With the 3rd consecutive term win of the government, rare disease patients and their caregivers wrote to the newly appointed Union Minister at MoH&FW- JP Nadda showing gratitude for the significant strides made during the previous term of the government, particularly the introduction of the National Policy for Rare Diseases 2021.
These patients and their caregivers appealed to draw ministry’s attention to the critical gaps that remain in the implementation of this policy. The lives of many patients (mostly children) suffering from chronic rare genetic disorders, such as Lysosomal Storage Disorders (LSDs), are still fraught with uncertainty due to the lack of sustainable funding and delays in the effective utilization of the allocated funds. The Centres of Excellence (CoEs), pivotal in this initiative, need to expedite the process to ensure timely access to life-saving treatments.
Below are the recommendations that these patients, caregivers, and advocacy groups given to the ministry:
I. Build continuum of care with sustainable funding support for Group 3a conditions
There is an immediate need to prioritise, ensure uninterrupted funding support for all notified ultra-rare diseases – conditions which are chronic in nature, but have approved therapies with excellent clinical outcomes
• The current budgetary provision up to INR 50 lakh is only one-time, thereby hindering the process of saving lives for patients diagnosed with these conditions. Several patients across the Centres of Excellence (CoEs) have once again been put off life-saving therapies after the one-time support exhausted
II. Provide equal weightage to all notified disease conditions under the National Policy for Rare Diseases (NPRD) 2021
Although notified as a Group 3(a) condition in the NPRD 2021, patients diagnosed with Pompe disease, Fabry disease, MPS I and II are still not getting the same priority at the Centres of Excellence (CoEs), thereby delaying treatment and posing serious risk to their lives
III. Enhanced Accountability and Efficiency: Direct the Centres of Excellence to expedite the process and ensure the efficient utilization of funds provisioned by the Ministry of Health & Family Welfare.
Many of the CoEs have still not been able to effectively utilise the funds provided by MoHFW, thereby limiting access to life-saving therapy. Repeated appeals to the CoEs and the ministry in this regard have not yielded any impact so far.
They thanked government’s continued support and intervention in this rare case, which largely impacts children. However, they urged the new health minister to prioritise the voice of this often-neglected community, and their needs with the urgency they deserve. There is an expectation that the issue of rare diseases to be on the forefront of the newly elected government’s agenda by including it as a top priority in the 100-day action plan. Providing this level of importance to rare disease patients will not only underscore the government’s commitment to this critical health issue but also catalyze urgent action and resource allocation to improve the lives of thousands of rare disease patients across the country.