The fate of Rare Disease patients like many others has been hard hit due to the ongoing Covid-19 pandemic. Already a community that was struggling for access to drugs and sustainable funding for treatment from the government, the patients; mostly children below the age of 10; are now left without any ray of hope.
With the delay in the finalisation of the National Policy on Rare Diseases, the only silver lining has been the judicial interventions from various state governments such as Kerala, Karnataka and Tamil Nadu, where court interventions have helped Rare Disease patients find access to sustainable treatment. However, the patients’ problems still remain at large who endure a long journey of multiple visits to doctors, numerous tests and yet frequent misdiagnoses before even being able to access drugs which requires sustainable lifelong funding.
Speaking on the issue, Mr. Manjit Singh, President, Lysosomal Storage Disorder Support Society (LSDSS), said, “Approximately 190 patients diagnosed with LSDs categorized under Group 3 disorders are not only awaiting financial support for their treatment, but have no hope to mobilize funds on their own to support the expensive treatment costs, thereby compromising their survival prospects.”
“All these patients have been medically evaluated by the respective state technical committees, and were eligible for funding for their respective treatments,” he added.
Since the lockdown, uncertainty has set in again. Some patients have run out of medicines and are forced to get stop-gap treatments from doctors. In other cases, where hospital visits are common, fear of the virus coupled with failure to get treatment has become a double-edged sword without adequate support from the government. Moreover pharmaceutical supply chains have been strained by border closures and quarantine measures.
The treatable Rare Diseases drugs in India fall under the ‘Orphan Drug’ category i.e. drugs meant to treat a condition that affects not more than five lakh people in India. Rare Diseases affect people from all statuses of life most of whom without support are not able to access and afford the treatment cost which is a lifetime affair post diagnosis. The draft National Policy on Rare Diseases (2020) with sustainable funding for treatable Rare Diseases can address such concerns. However the draft has been pending since it was introduced in the parliament in February 2020; furthermore due to the ongoing pandemic.
Commenting on the issue, Dr. Ashok Gupta, Professor of Pediatrics SMS Medical College Jaipur, in charge of Rare Disease Center JK Lon Hospital and Chairperson Technical Committee on Rare Disease Government of Rajasthan, said “We need to have an appropriate mechanism for Rare Disease patients, who are mostly children. Collectively, the number of Rare Disease patients is fairly large. A few of the Rare Disease patients have disabilities that require support through a consistent mechanism. The required support can be given by putting in place a National Policy on Rare Diseases, which I believe will solve this issue at hand.”
