New Delhi, May 18 (IANS) The Delhi High Court has taken significant steps towards addressing the treatment of rare diseases by forming a five-member committee responsible for implementing the National Rare Disease Policy-2021.
The committee’s mandate includes procuring and “indigenising” therapies and drugs for patients, primarily children, who suffer from these rare diseases.
The National Rare Diseases Committee will comprise members such as the Secretary of the Union Health Ministry or their nominee, the Director General of the Indian Council for Medical Research (ICMR), the Drug Controller General of India, and Dr Madhulika Kabra and Dr Nikhil Tandon from AIIMS, Delhi, as ordered by the court.
The court’s decision came in response to a batch of petitions highlighting the challenges faced by children with rare diseases.
Justice Prathiba M. Singh emphasised the need for urgent steps and close coordination between the medical community, therapy providers, and government agencies.
“Currently, the lack of centralised coordination among Centres for Excellence results in delayed availability and inadequate therapies for rare disease patients,” said Justice Singh.
The court acknowledged that despite the government’s willingness to find solutions for rare diseases, the lack of data, awareness, and availability of treatment pose significant obstacles. Additionally, exorbitant prices of drugs and therapies further hinder easy and timely access to medicines.
To ensure effective implementation of the 2021 policy and extend its benefits to patients, the court deemed it appropriate to establish the National Rare Diseases Committee.
“The committee’s responsibilities will encompass all necessary steps for policy implementation,” said Justice Singh while listing the matter for next hearing on May 29.
The petitioners, who are children suffering from various rare diseases such as Duchenne Muscular Dystrophy (DMD) and Mucopolysaccharidosis-II or MPS-II (Hunter Syndrome), sought uninterrupted and free-of-cost treatment due to the high expenses associated with their therapies.
The court highlighted that AIIMS currently has 517 enrolled DMD patients, out of which 312 can receive treatment. Moreover, 189 patients are registered for spinal muscular atrophy and 166 for lysosomal storage disorders, gaucher, MPS, and other rare diseases.
The newly-formed committee will address the immediate needs of petitioners whose treatment has been halted due to funding issues. It will also engage with therapy providers or manufacturers to ensure the prompt commencement of adequate treatment for these patients.
The committee’s responsibilities extend beyond immediate actions. It will periodically review the National Rare Disease Policy and recommend any necessary changes. Additionally, it has the authority to consult relevant individuals or organisations to fulfill the policy’s overall objectives.
The court directed a meeting between Hanugen Therapeutics Private Ltd, the Biotechnology Industry Research Assistance Council, and representatives of the ICMR to establish a timetable for DMD clinical trials based on approved protocols.
In previous orders, the court had instructed AIIMS to initiate treatment for eligible children with rare diseases and requested the government to provide funds for their treatment. It had also issued various directions related to rare disease treatment, including the notification of the National Health Policy for Rare Diseases and the establishment of a national consortium for research, development, and therapeutics.
The court stressed the urgency of addressing the medical condition of 40 children suffering from rare diseases and expects the AIIMS Rare Diseases Committee to act promptly in these matters.
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