Early intervention in rare disease can save lives, Centre’s policy an encouraging step: Bruno Jolain, Roche India

Pharma major Roche’s Evrysdi is the first and only approved treatment available in India for patients suffering from spinal muscular atrophy (SMA). It is an expensive drug as it costs Rs 6 lakh a bottle, and patients above 20 kg need about 30 bottles a year in the maximum-use case.

Roche, which introduced Evrysdi drug for SMA patients in India last year, said the response to the drug has been encouraging as close to 200 prescriptions have been generated which outlines that people have shown interest in taking therapy.

Moneycontrol spoke to Bruno Jolain, chief medical director, Roche India, on the company’s plan for the rare disease drug, the pricing challenge and on government policies for the disease. Edited excerpts:

In mid-2021, Roche forayed into rare disease treatment in India with the introduction of Evrysdi for SMA patients. What has been the response so far in India?

We’ve brought Evrysdi extremely fast to India. Before this drug was actually made available and approved, a good number of patients actually could benefit from it through the compassionate use programme (a treatment option that allows for the use of medicines that have not yet been authorised). So far, 70 patients have benefited from this, before Evrysdi was even approved, and continue to benefit from it.

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We continue to supply it free of cost. So it has actually been a wonderful story of commitment and doing things fast in India.

What is the key challenge in the Indian context?

Of course, there are some challenges. I think that the drug is available but the funding aspect and access is really what is ahead of us. That’s what we now are working extremely hard on and we need to find some solutions for that.

The good news is that in the last one year we have had close to 200 prescriptions, which means so many patients have visited doctors. So the initial success has been pretty encouraging, but we have a long way to go.

If we talk in terms of growth, has Roche set a target for Evrysdi in India?

I don’t think we have a number target like that. An ideal scenario is where every patient who needs a therapy for SMA should get one. Early intervention saves lives, because it’s a genetic disease, it’s the world’s single largest genetic cause of death of children. The data says that about 80 percent of children do not see that fourth birthday.

The Indian government has a website for people suffering from rare diseases and people can contribute there. How crucial is the government policy in this direction?

To launch a policy for rare diseases is itself an extremely encouraging step. We already are seeing that certain reimbursement ecosystems are already coming forward and helping patients. We have seen some success in Kerala, where there is a new crowdfunding mechanism. Support is also coming in Rajasthan, where the government has launched a portal where they are looking at rare diseases. I think more and more states will come forward.

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Drugs related to rare diseases are almost always associated with high costs. How significant is the challenge to bring that pricing down and what is the company doing in this regard?

I think it’s important to understand that pricing is a factor of various combinations. It took us nine years to develop this product. Now, there’s development costs, there are trial costs. Drug development is an extremely risky business. More importantly, the addressable patient population is extremely small and then you have this entire thing of duties and taxes.

The government imposes both GST (goods and services tax) and customs duty on the drug. If this can be brought down, the benefit will eventually be passed on to the patients.

We have introduced a patient support programme wherein we said, while there is a particular MRP (maximum retail price) of a product, how do you really make it more accessible to patients? If the patient buys two bottles of it, they get three bottles free and after the second year, it becomes buy one, get two free. So it’s practically discounting at the range of about 60-66 percent.