Representatives of patients with rare diseases are calling for government intervention to access treatment

Patient representatives and treatment campaigners have urged the Government to encourage domestic drugmakers to produce generic versions of drugs to treat rare diseases, including spinal muscular atrophy (SMA) and cystic fibrosis.

Pointing to Risdiplam used to treat SMA, they said, Roche sought a permanent injunction to prevent Hyderabad-based Natco Pharma from manufacturing a generic version of Risdiplam on the grounds that it was a patent infringement. That would affect access to affordable versions of the drug, activists said, because it blocks generic supplies.

SMA related petitions

The Delhi High Court has taken up SMA petitions since early 2021, underscoring the need for a coordinated treatment framework given the challenges of high drug costs and limited access, they said.

“Roche’s patent for Risdiplam runs until May 2035, allowing them to charge around ₹6 lakh per bottle, while manufacturing costs suggest the drug could be made available at a price of ₹3,024 annually,” they said the activists. The monopoly on the drug could hamper the Union Health Ministry’s efforts “to benefit from domestic manufacturing and competitive pricing through joint procurement (auction) strategies,” they added.

What Roche said

In his response to the development involving Risdiplam, Roche said line of business“Our focus remains on discovering and developing innovative medicines and diagnostics that will change the standard of care in the future. At the same time, we are also committed to protecting our innovations within the scope of the applicable laws in the countries in which we operate, and we believe that the laws provide sufficient guarantees for the protection of innovation.”

“At Roche, drug innovation and its access to patients come first,” he said, adding that they are committed to delivering healthcare innovations that improve patients’ lives “while ensuring that as many patients as possible have access to our products”.

“Overwhelming” barriers.

Meanwhile, for cystic fibrosis, health advocates pointed out, there were no options for patients in India because patent barriers were “overwhelming”.

The CF drug continues to be unavailable in India because Vertex (the holder) has not registered it with Indian regulators, they said, adding that it has an annual price tag of around INR 1 crore for those who import the drug under a personal use license . .

Last year, a patient-led campaign was organized in South Africa, Ukraine, Brazil and India for access to the CF therapy, Trikafta, from US biotech company Vertex Pharmaceuticals. The company had then told this correspondent that it was exploring initiatives to make its drug affordable in India.

A company spokesperson said: “In some low- and middle-income countries, such as India, where access challenges are significant and where we do not currently have a presence, we are actively evaluating options that can facilitate access to our medicines , including a product donation program, which we started piloting last year.”