KOCHI: Many patients dealing with rare diseases that need frequent medical attention were affected due to the Covid-19 pandemic. The community that was already struggling to get access to drugs and sustainable funding for treatment is finding it hard to hold on — especially children under the age of 10. The delay in finalisation of the National Policy on Rare Diseases is one of the main reasons for this.
Currently, the only silver lining for these patients is the judicial interventions from various state governments including Kerala, Karnataka, and Tamil Nadu. These have helped many struggling patients to find sustainable treatment. “Genetic rare diseases are serious, chronic and fatal disorders, often requiring long term, specialised treatments/ management and sometimes causing severe disabilities,” said Dr Mohandas Nair, pediatrician, Kozikhode Medical College.
However, the elaborate procedure, including visits to doctors, numerous tests, and access to drugs require lifelong funding. “Approximately 190 patients diagnosed with Lysosomal Storage Diseases (LSDs), categorised under Group 3 disorders, are awaiting financial support while having little hope of being able to mobilize funds,” said Manjit Singh, president, Lysosomal Storage Disorder Support Society (LSDSS).
Many patients and their families are worried about losing the state funding. “All these patients have been medically evaluated by the respective state technical committees, and were found eligible for funding. Running out of medicines during the lockdown forced them to take a break from doctors and treatment. If the state governments withdraw their benefits, it would be impossible for us to find funding,” said the father of a hemophilia patient.
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. The draft National Policy on Rare Diseases (2020) can address the concerns related to such diseases and their treatment. However, the draft has been pending since it was introduced in the parliament in February 2020 due to the pandemic.Dr Mohammed Asheel, executive director, Kerala Social Security Mission, said a solid premise for sustainable treatment funding is needed in the National Policy for Rare Diseases and attention should be given to availability of medicines.