Former President of India A.P.J. Abdul Kalam on Friday said that a coordinated effort at the national level is the need of the hour for more research and understanding rare diseases in the country. The former president also felt that there was a need to share knowledge among patients, doctors and researchers to effectively tackle rare diseases.
The former president, who was participating in the inaugural of a conference on ‘Raising Awareness on Rare Diseases’ here on Friday, acknowledged that due to number of cases being low, Governments do not consider rare diseases as a public health issue.
“There is a need for a whole ecosystem consisting of doctors, a registry to record the prevalence of rare diseases, bio banks, support groups, more research on drug discovery and of course a regulatory framework. Each component is complex and there is a lot of work ahead,” Mr. Kalam said.
A majority of ailments like ALS (Amyotrophic Lateral Sclerosis), Muscular Dystrophy, congenital heart diseases, thalassemia, sickle cell anaemia and many more come under the category of rare diseases. Experts in the conference pointed out that there was also need to promote development of drugs for rare diseases, which are quite often known as orphan drugs.
Members of Indian Organisation for Rare Diseases (IORD), who organised the conference, said that at present, India is yet to adopt a definition for rare diseases. “We do not have a public policy on rare diseases and there are no trained doctors or diagnostic facilities to identify the ailments. There are no incentives for pharma sector to develop drugs for rare diseases and it’s tough to find information on rare diseases for families and even professionals,” said president, IORD, Dr. Ramaiah Muthyala.
Experts in the conference said that worldwide, rare diseases affect one in 10 people. “There are close to 7,000 rare diseases today and many rare caused by genetic factors. However, we have drugs to manage only 400 rare diseases. There is also no awareness and persons with such ailments are treated as social stigma. A lot of work has to be done in this field,” Dr. Muthyala felt.
