India’s rare diseases policy hangs in balance

More than 70 million people across India suffering from rare diseases do not have any government policy to fall back on—thanks to the abrupt withdrawal of the national policy for treatment of rare diseases (NPTRD) by the central government, which it announced in the Delhi high court on November 30. All that this significant population can look forward to is ‘an interim arrangement’ which the government is supposed to present in the high court on February 8, 2019.

Following the move, questions arise as to why a policy, which was in place for more than a year and in consideration for a corpus of Rs 100 crore, was suddenly taken off, and what will come next? According to a gazette notification issued on December 19, 2018, by the Union ministry of health and family welfare: “The central government has decided to review the policy in light of information and updates received for further improvement. It has been kept in abeyance till a revised policy is issued.”

While experts are scrambling to find a reason behind the government’s stand, another notification, issued on December 16, 2018, says that a 10-member committee has been formed under the chairmanship of Director General of Health Services, Dr S Venkatesh, to draft a revised policy.

The committee has been given eight weeks time from the date of issuance of the notification. When Down To Earth contacted Ajay Kumar, under secretary, who issued the notification on the behalf of the government, he said, “No meeting of the committee has taken place yet. And, therefore we, at this stage, can’t say anything conclusively about the deadline of the submission of the report.”

Among the four terms of reference of the committee, one is to define ‘rare diseases’ for India. “This is no less than criminal. People are dying due to lack of medicines and you say you are looking for definitions. You can define and improvise that definition in time, but till then you can at least start giving them medicines,” Prasanna Shirol, co-founder and executive director of Organisation for Rare Disease India (ORDI) told Down To Earth.

Not every rare disease warrants medicines. According to government’s own policy document, there are 7,000 – 8,000 rare diseases, but less than 300 have therapies available to treat them. About 95 per cent rare diseases have no approved treatment and less than one in 10 patients receive disease specific treatment.

However, the problem is availability of drugs, which the government policy itself underscored. “Where drugs are available, they are prohibitively expensive, placing immense strain on resources of families, health systems and donor agencies alike… It is estimated that for a child weighing 10 kg, the annual cost of treatment for some rare diseases may vary from Rs 18 Lakh to 70 lakh,” states the NPTRD document.

So far, only around 450 rare diseases have been recorded in India, the most common being Haemophilia, Thalassemia, sickle-cell anaemia and primary immuno deficiency in children, auto-immune diseases, Lysosomal storage disorders such as Pompe disease, Hirschsprung disease, Gaucher’s disease, Cystic Fibrosis, Hemangiomas and certain forms of muscular dystrophies.

Beside a Rs 100 crore corpus of the central government, another corpus was to be maintained by the state governments under the ratio of 60:40. The government’s decision to keep the policy in abeyance gives the impression that it was not a well-thought move.

The fact remains, as stated in the NPTRD, that the recommendations of three senior committees—V K Paul (AIIMS-Delhi), I C Verma (Institute of Medical Genetics Genomics, Sir Ganga Ram hospital) and Deepak K. Tempe (Maulana Azad Medical College)—had defined rare diseases as well as the drugs to be administered to them.

“How could the government negate all this work in one go? In the name of further improvement it was silly to throw the baby out of bathwater,” says Shirol. The policy, incidentally, had well cut out role for six union ministries and spelt out nine short-term and 13 long-term measures. The government U–turn in the high court seems to be sudden while replying to a bunch of petitions filed by rare diseases patients.

A shell-shocked court said in its order: “This court had previously been handed over an Office Memorandum dated 27th July, 2017, in which it had been stated that the Government of India had formulated a National Policy for the treatment of rare diseases in India which had been approved by the Union Minister for Health and Family Welfare. The said National Policy had also been placed on record. Six states, including Delhi, have already constituted committees in accordance with the said National Policy… Without filing any affidavit withdrawing the earlier affidavits and/or the National Policy on record, a presentation (made by health ministry’s joint secretary Manohar Agnani) contrary to the existing policy has been made! This court is of the prima facie view that the somersault does not reflect well on the decision making process in the Ministry.”

The government also admitted in the court that not a single penny was actually assigned to the designated corpus.

The court added, “In the prima facie opinion of this court, the flip flop in the decision making process can prove to be very costly inasmuch as one of the patients whose case was directed to be processed by this court vide order dated 02nd February, 2018, has unfortunately expired during the pendency of the present proceedings.”

Interestingly, counsel for the patients, Ashok Agarwal, says that he did not present any objection to the policy and cannot understand why the policy was put in abeyance. “We did not have any problem. We can only infer that the government had no intention, whatsoever at all, to implement its own policy and therefore this step was taken,” says Agarwal.

Meanwhile, experts say that amid this flip flop, the patient have suffered the most. “About 200 patients had registered after this scheme was notified. Six have died. Many more will because these are progressive diseases. The earlier a treatment starts, better the prognosis is,” says Shirol, hoping that the government would present something concrete in the upcoming hearing on February 8, 2019.