Recently Rare Disease Day was observed by the Indian Society for Clinical Research (ISCR). The event held at Bengaluru aimed to raise awareness by educating people through media. Among those attending were Prasanna Shirol, Founder Member, Organisation for Rare Diseas India (ORDI), Vikas Sharma, Member, Ethics Council, Indian Council for Clinical Research, Premroop Alva, from Haemophilia Society, Bangalore and author Ramgopal Vallath. “There are over 70 million patients in India who suffer from more than 7000 rare diseases. Yet there is no supportive therapy,” said Shirol.
“In India we consider a disease rare if it affects 100 people per 100,000 population, but given the large population the absolute numbers are significant for rare diseases to receive quick and urgent attention,” said Sharma.
The rare diseases causes a range of physical, cognitive and developmental disabilities and occurs in about one in 10,000 live births. The disorder can result in a number of medical issues, including less resrictive to completely dilapidated conditions.
Sharing his experience vallath who suffers from Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) said, “A majority of patients in India cannot afford the current line of treatment nor is there an option to access clinical trials for these diaeases in India.” He was able to participate in a stem cell trial for CIDP in US after which his condition improved significantly. Alva who suffers from hemophilia said, “There is no cure as yet for hemophilia and it can only be managed by medication. Lack of awareness and the high cost of treatment have made life difficult for hemophilia patients.”
The lack of treatments make clinical trials important for rare diseases. “Patients suffering from rare diseases have an equal right to benefit from new medicines and treatments or choose to be part of clinical trials,” said Shirol.
Added to this lack of policy is also a challenge faced by rare disease patients. The policy will strengthen diagnostics, provide access to the available treatment option and look at newer therapies to benefit patients suffering from rare diseases. A draft of Rare Diseases and Orphan Drugs Development policy is in the making. Once finalised and approved the policy will help create awareness among the medical professionals to enable early detection. Moreover, it would also create a registry of rare diseases for researchers enhance cilinical trial.
Drug development for rare diseases are called orphan as neither the drug manufacturers nor the government are actively persuing them. Sharma said that there is need to create supportive environment in India for clinical research in rare diseases. The number of clinical trials taking place in India has reduced drastically in the last few years and the number of clinical trials in rare diseases is even more insignificant, he added.