Gaucher’s disease (metabolic disorder causing fat build-up in organs) comes for as much as 1.8 crore. These prices make it impossible for patients to avail of treatment, say industry sources.

According to the Lysosomal Storage Disorders Support Society (support group) at least 20 patients across India, suffering from rare diseases such as Pompe, Gaucher’s, Wilson’s etc., have died in the last two years due to lack of medicines. The conditions that orphan drugs are used to treat are referred to as orphan diseases.

High import costs

“Exported as powder, when the APIs are shipped back to India as a tablet, we need an import licence. For that, we have to pay 10% of the cost of the drug along with the cost of the package. Also, since some drugs are unstable above room temperature they have to be sent in a cold box. This further hikes the cost,” said Ramaiah Muthyala, president, Indian Organisation for Rare Diseases. According to him, only eight of these 400 drugs are actually expensive while the price of the rest is just inflated.”Encouraging Indian companies to manufacture the drugs is the solution to tackle these expensive price tags,” he added. In fact, many industry experts said that these drugs could easily be made available for a fraction of their current cost – for as little as 20,000 to 3 lakh – if generic drug manufacturing companies in India develop them.

But that isn’t an easy affair, claim local firms that have started foraying into this business. For starters, there is no database of patients to ascertain the requirement of drugs. Also, India lacks a proper channel for marketing such drugs, say industry players.

Regulatory hurdles

“Even though we have a ready product to treat Dravet Syndrome, which has brought down the cost to one tenth the price of the US product, it’s a challenge to find patients,” said Dr Jagadeesh Rangisetty, CEO, Biophore Pharmaceuticals. The company is one of the three Indian firms that recently took up manufacturing of orphan drugs. Only about 12 drugs are being manufactured currently, while for all other diseases patients are dependent on foreign sellers. “It was a challenge to even start developing the drug,” Rangisetty said. “Since our product, Cannabidiol, has ingredients found in marijuana, it took us five years to educate government agencies about it and assure them that the constituent is not considered narcotic around the world. It took another one-and-half years to get the clearances from the DCGI office,” he added.

The official also stressed that lack of incentive, R&D support and a channel to buy and sell products are factors discouraging firms from manufacturing drugs. The US govt allows seven years exclusive rights to the company developing a drug for rare diseases to sell these.

Cumbersome process

“In the absence of a patients database, we usually get in touch with the handful of distributors who procure the drugs approved for marketing in India. But this takes at least a few weeks,” said Dr Ashwin Dalal, nodal officer rare diseases of Centre of Excellence (CoE) in Hyderabad. It is one among 12 such CoEs across India. The process is even more cumbersome when hospitals have to import a drug that is not approved for marketing in India but can be procured for a certain patient in a particular case, said industry sources. “This delay can be fatal for patients,” said Muthyala.