Pharma major Roche may have earned over a hundred times what risdiplam , a drug used to treat spinal muscular atrophy (SMA), should cost to bring to market, according to calculations by public health activists.
After Roche declined to share information on the cost of clinical trials, Knowledge Ecology International (KEI), a US-based non-profit working on access to affordable medicines, calculated the costs and pegged them at about $50 million. Roche earned $5.8 billion from the sale of risdiplam in the 2021-2024 period, about 115 times the calculated cost.
In response to the calculations sent to it by KEI, Roche stated: “Only a small fraction of projects make it ‘from bench to bedside’ and we have to absorb the cost of failing projects as well, while your calculation only addresses successful development. Similarly, our dedication to innovation extends to the continuous development of next-generation therapies—a commitment clearly reflected in our ongoing development work of new treatments in SMA.”
The three trials of risdiplam cited in the US Food and Drug Administration (FDA) involved 467 participants. The four trials with a start date before FDA approval involved 120 participants. KEI estimated per patient cost of conducting the trial by combining several data sources on the cost of conducting clinical trials.
The United States provides a tax credit for qualifying clinical trials for rare diseases. After calculating the cost of the clinical trials, when KEI applied the orphan drug tax credit to the trial cost, the net cost ranged from $11 million to $25 million. Clinical trials typically constitute 60-70% of drug development costs.
Roche was also awarded a Rare Pediatric Disease Priority Review Voucher (PRV) in August 2020. The PRV is an incentive designed to encourage development of drugs and biologics for serious or life-threatening rare diseases. The PRV allows earlier market entry and extends the effective period of the monopoly by several months. The PRV can be sold to third parties. In 2020, the market value of a PRV was roughly $100 million, more than the Roche outlays on trials, pointed out KEI.
Since 2003, when it was established, the Spinal Muscular Atrophy Foundation (SMAF) has invested $100 million to develop drugs for this condition. Roche paid $30 million dollars to get an exclusive worldwide license to PTC Therapeutics' SMA programme, which it had developed in collaboration with SMAF.
Roche’s costs of manufacturing the drug are not significant, with a year of treatment requiring less than 2 grams of risdiplam, which can probably be manufactured by Roche at less than $50,000 per kilo, or $50 per gram, estimated KEI. In India, the treatment with risdiplam typically costs Rs 72 lakh per adult patient per year, which is unaffordable for most.
According to Roche’s response to TOI, as of October 2024, of the 168 patients receiving Roche’s treatment for SMA, 56 continued to be treated for free under the company’s Compassionate User Program (CUP), 53 were covered under various government policies -- Central Government Health Scheme (CGHS), Defence, Employee State Insurance (ESI) and Railways -- and the remaining 59 purchased the drug under Roche’s Patient Access Program, launched in 2021, “under which Roche provides free bottles of the treatment for every bottle that is purchased”.
Cure SMA, a trust formed by parents of children with SMA, has 1,800 patients registered and the government portal for rare disease has 700 SMA patients registered.
Lack of affordability has made patients approach courts to help bring down costs through local manufacture or through price negotiation by the government. Roche has opposed generic manufacture of the drug by an Indian company. A bottle of risdiplam costs Rs 2 lakh in India while it costs just Rs 44,700 in China and Rs 41,000 in Pakistan, according to an affidavit filed by an SMA patient in court. The Indian generic manufacturer, NATCO, has pegged the cost at Rs 15,900 per bottle with scope for further reduction if the government purchased it in bulk.
“On average, it takes ten years to develop a new drug and only one out of ten drugs in early development will eventually make it to patients. And it is very complex to estimate the total cost of developing a new medicine, especially for extremely rare neurological diseases such as SMA. The cost of development goes well beyond preclinical research, clinical trials, and post-marketing studies, requiring many years of investment. There are also costs associated with ensuring safe and quality manufacturing setup and operations, and building a global and local infrastructure to ensure access around the world.,” stated Roche in its response to TOI.
