Gene Therapy

Gene Therapy May Be More Cost Effective for Treating Haemophilia B Than Other Methods

Georgi Makin
7 June 2021

A team from St Jude Children’s Research Hospital has explored the costs associated with manufacturing and distributing a severe haemophilia B gene therapy compared to other treatment opportunities, providing the first report of its kind for gene therapy.

Through investigations into the costs associated with gene therapy production, the team reported a disparity between the costs calculated and price currently reported for haemophilia gene therapy development of over $2 million.

Relying on data from the hospital itself, the researchers calculated the cost per patient administered the gene therapy for haemophilia B, including the respective cost of manufacture, distribution and 5-year follow up. The total cost per patient, the team deduced, stood at $87,198.

As Nickhill Bhakta from the St Jude Global Paediatric Medicine Department explained: 

“Many factors go into setting the price of new pharmaceutical drugs. Production costs are just part of the equation, and while they do not tell the whole story, they are all too often missing from the conversation.”

For insurers and third-parties concerned about the issue of reimbursement, data surrounding the comparison of treatments available for haemophilia B have previously been limited.

The team hopes the report – published in the journal, Blood –  will facilitate evidence-based discussions around price equity, as well as promoting greater transparency around the costs associated with lifesaving therapies.

Current treatment opportunities for haemophilia B include infusions with clotting-factor replacement therapy, which may be necessary several times a week, with costs reportedly ranging from $300,000–776,000 per year.

A gene therapy for haemophilia B, however, involves a single outpatient infusion, and could be effective for several years.

“More and more data show the success of gene therapy in haemophilia with reduced bleeding rates and factor use and significantly improved quality of life for these patients,” added Ulrike Reiss from the St Jude Department of Haematology. 

The analysis conducted by the St Jude team focussed on an experimental AAV-mediated gene therapy developed by St Jude as well as University College London and the Royal Free Hospital. The team compared the costs associated with, and cost effectiveness of the gene therapy against two other approaches: on-demand and preventative clotting factor replacement therapies.

Over 100 treatment scenarios were considered from the perspective of third party payers, with additional factors – such as age, for example – also included in the analysis.

The team determined that for treatment of haemophilia B, even if the cost of gene therapy costed up to $3 million, gene therapy would indeed be the most cost effective treatment opportunity in 92% of the scenarios considered.

As Nancy Bolous from St Jude Global Paediatric Medicine summarised: 

“Gene therapy is different because unlike other treatment approaches, it is a long-lasting, one-time treatment and may require a big upfront payment, a payment that can make the affordability seem questionable. Our analysis’ importance is that it sheds light on the many factors that could play a role in the decision-making process regarding reimbursement.”

While the report focussed solely on treatments for haemophilia B, the team has suggested it could serve as a template for future cost effectiveness studies of other novel gene therapies.

Source: St Jude Children’s Hospital press release