Policies and Guidelines
Policies relating to rare diseases are significant in its ability to transform patient lives. By formulating and implementing pro-rare disease policies; patients will be able to access quality treatment and care, better access to drugs and clinical trials, multidisciplinary care and psychosocial support.
India is still in the nascent stage of a clearcut National Rare Disease Policy guidelines. Since health is a state subject, each state has come up with a set of policies to improve patient care and access to treatment.
It is also significant that rare diseases like the blood and bleeding disorders, muscular dystrophies and multiple sclerosis have been included in the Rights of People with Disabilities Act 2016. This will not only improve and strengthen healthcare practices but also provide legal support to children and adults in accessing education and other services in the country.
Patient support groups are important for advocacy, activism and even pushing key research forward. In Europe and the United States, patient support groups have played a key role in bringing in new drugs and treatment. For ex: The Alkaptonuria Society (AKU Society) in the UK played a major role in researching drugs for repurposing towards AKU treatment.
Patient support groups also bring together patients across the country and provide a sense of unity and dispel the feeling of being alone in the fight against rare diseases. Patient support groups have been active in India in advocating for rare diseases and even shaping policy outcomes.