Spinal Muscular Atrophy drug: regulatory approval should guide Kerala’s procurement policy, say experts

The treatment of Spinal Muscular Atrophy (SMA), a rare progressive genetic disorder that destroys motor neurons and weakens muscles, leading to severe disability and sometimes, premature death, is emerging as a contentious issue in Kerala.

Kerala has, since 2021, been procuring the high-priced patented SMA drug, Risdiplam by Roche pharma—which costs ₹5.4 lakh a bottle and was being procured by the State government at a negotiated price of about ₹1-₹1.25 lakh—to be given free of cost to SMA patients in the State.

But, now that a generic version of the drug by Natco pharma, approved by drug regulatory bodies, is available in the country—priced at 97% lesser cost than its patented version—the State government has been looking at procuring the generic version as this will enable the State to widen the drug access to more SMA patients and utilise the savings towards the treatment of other rare diseases, which are equally expensive.

Advocacy groups’ demand

However, patient advocacy groups such as Cure SMA have been arguing vociferously in Kerala that the efficacy of the generic drug is yet to be tested through clinical trials and that the State government should continue to supply the patented molecule till the generic’s efficacy is proved.

Public health experts and doctors have now urged the State government that public health is all about maximising access to people and that unless credible scientific evidence demonstrates that the approved generic is less safe or less effective than the original molecule, regulatory approval should remain the primary basis for procurement decisions in publicly funded health programmes.

The irony is that, the debate over patented drug versus generics over SMA drug is something unique to Kerala—the only State to utilise public funds for providing free SMA treatment—while in the rest of the country, the complaints are about SMA patients not getting adequate access to the generic drug, priced at ₹15,900 a bottle.

“Generic medicines do not need to repeat clinical trials, they just need to demonstrate bioequivalence—that is, the generic drug should demonstrate that it delivers the same amount of active ingredients into the bloodstream at the same rate as the original. I can understand an SMA patient’s mother feeling anxious about quality issues but the generic drug manufactured in India alone can make SMA treatment accessible to patients,” a prominent neurologist in the capital said.

Approved nationally

“There is no room for a debate here because the generic version of Risdiplam has been approved for marketing by national drug regulatory bodies. Generics are what makes expensive drugs affordable to all sections of society. The government would be able to procure the generic SMA drug—Risdinat—at further lower prices so that we can sustain the treatment for a lot many SMA patients,” a senior Health official said.

He pointed out that there are several other rare diseases and that the government has to take care of all those patients too with the limited resources at its disposal.

“Kerala cannot continue to procure the original molecule at high prices when an approved generic formulation is available at a fraction of the price. An adult SMA patient weighing over 20 kg requires roughly 2.5 bottles every month. Using the money needed to procure a single bottle of the patented drug, nearly three months of treatment can be ensured for an adult patient with the generic medicine. The procurement policy of the State needs an urgent review,” said B. Ekbal, public health expert.

Newly approved generic medicines often accumulate long-term post-marketing data only after they enter widespread clinical use. Public procurement policy cannot reasonably be based on speculative concerns, he added. In the absence of evidence demonstrating any clinically meaningful deficiency in the generic formulation, continuing to procure a product costing nearly eight times more cannot be justified on speculative grounds alone.

Ultimately, the success of a publicly funded health programme should be measured not by how much it spends on medicines, but by how many patients receive timely, effective treatment with the resources available, Dr. Ekbal added.