Khyati was four months old when her parents suspected something was wrong. She was not able to lift her head up, a typical milestone for infants her age. A month later, her parents were told to get Khyati tested for spinal muscular atrophy, or SMA, a genetic disease, causing muscle weakness and progressive loss of movement, due to deterioration of motor neurons. Muscle functions that are affected include breathing, swallowing and basic movement and when left untreated in its most severe forms, it can lead to permanent ventilation or death in 90% of cases by age 2. Khyati is now 10 months old, and her parents have a herculean task ahead of them â€” to raise Rs 16 crore to procure a one-time drug called Zolgensma by Novartis, one of the few drugs for SMA. It can only be administered to children below the age of two to arrest the progression of the disease.
Khyati was prescribed Zolgensma in February, when she was six months old. SMA has multiple types, but most children have type 1 SMA â€” where they do not have the SMN1 gene. Khyati was born with two copies of SMN2.
The recent spotlight on Zolgensma is due to crowdfunding campaigns to raise funds to afford the drugâ€™s Rs 16 crore price tag. Some have been successful â€” Ayaansh in Hyderabad and Muhammed in Kannur â€” but many more parents, such as Khyatiâ€™s parents, have been trying to garner funds for months.
Raman, Khyatiâ€™s father, tells TNM that they started a crowdfunding campaign in March, but then hit a roadblock because of the second wave of COVID-19. Though itâ€™s now picking up again, the family still has a long way to go. So far, they have managed to raise Rs 3.3 crore. [You can donate to Khyatiâ€™s campaign here.]
Pricing of the drug
It is important to note that Zolgensma isnâ€™t marketed in India, and has to be imported from the US if prescribed by a medical practitioner. At $2.125 million, it is touted to be the worldâ€™s most expensive drug. The price, according to industry sources, is due to the low incidence of the disease, which means a smaller number of patients, as well as the significant costs in the research in gene therapy and the drug development process. For now, when children are prescribed Zolgensma, their guardians must write to Novartis and import the same from the US.
Another reason for the cost of the drug is because the only way to obtain it is to import it. A senior executive at a pharma firm says drug pricing varies significantly between India and the USA â€” in India, the cost of a product is only a fourth or a fifth of what it costs in the USA. The price of Zolgensma is due to its purchase at tariff from the company in the US.
So how can the cost of the drug go down? The only way, according to the pharma executive, is if the drug is introduced in India, and negotiations are held by the Union government.
A drug is only introduced to a market like India after it is introduced in the country of innovation â€” the location where the drug is developed â€” as well as when there is a sizable market opportunity, expected prevalence of the disease in the future and feedback from healthcare practitioners, among other factors.
Though Novartis did not offer a timeline for if or when it would bring the drug to India, the pharma executive, who is not associated with the company, noted, â€śI think they will be very hesitant because you need to have a minimum volume. You donâ€™t want to sell 100 vials or 1000 tablets of a drug. You want to sell a sizable quantity because while you may distribute any quantity, there may be a lot of onerous obligations on you to track adverse events, run a pharmacovigilance process, ensure regulatory activity, etc.â€ť
â€śThe only way it can be reduced is if it is licenced in India by someone, and priced in India depending on the purchasing power in India,â€ť the pharma executive adds.
This leaves few avenues for the cost of the drug to be subsidised in the country at present â€” and that falls on the government to directly approach Novartis to negotiate the price of a drug. Drug pricing is based on the country, and in this case, would be a licence by the Drugs Controller General of India for the Indian region.
A Novartis spokesperson told TNM that the final pricing of Zolgensma and reimbursement decisions are determined at the local level, and that it is priced â€śat around 50% less than multiple established benchmarks, including the 10-year cost of current standard in chronic SMA treatment.â€ť That means, according to Novartis, that the one-time use of Zolgensma is ultimately more cost-effective than other drugs, which require years or lifelong treatment cycles.
â€śIn the EU, Zolgensma is cost-effective vs. the current standard in chronic care, resulting in cost savings to healthcare systems. Within 10 years or less post-treatment, some countries across Europe could realise cost savings estimated between â‚¬0.4 - â‚¬1.9 million in drug cost per patient,â€ť the company added.
What is Zolgensma?