After Roche declined to share information on the cost of clinical trials, Knowledge Ecology International (KEI), a US-based non-profit working on access to affordable medicines, calculated the costs and pegged them at about $50 million. Roche earned $5.8 billion from the sale of risdiplam in the 2021-2024 period, about 115 times the calculated cost.
In response to the calculations sent to it by KEI, Roche stated: “Only a small fraction of projects make it ‘from bench to bedside’ and we have to absorb the cost of failing projects as well, while your calculation only addresses successful development. Similarly, our dedication to innovation extends to the continuous development of next-generation therapies—a commitment clearly reflected in our ongoing development work of new treatments in SMA.”
The three trials of risdiplam cited in the US Food and Drug Administration (FDA) involved 467 participants. The four trials with a start date before FDA approval involved 120 participants. KEI estimated per patient cost of conducting the trial by combining several data sources on the cost of conducting clinical trials.
The United States provides a tax credit for qualifying clinical trials for rare diseases. After calculating the cost of the clinical trials, when KEI applied the orphan drug tax credit to the trial cost, the net cost ranged from $11 million to $25 million. Clinical trials typically constitute 60-70% of drug development costs.
Roche was also awarded a Rare Pediatric Disease Priority Review Voucher (PRV) in August 2020. The PRV is an incentive designed to encourage development of drugs and biologics for serious or life-threatening rare diseases. The PRV allows earlier market entry and extends the effective period of the monopoly by several months. The PRV can be sold to third parties. In 2020, the market value of a PRV was roughly $100 million, more than the Roche outlays on trials, pointed out KEI.
Since 2003, when it was established, the Spinal Muscular Atrophy Foundation (SMAF) has invested $100 million to develop drugs for this condition. Roche paid $30 million dollars to get an exclusive worldwide license to PTC Therapeutics' SMA programme, which it had developed in collaboration with SMAF.
Roche’s costs of manufacturing the drug are not significant, with a year of treatment requiring less than 2 grams of risdiplam, which can probably be manufactured by Roche at less than $50,000 per kilo, or $50 per gram, estimated KEI. In India, the treatment with risdiplam typically costs Rs 72 lakh per adult patient per year, which is unaffordable for most.
According to Roche’s response to TOI, as of October 2024, of the 168 patients receiving Roche’s treatment for SMA, 56 continued to be treated for free under the company’s Compassionate User Program (CUP), 53 were covered under various government policies -- Central Government Health Scheme (CGHS), Defence, Employee State Insurance (ESI) and Railways -- and the remaining 59 purchased the drug under Roche’s Patient Access Program, launched in 2021, “under which Roche provides free bottles of the treatment for every bottle that is purchased”.
Cure SMA, a trust formed by parents of children with SMA, has 1,800 patients registered and the government portal for rare disease has 700 SMA patients registered.
Lack of affordability has made patients approach courts to help bring down costs through local manufacture or through price negotiation by the government. Roche has opposed generic manufacture of the drug by an Indian company. A bottle of risdiplam costs Rs 2 lakh in India while it costs just Rs 44,700 in China and Rs 41,000 in Pakistan, according to an affidavit filed by an SMA patient in court. The Indian generic manufacturer, NATCO, has pegged the cost at Rs 15,900 per bottle with scope for further reduction if the government purchased it in bulk.
“On average, it takes ten years to develop a new drug and only one out of ten drugs in early development will eventually make it to patients. And it is very complex to estimate the total cost of developing a new medicine, especially for extremely rare neurological diseases such as SMA. The cost of development goes well beyond preclinical research, clinical trials, and post-marketing studies, requiring many years of investment. There are also costs associated with ensuring safe and quality manufacturing setup and operations, and building a global and local infrastructure to ensure access around the world.,” stated Roche in its response to TOI.
You may also like
Bring missing files, court tells ED, halts notices to Gandhis
Labour's appeal for homes for migrants as Channel crossings skyrocket
Congress targets govt: Security, intel failure behind attack
How to apply for the new series of Big Brother as Jack P Shepherd wins celebrity version
Paddy McGuinness breaks silence over Freddie Flintoff 2 years after crash ordeal
