Safety vs affordability: Keralam parents question SMA drug switch

When Lakshmi, of Neyyatinkara, Thiruvananthapuram, the mother of a three-year-old boy with Spinal Muscular Atrophy (SMA), noticed that SAT Hospital in Thiruvananthapuram had changed the medicine supplied to her child, the labels on the new packaging alarmed her. The medicine, supplied in place of Roche’s expensive drug, carried the words 'physician sample' and 'for clinical trials only'.

For Lakshmi, the concern was not about the price of the medicine but whether the cheaper alternative could deliver the same results as the treatment her child had been receiving.

“There are many children who were diagnosed with the prediction that they would never walk, but are now running around. We are all for cheaper medicines. But we need clinical assurance that the new medicine is equally effective and safe. When it comes to our children, we cannot take chances. We need to know whether the medicine has been adequately evaluated, whether it works as well as the one they were receiving, and whether there is enough evidence to assure us that the change will not affect their progress,” she said.

Risdiplam drug row

Lakshmi’s concerns have turned into a controversy over the supply of Risdiplam, a drug used to treat SMA, through the state’s free-treatment programme. Parents and Opposition Leader Pinarayi Vijayan have questioned the switch to Natco Pharma’s generic version of the medicine, while the government has defended the preference for Indian-made drugs, citing quality assurance and regulatory oversight.

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Health Minister K Muraleedharan said the government would look into the concerns raised by the Opposition and parents. “The expert committee is of the view that Indian-made drugs should be given preference, with quality assurance and control. As of now, no complaints have been received about the quality of the drugs. It was the earlier government that took the decision to move to generic medicines. If the Leader of the Opposition has concerns, and parents have concerns, the government will inquire into them,” he said.

The controversy has brought into focus the balance between making expensive treatment affordable and ensuring that families have adequate information and confidence in changes to medication for a serious genetic disorder.

The cost of treatment and the switch

Roche markets Risdiplam under the brand name Evrysdi, while Natco Pharma has introduced a generic version. Figures cited in the debate put the price of Roche’s medicine at around Rs 6-7 lakh, against approximately Rs 16,000 for Natco’s version. Kerala had negotiated a price of around Rs 1.5 lakh for Roche’s medicine for its free-treatment programme.

The previous Left Democratic Front government had provided free treatment to children up to the age of 12 and announced plans to extend the benefit to patients up to 25 years, with Rs 30 crore earmarked for the purpose. Around 118 children currently receive the medicine through the government programme.

The switch to Natco’s product under the UDF government has raised anxiety among some parents, who say the change in brand and packaging labels was not adequately explained.

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For families whose children have shown improvements while receiving the earlier medicine, the question is not simply whether a cheaper drug is available, but whether the new product offers comparable clinical benefits.

What does bioequivalence mean?

Senior pathologist Dr K P Aravindan, former president of Kerala Sastra Sahitya Parishath, the popular science organisation, argues the controversy risks discrediting an Indian pharmaceutical company that has helped make expensive medicines more affordable. He cautioned against describing the switch as drug trials on children without establishing the relevant facts. “Calling this ‘drug trials on children’ is the style of those seeking sensationalism,” Aravindan said.

“Bioequivalence has three components. The first is whether the formulation contains the required quantity of the active pharmaceutical ingredient. The second is whether the drug is adequately absorbed into the body. These are the preliminary studies that a drug must clear to obtain regulatory approval. The third component is whether the medicine produces the intended clinical benefit in patients. That can be established only by assessing its effects in patients. In the case of Natco’s Risdiplam, I do not know whether this clinical effect has already been established or whether it is still being evaluated,” he said.

Aravindan cited Natco’s record in making expensive cancer medicines more accessible and its role in legal battles over access to patented drugs. He argued that portraying the company as having endangered children without establishing the facts could benefit multinational pharmaceutical companies holding patents on expensive medicines.

Pinarayi Vijayan demands inquiry

Leader of Opposition Pinarayi Vijayan has accused the Health Department of failing to address concerns over the distribution of medicines carrying the labels “Physician Sample” and “For Clinical Trials Only”.

“Medicines bearing experimental labels being administered to sick children without the full consent of their parents (informed consent) cannot be treated as a trivial matter,” he said.

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He alleged violations of the Drugs and Cosmetics laws and procurement rules, and demanded action against those responsible. “Are children suffering from serious diseases being turned into mere experimental subjects?” he asked.

Vijayan also accused the government of disregarding concerns raised by Cure SMA, the collective of families affected by the disorder. He said he had written to the Health Minister about the issue but had received neither a response nor action.

He further alleged that the allocation of Rs 30 crore announced by the previous government to extend free treatment to patients up to 25 years had been reduced.

Regulatory status

The minister’s response that the government would inquire into the concerns leaves the key questions to be addressed through that process, including the medicine’s regulatory status, the reasons for the labelling and the information provided to families.

The debate is not merely about the price difference between two versions of Risdiplam. Natco’s lower-priced medicine could significantly reduce the cost of treatment and help the state sustain or expand support for people with SMA. But affordability alone cannot resolve concerns about a change in medication, particularly when parents believe their children have benefited from the earlier treatment.

Aravindan has argued that the controversy should not be used to undermine generic medicines without evidence. Parents such as Lakshmi, meanwhile, want clear clinical assurance before accepting a switch in treatment.

The government now faces the task of establishing the medicine’s regulatory status, clarifying the labels and explaining the clinical evidence to affected families. For parents, the central concern remains whether the cheaper alternative can offer the same assurance of safety and effectiveness as the medicine their children have been receiving.