AstraZeneca India made its entry into the treatment of rare diseases in India on Tuesday. The company’s ‘Selumetinib’ for Indian patients has been approved by regulators. Regarding rare diseases, there is neither a precise estimation of the rate of mortality or morbidity in India nor an understanding of the disease burden on the public healthcare system.
However, it is estimated that rare diseases affect more than 400 million people worldwide, and approximately 7000 rare diseases have been recorded and identified at the present time. It implies that additional access and research into rare diseases are required to better prepare us to develop the appropriate medicines for patients who have been diagnosed with or live with known rare diseases and to combat the burden.
According to Prasanna Shirol, Executive Director and Co-Founder of Organization for Rare Diseases India (ORDI), patients with rare diseases require prompt diagnosis, prompt treatment, and long-term supportive care. As a result, government funding is just as important as private or philanthropic funding. It is essential to raise awareness among all stakeholders, including the general public, medical professionals, basic and clinical researchers, and policymakers.
In addition, Prasanna Shirol said that global pharmaceutical companies have high expectations due to the extremely limited availability of any rare disease treatment in India. Patients with rare diseases, particularly those with neurological conditions, benefit greatly from the introduction of new products by companies like AstraZeneca, and they can also hope to live longer than the general population.
AstraZeneca also announced in a press release that on Rare Disease Day, it will collaborate with the Organization of Rare Diseases in India (ORDI), a non-profit organization dedicated to enhancing the quality of life for people with rare diseases.