Zolgensma replaces the function of the missing or non-working SMN1 gene with a single dose. â€śThe therapy is administered as a single, intravenous (IV) infusion, and delivers a new, working copy of the SMN1 gene into a patientâ€™s cells, halting disease progression,â€ť a Novartis spokesperson said.
The Novartis drug is one of the only three drugs in the world for SMA â€” Biogenâ€™s Spinraza and recently, Roche and Genentechâ€™s Evrysdi are the other two. Spinraza costs $750,000 for the first year and $375,000 for every subsequent year. Evrysdi, an oral drug which may be coming to India soon, requires the patient to take it for their entire lifetime, and can cost up to $340,000 per year.
â€śEven the stopping of progression of the disease â€” the degradation and the dying of these neurons and muscles is arrested, that itself is a big win for us,â€ť Archana Panda, co-founder and director of the CureSMA Foundation of India, a parent-led community for SMA patients, says.
The question of how to raise funds
While insurance companies as an avenue for payment are available to some families in the United States, insurance is not an option for families in India currently. Archana, of the CureSMA Foundation, notes that they approached the Insurance Regulatory and Development Authority, but anything genetic is not covered, and nor is the coverage of insurance extensive in the country.
According to Khyatiâ€™s father Raman, the only help they receive under some insurance policies is hospitalisation costs as motor neuron diseases.
The only other way to access the drug is the companyâ€™s global Managed Access Program launched last year, where the company provides by a lottery system upto 100 doses of the drug at no cost in countries where gene therapy is not approved. Children in various countries, including in India, have received the drug through this program.
While the company refused to divulge figures on how many children have received the drug so far in India, it stated that in total, over 1,200 children worldwide have received the drug.
Apart from the cost of the drug itself, customs duty and GST can both be levied on import, which comes up to roughly 30% of the cost of the drug. Finance Minister Nirmala Sitharaman said earlier this year that the customs duty on such lifesaving drugs is waived off, but will attract GST of 5%. Ad-hoc exemption is given on a case-to-case basis when a request is received, she had said.
Currently, parents of children importing this drug do still have to obtain the paperwork for a customs duty waiver, as well as appeal to the Union government to exempt the imported drug from GST.
Archana questions why the process canâ€™t be streamlined for life saving diseases. Cure SMA says it has over 550 registered patients in India, though the number in India is definitely far higher, she noted. The organisation has been reaching out to various stakeholders to increase awareness, and is working on trying to make drugs affordable.
In order to make the drug more accessible, public health activist Dinesh Thakur says that two measures primarily work in other countries â€” better organisation and coordination among the small group of people afflicted with rare diseases; and better representation and lobbying with the government for more responsive policies.
Policymaking and accessibility
The Union government recently released the National Policy for Rare Diseases, 2021, which classified various rare diseases into three categories, based on level of treatment available and its cost. It states that the government will not be able to cover the high cost of treatment, and that it will set up a voluntary crowdfunding mechanism.
But the recommendations of crowdfunding and CSR as two alternatives are poor policy choices, says Dinesh Thakur.
â€śThe policy needs to leverage the ability of the government to procure drugs and make it more affordable. But then, like most policies in India, this one seems to have been written bereft of the benefit of any underlying data about the number of patients afflicted with rare diseases,â€ť he said.
In addition, the policy neither provides timelines nor does it outline eligibility.
Archana, too, says they are extremely unhappy with the policy, and notes that classification should have been prioritised on how rare and life-threatening the disease is.
For now, CureSMA has tied up with crowdfunding platforms to help raise funds for the drug.
Archana questions why the government has not worked on bringing drugs which have been available for the last four years (Spinraza received USFDA approval in late 2016), due to which no data is available as well for the Indian population.
CureSMA has even suggested a multi contributory model, wherein some percentage of funds can come from the Union government, some from state government, some from MP and MLA funds, from temple trusts, from CSR funds and so on, stating that post COVID-19, priority on healthcare must be at its highest.
And as things stand, Khyatiâ€™s hope is in the remaining amount being raised.
â€śIt has been done four times till now [cases where the money has been raised]. We frankly believe there is a power in the community and it is also being proven every time. Since it has happened four times, that is the only ray of hope for us,â€ť Raman says.