Understanding Rare Diseases: A Global and Indian Perspective
Deepak Maramwar
Associate Director - Regulatory Affairs @ AstraZeneca | Certified Independent Director
Rare diseases (Orphan diseases) are those affecting a small fraction of the population. Disease in the United States is considered uncommon when it affects less than 200,000 individuals. Based on the Ministry of Health and Family Welfare, in India, the definition of rare diseases encompasses any condition prevailing in less than 1 in 2,500 people. Despite the fact that rare diseases individually comprise only a few cases, if aggregated, they constitute millions of cases worldwide, making them a concern with regard to diagnosis, therapy, and management.
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There are approximately 7000 rare diseases found in the world, which impact about 400 million people. Most of these disorders are genetic and manifest in infancy. Most of these conditions are chronic and even life-threatening. Medical research has moved a long way, and now even rare diseases are being treated. Unfortunately, until now, more than 95% of the rare diseases have no treatments yet. One of the major challenges in advancing novel therapies into the market is its expensive drug development, limited patient group, and complex regulatory requirements.
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In India, the prevalence of rare diseases is estimated to be 70 million people. Indian scenario is associated with certain challenges including inadequate awareness, delayed diagnosis, and sparse specialized healthcare setups. Treatment cost is also extremely high, and insurance coverage is rarely guaranteed, leading to further burden on the patients and their family. In addition, no nationwide registry exists to facilitate data acquisition and research in an efficient manner.
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The Indian government has also noticed these problems and is trying to address them. For example, the National Policy for Rare Diseases (NPRD) was introduced into the country with the aim of ameliorating the diagnosis and treatment conditions. Recently, the Drug Controller General of India (DCGI) has asked state authorities to speed up the approval process for rare disease drugs with the goal to bring down the approval period to 90 days. It will encompass the arrangement of import of rare drugs and the priority testing of such drugs in government laboratories.
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The direction of rare disease management in India appears to be promising, with increased research work, likely policy amendments, and better collaboration among stakeholders. Advancements in gene therapy and personalized medicine offer hope for more effective treatments. The government's commitment to streamlining regulation and making medicines easier to access will play a key role in assisting patients to achieve an optimized treatment.
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Regulatory professionals play a crucial role in this landscape. Their expertise ensures that therapies for rare diseases meet safety and efficacy standards while navigating complex regulatory pathways. Through accelerating approvals and compliance, they serve as a means of getting much-needed treatment to patients quickly.
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Addressing the challenges that rare diseases present can only be achieved by the interaction between the elements within government agencies, healthcare, researchers, regulatory, and patient advocacy bodies. By working together, we can raise awareness, increase access to therapy, and as a result enhance the quality of life for people suffering from rare diseases in India and globally.
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Good read ??
Global DMS Team Lead (Quality)@ Sanofi #Ex-Endo Par formulation# Ex Indocoids
2 个月Insightful