Integrating rare disease management in public health programs in India: exploring the potential of National Health Mission

Abstract

Rare diseases (RD) are conditions that affect a small number of people and hence do not get the focus on government health priorities in a resource-constrained setting such as India. Therefore, it is essential to focus on strengthening and utilizing the existing public health framework for the optimal usage of healthcare resources. In this regard, National Health Mission (NHM) is one of the crucial programs initiated by the government of India to address the health needs of the under-served. As Phase 1 of the NHM moves towards completion, we explored the Reproductive, Maternal, Newborn, Child, and Adolescent Health (RMNCH + A) program under NHM to assess their potential and limitations to aid RD care. We found that some of the disease-prevention initiatives of NHM address certain RDs and can easily be expanded to manage many such preventable RDs. In addition, NHM programs can provide a unique epidemiological data repository to strengthen the National Rare Disease Registry. These programs can also play important role in providing a continuum of care for many RDs that need lifelong management. However, existing programs have a limited scope to provide specialized RD-related treatments, which is better served in a more focused system. Thus, considering RDs in the design of the existing programs may help RD management better through prevention, data collection, and providing a continuum of care.

Balancing patient need with public policy in rare diseases – A legal perspective

“…A classic example would be the cost of voretigene neparvovec, sold under the brand name Luxturna, a gene therapy medication for the treatment of Leber congenital amaurosis (LCA) developed by Spark Therapeutics. [ 2 ] The cost of the one-time therapy is USD 850,000, which is over INR 5 crores, which puts it out of reach of most patients. Finally, the lack of epidemiological data in India on rare diseases makes it very difficult to assess the economic burden, morbidity, and mortality of these diseases, further widening the gap between demand and supply.…”