Rare Diseases- The Need to Address this Common Problem, Health News, ET HealthWorld
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Rare Diseases- The Need to Address this Common Problem
Worldwide, 7000 rare diseases affect 350 million people with the majority of them being children. Moreover, according to the Indian Society for Clinical Research, there are 70 million patients in India suffering from such disorders, pointing to the urgency to address the issue. Taking cognizance of the need to secure treatment for patients of rare diseases, the government launched the National Policy for Rare Diseases in March 2021.
March 30, 2023, 07:09 IST
by V. Simpson Emmanuel
In 2018, the revelation by the late actor Irrfan Khan that he is suffering from a rare disease stirred the country upside down. Till then, rare diseases have rarely been the topic of mainstream healthcare discussions, but Khans acknowledgement of the disease prompted people to sit up and take notice. Contrary to the general perception, rare diseases are not uncommon. Worldwide, 7000 rare diseases affect 350 million people with the majority of them being children. Moreover, according to the Indian Society for Clinical Research, there are 70 million patients in India suffering from such disorders, pointing to the urgency to address the issue.
Characteristically disabling, rare diseases are chronic, severe, progressive, and debilitating. They radically impact the quality of lives of patients wherein they suffer from a lack of self-sufficiency and are unable to do even simple daily tasks. Most of these disorders are genetic and without any exact cause. Moreover, relatively similar symptoms make the initial misdiagnosis common leading to subsequent delays in treatment, which in turn can be life-threatening the dearth of diagnostic facilities, unfamiliarity on part of the physicians concerning symptoms, and lack of awareness about genetic screening are some of the other issues related to rare diseases.
It would be worthwhile to note that treatments are available for some of the rare diseases which have proven to be highly effective in enabling a better quality of life for patients. Therefore, patients must get access to the right treatment at right time. Concerted efforts are required to create awareness around this. Taking cognizance of the need to secure treatment for patients of rare diseases, the government launched the National Policy for Rare Diseases in March 2021. Even as the policy acknowledges funding gaps and entails provisions for financial support of up to Rs. 50 lakhs, competing health priorities today make it difficult for the government to fully finance such treatments.
The Central government has also attempted to boost accessibility by exempting basic customs duty on rare disease drugs that are imported for personal use or by institutions. However, it is critical to understand that the challenges are manifold. For instance, importing a drug at the patient level would mean higher intermediary margins, complexities of clearance formalities, and supply chain risks related to storage and transportation among others. Extending tax benefits to rare disease drugs that are approved for sale in India will not only help bring down the cost of treatment but also significantly enhance accessibility for Indian patients. It is critical to understand that the challenges are manifold, and a systematic approach would be required to address them. This might include taking measures that can connect across fragments and facilitate the right sort of intervention for the right patient.
Since rare disease patients struggle through their everyday lives, we as experts need to develop a robust support system that can help them deal with individual problems. Furthermore, it is imperative to include rare diseases in the medical curriculum so that healthcare professionals can identify the symptoms and timely action can be taken. Additionally, genetic testing in patients needs to be a requisite, specifically in high-risk cases where there has been a family history of any rare disorder.
While these are some of the steps which would be effective in overcoming some of the issues, government intervention would act as the foundation to tackle this health challenge. We, as a country, have started taking steps in this direction and are ready to address the challenge of rare diseases. The National Rare Disease Policy is quite comprehensive and has been revised to cater to the specific aspects related to the diseases. Once implemented efficiently, it will lead to making a huge difference in the lives of patients. Efforts are underway to overcome the problem of rare diseases in the country. However, a lot of groundwork will be required if the efforts are to be successful. A behavioural change, most importantly, on part of the medical fraternity and patients, would provide support to the governments initiatives. A patient-centric approach would have to be at the bottom of all initiatives since it would provide a better understanding of the possibilities and limitations of the challenges at hand.
Rare diseases are poised to become one of the biggest health concerns in the country and a steady upswing in mortality is only worsening the situation. A coordinated strategy involving healthcare professionals, patients, and the government will be necessary to tackle the problem from different angles. Such a system would play an essential role in improving the quality of care and outcomes and address the unmet clinical needs of patients suffering from rare diseases.
V. Simpson Emmanuel, Managing Director & CEO, Roche Products (India) Pvt. Ltd. & Serina Fischer, General Manager, Takeda Pharmaceuticals India Pvt Ltd.
(DISCLAIMER: The views expressed are solely of the author and ETHealthworld does not necessarily subscribe to it. ETHealthworld.com shall not be responsible for any damage caused to any person / organisation directly or indirectly.)
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Thursday, March 30, 2023 at 1:39 am