With the cost of drugs for spinal muscular atrophy (SMA) running into crores of rupees a year, a Rajya Sabha MP has written to the health minister requesting him to operationalise provisions in the National Policy for Rare Diseases 2021 “to authorize one or more generic manufacturers to produce Risdipalm”, a lifesaving drug for children born with SMA. Drug pricing experts estimate that generic production of the drug by a local manufacturer could bring down the treatment cost from one crore rupees to just over Rs 3,000.
“Considering the technological capacities of the Indian pharmaceutical industry, Risdiplam can be produced locally at a fraction of the current price and made available to all needy people. However, the patent protection on the Risdiplam legally prevents the local companies from producing the affordable generic version. According to news reports, Roche the patent holder of Risdiplam, approached the Delhi High Court to prevent an Indian company from producing the afordable version of Risdiplam,” stated the letter adding that clearly, the quest for high profits was resulting in the denial of a dignified life to SMA patients.
The maximum retail price of one bottle of Risdiplam is a little over Rs 6.2 lakh. Therefore, the cost of Risdiplam can be between Rs 72 lakh for children, and up to Rs 1.86 crore for an adult because an adult needs nearly 30 bottles a year. Rajya Sabha MP Harish Beeran pointed out in his letter that experts had calculated that through the local production of Risdiplam it could be made available to Indian patients for just Rs 3,024 per year.
Para XI of the implementation strategy of National Policy for Rare Diseases 2021, states: “Department of Pharmaceuticals, Department for Promotion of Industry and Internal Trade (DPIIT) will be requested to promote local development and manufacture of drugs for rare diseases by public and private sector pharmaceutical companies at affordable prices and take legal/legislative measures for creating a conducive environment for indigenous manufacturing of drugs for rare diseases at affordable prices.”
Citing this para, the letter added that even public sector units (PSUs) could be encouraged for local manufacturing of Risdiplam as it was a small molecule which was relatively simple to manufacture, unlike the other two drugs, Zolegsma a gene therapy for treating children below two years, and Nusinersen or Spinraza, an injection used to treat children and adults. Section 100 of the Patents Act 1970 also provides enough powers to the government to authorize one or more generic manufacturers to produce Risdipalm in public interest.
It is estimated that 8,000-25,000 kids are born every year in India with SMA. However, without systematic screening this is only a guesstimate. With Zolegsma costing Rs 17 crore and Risdiplam costing about a crore, it is beyond the reach of both central and state governments as well as the people, stated the letter.
Beeran urged for measures to screen for SMA, establishment of a registry for SMA and other rare diseases, R&D funds to develop low-cost gene therapy option for SMA and for national pool procurement of medicines and diagnostics for SMA and other rare diseases.
