Biogen Announces European Ex-factory List Price for Nusinersen

09 June 2017

Expected to be up to €270,000 (approximately £235,000) per individual per year for the three-dose maintenance year, but the prices the NHS pays for medicines are not necessarily a reflection of the list prices.

We will follow up further on the implications of this announcement. In the meantime, we asked Biogen to tell us more. We received this late yesterday as follows: 

Statement for SMA Support UK, from Biogen, following disclosure of EU list price for nusinersen

On Thursday 1st June 2017, Biogen announced that the European Commission has granted marketing authorisation to nusinersen for the treatment of 5q Spinal Muscular Atrophy (SMA). The approval of nusinersen was based on positive results from multiple clinical studies in more than 260 patients treated with nusinersen, and has demonstrated breakthrough clinical value, making a differences in the lives of people with SMA across all disease types.

In addition to announcing the marketing authorisation, Biogen also disclosed nusinersen’s list ex-factory price (ie, price at the factory) for the three-dose maintenance therapy expected to be up to €270,000 (approximately £235,000) per individual per year – generally in line with the annual cost of other rare disease therapies in Europe. Nusinersen’s first year cost in most cases will be higher due to the initial loading dose regimen that includes four loading doses and two maintenance doses. There is no single EU price, and none of the European country level prices have been published yet:  the price of nusinersen is expected to vary country by country and takes into account a number of differences on a local level.

Biogen recognises that the NHS faces difficult decisions on how to allocate finite resources so that patients have optimum and equitable access to new and innovative treatments. New generations of drugs continue to evolve and shape the systems and processes in place to support appropriate and rapid uptake of medicines that demonstrate meaningful improvements in patient outcomes.

The prices that the NHS pays for medicines are not necessarily a reflection of the list prices available in the public domain.  Prices for branded prescription medicines in the NHS are currently set through negotiation between manufacturers and the Government and are controlled by the Pharmaceutical Price Regulation Scheme (PPRS)1, a voluntary agreement between the Department of Health and the pharmaceutical industry, designed to regulate the profits that companies can make from NHS sales. In practical terms, this means a limit is applied to the overall cost of branded medicines, and results in quarterly cash rebates paid back to the NHS if this limit is exceeded.

In order for the NHS to evaluate which medicines it can fund, there are several routes through which licensed medicines can be assessed and/or commissioned to enable patient access on the NHS. In England, Wales and NI, these routes are managed either by the National Institute for Health and Care Excellence (NICE) or NHS England (in Scotland, this is the Scottish Medicines Consortium and NHS Scotland). NICE is an independent body that closely examines both the clinical benefit and cost effectiveness of medicines, and determines whether the medicine represents value for money to the NHS at the proposed price. If a positive recommendation is made, there is a mandate for that medicine to be commissioned by NHS England or other parts of the NHS.

In addition, pharmaceutical companies can improve the cost-effectiveness of a drug by proposing Patient Access Schemes to enable improved access and support to the NHS – these are financially based or outcome-based and are often a combination of the two.   

For medicines for rare and ultra-rare conditions, NICE has a specific assessment route, known as the Highly Specialised Technology (HST) programme designed to provide an appropriate framework to determine fair value for the NHS for higher priced medicines that demonstrate breakthrough clinical value.  As has been seen recently, such evaluations are also likely to involve the development of agreements that allow patients access to treatments whilst enabling clinicians and the manufacturer to continue to collect data to support further evaluations of value and benefit after a defined time period.

Biogen believes the most appropriate route for assessment of nusinersen is the HST route which allows for a more flexible approach and optimizes the potential for access within this particular patient population.

Despite many challenges, the last decade has seen great advances in care brought about by the introduction of medicines for rare and ultra-rare conditions, and demonstrates the positive outcome of successful negotiation and agreement between industry and the NHS to bring vital medicines to those patients that need them the most.

Biogen continues to collaborate closely with relevant stakeholders including clinicians, the patient community, NICE and the NHS to ensure that patients and families affected by SMA have access to nusinersen through the most appropriate evaluation methodology and funding routes without unnecessary delays.