Rare Diseases crowd-funding

Rare Diseases crowd-funding

Subject: Science

Context: After being pulled up by the Delhi High Court, the Centre on Wednesday said that it has operationalised a digital platform for crowd-funding for expensive treatment for rare diseases among children.

Concept:

• The portal, http://rarediseases.aardeesoft.com has operationalised by the center government a digital platform for crowd-funding for expensive treatment for rare diseases among children.

The Rare Diseases Policy

• The Rare Diseases Policy aims to lower the high cost of treatment for rare diseases with increased focus on indigenous research with the help of a National Consortium to be set up with Department of Health Research, Ministry of Health & Family Welfare as convenor.
• Increased focus of research and development and local production of medicines will lower the cost of treatment for rare diseases.
• The policy also envisages creation of a national hospital based registry of rare diseases so that adequate data is available for definition of rare diseases and for research and development related to rare diseases within the country.
• The Policy also focuses on early screening and prevention through primary and secondary health care infrastructure such as Health and Wellness Centres and District Early Intervention Centres (DEICs) and through counselling for the high-risk parents.
• Screening will also be supported by NidanKendras set up by Department of Biotechnology.
• Policy also aims to strengthen tertiary health care facilities for prevention and treatment of rare diseases through designating 8 health facilities as Centre of Excellence and these CoEs will also be provided one-time financial support of up to Rs 5 crores for upgradation of diagnostics facilities.
• A provision for financial support up to Rs. 20 lakhs under the Umbrella Scheme of RastriyaArogyaNidhi is proposed for treatment, of those rare diseases that require a one-time treatment (diseases listed under Group 1 in the rare disease policy).
• Example of Rare Diseases: Lysosomal Storage Disorders (LSD), Pompe disease, cystic fibrosis, muscular dystrophy, spina bifida, haemophilia etc.
• India has close to 50-100 million people affected by rare diseases or disorders, the policy report said almost 80% of these rare condition patients are children and a leading cause for most of them not reaching adulthood is due to the high morbidity and mortality rates of these life-threatening diseases.