Fund crunch in PGI, Chandigarh, hinders treatment of rare diseases

Naina Mishra

Chandigarh, April 2

It took three years for Harmeet Kaur to find that her child is suffering from a rare disease and she has been keeping high hopes for his recovery, as he turns eight this year. He has been diagnosed with duchenne muscular dystrophy (DMD), a rare genetic disease, the treatment of which costs about Rs6-7 crore.

The disease causes progressive weakness. Patients typically lose the ability to walk when they reach 8 to 12 years of age, and die as a result of respiratory or cardiac failure.

The abnormally high cost of treatment for rare diseases is making the treatment impossible even as the PGI, Chandigarh, is made the centre of excellence for rare diseases by the Government of India.

“Initially, my son was diagnosed with kidney infection as he could not pass urine. Gradually, we noticed that he could not walk even at the age of three years. The doctors suspected that the condition could be because of the illness he sustained during the birth. But after running a number of genetic tests, he was diagnosed with DMD,” said Harmeet, who is seeking treatment for her son at the PGI here.

“Doctors say his medicine is under trial, but whenever it becomes available, it will cost around Rs6-7 crore. Even if I sell my home and myself, I cannot collect so much money for my child. He is getting weaker day by day. So many children suffering from a similar condition are forced to use a wheelchair,” she added.

Dr Inusha Panigrahi from the Department of Advanced Paediatrics Centre, PGI, who deals with rare diseases, said, “There is a portal of the Government of India where private entities donate funds for the treatment. The awareness is so low that only Rs65,000 were donated in the past month. A single injection for certain rare diseases like spinal muscular atrophy (SMA) costs even Rs16 crore. Now, who will bear the cost of treatment for such patients? The Central Government also have limited resources.”

Dr Panigrahi shared that every month, there are three or four patients diagnosed with lysosomal storage diseases (inherited metabolic diseases that are characterised by an abnormal build-up of various toxic materials in the body cells). We are treating 10-12 patients and some of them have died. Recently, a patient with Gausher’s disease (inherited disorder that affects many of the body’s organs and tissues) died.

Bittu, a resident of Himachal Pradesh, is seeking treatment at the PGI for his child who is suffering from the Gausher’s disease.

“The annual cost of treatment is Rs70-80 lakh for this disease. We feel helpless as we are unable to arrange the funds.”

Donations Meagre

The National Policy for Rare Diseases (NPRD), 2021, which has the provisions for financial assistance through crowd funding to the patients suffering from the diseases, for which a definite treatment is available but involves very high cost and lifelong therapy. The amount of donations that have been channelled to the portal is Rs1,18,016 to date. The donors have the choice of making donations directly to different centres of excellence.