The Indian Society of Clinical Research (ISCR) and the Organisation for Rare Diseases India (ORDI) are alarmed about the lack of treatment option for 70 million rare disease patients in the country and are keen to intensify clinical research. This would not just drive drug development but provide access to expensive drugs that under research to patients of rare diseases.

On the occasion of the Rare Disease Day observed annually on February 28, ISCR and ORDI teamed up to highlight on this year theme Living with a Rare Disease. The two are concerned over the millions of caregivers, friends and relatives of patients whose daily lives are impacted and are living day-by-day, hand-in-hand with rare disease patients.

According to the EveryLife Foundation for Rare Diseases, 95 per cent of rare diseases lack a single FDA approved treatment.

“We need to create a strong ecosystem for rare diseases in India, including a patient registry and a supportive national policy and regulatory environment. Patients suffering from rare diseases have an equal right to benefit from new medicines and treatment available to patients in other parts of the world.  There are over 70 million patients in India who suffer from an estimated 7000 plus rare diseases. Yet there is no treatment for more than 90 per cent of these diseases, patients of whom have to depend only on supportive therapy,” said Prasanna Shirol, Founder Member, Organisation for Rare Diseases India at an event organized by ISCR .

“Awareness, accessibility and affordability are key needs of patients suffering from rare disease and early diagnosis is a critical challenge in the management of rare diseases,” he added.

“We consider a disease rare in India if it affects 5000 of our population or less and given the large population we have. The absolute numbers are significant for rare diseases to receive quick and urgent attention. Given the huge burden of rare disease we have in India, there is an urgent need to invest in clinical research to ensure that patients have access to new drugs and treatment that research makes possible,” said Dr Vikas Sharma, Member, Ethics Council, and ISCR.

Patients who were also present at the event suffering with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), a debilitating auto immune disorder, said that life with this condition was a one of constant struggle and suffering. Therefore the aim is to create greater awareness about the disease and its treatment in India.  

“Patient support groups have become an important source of help and support for patients suffering from rare diseases,” according to the Bangalore Hemophilia Society adding that no cure, low awareness and the high cost of treatment have made life difficult for hemophilia patients.

India has 17 per cent of the world’s population, 20 per cent of the highest disease and yet less than 1.5 per cent of global trials take place in India,” said Suneela Thatte, president, ISCR.