Access to SMA therapy: Iman’s Story

Recent years have seen several blockbuster treatments enter the market, many rooted in publicly funded foundational and pre-clinical research, and benefiting from government subsidies. In particular, we have conducted extensive research on Spinal Muscular Atrophy (SMA), which has regularly hit the headlines in recent years due to the cost of its treatment options. Following the 2016 approval of Spinraza, the first disease-modifying therapy for SMA, Novartis brought Zolgensma to market with a list price of $2.1 million per dose in 2019 (the current UK list price is £1.79 million/USD $2.48 million per dose, link). KEI has written extensively on the patent landscape and public funding of Spinraza, and more recently on the public funding involved in the development of Zolgensma.

KEI is working towards global access to these essential therapies. We are particularly focused on the access gap in middle- and low-income countries. Lack of ability to pay leaves these treatment options unregistered in these countries, not seen as viable markets by drug companies. Although much of KEI’s research and advocacy focuses on system level changes, we also push to make the case for access for individual patients where we feel we can help.

Following on from our interview with Tasnim [link], we reached out to two drug companies that manufacture the only existing approved treatment options for Iman given her age, requesting access to Biogen, who make Spinraza, and Roche, who make Evrysdi. Neither drug has even been registered in South Africa, let alone is reimbursed through either the national health system or by private insurers. As such, access would be through either Section 21 of the South African Medicines Act, where clinicians apply to allow the sale of unregistered products, or through patient access schemes where clinicians would apply directly to drug companies. In the case of the former, the price of the medicine in question has been wholly prohibitive. Iman’s paediatric neurologist has been unsuccessful in all attempts to pursue the latter.


KEI contacted Mr. Vounatsos, Chief Executive Officer at Biogen, requesting he explore options for enabling access to Spinraza for Iman on 20th April 2021. The following day, we received several responses, including from the President and Medical Head for the Intercontinental Region (covering Latin America, Australia, Asia, Japan, Middle East and Africa), and were eventually connected with the Medical Head, MART (Middle East, Africa, Russia/CIS & Turkey).

On 24th May 2021, KEI received the following:

Dear Kavian,

Biogen has conducted extensive internal discussions and also liaised with Clinigen and agreed to supply Spinraza for the lowest price that is possible for South Africa.

The price will be calculated on the number of vials required (7 vials for the first year and then 3 vials/year thereafter).

It you feel that this is could be a practical solution, I’d be grateful if you could advise on supply requirements and we can work from there.

My best wishes,

In response, KEI asked what this lowest cost per vial would be for South Africa and how this was calculated. Biogen offered to “arrange a meeting with yourself and some key stakeholders in Biogen, to discuss the situation in detail, in order that we can fully explore the dynamics/logistics etc of the request.”

This call was organised for 11th June 2021. Notably, the presence of Tasnim, Iman’s mother, was refused. Despite promise of presence of ‘key stakeholders’, we were informed on the call that the staff members from the business department would not be attending, only the clinical representatives. As such, issues of pricing could not be discussed.

Several attempts were made to reorganise the call. Despite confirming our availability at as short notice as a day, Biogen subsequently cancelled the rearranged call. We await a further rearranged date, over 6 weeks since the original call.


Our correspondence with Roche was even less fruitful.

KEI similarly contacted Dr Severin Schwan, Chief Executive Officer of the Roche Group, also on 20th April 2021.

We received a swift response from the senior vice-president of Roche, before being passed onto the ‘Lifecycle Leader’ and Global Scientific Director, and finally being connected with the South African representative, all within 48 hours of KEI’s initial email. As requested, KEI connected Roche with both Iman’s mother, Tasnim, and Iman’s paediatric neurologist.

Now, however, more than three months down the line, there has yet to be contact made by Roche’s South African representative regarding access to Risdiplam with either the patient’s family, clinician or us, despite several emails following up on the matter.


Despite direct attempts to liaise with both companies by the mother and the patient’s clinician, and further attempts by KEI to this end, no meaningful progress has been made towards attaining access to therapy for Iman.