Gene therapy market access and pricing trends

Gene Therapy Market Access and Pricing Trends


A Dive into the Complex and Expensive World of Gene Therapy Pricing and Access

Zolgensma: “the most expensive drug ever given on the NHS”

At the start of June, a 5-month-old baby boy became the first NHS patient to receive Zolgensma, a ‘life-changing’ gene therapy for the devastating genetic condition spinal muscular atrophy (SMA). At £1.79 million per dose, Zolgensma is the most expensive drug ever given on the NHS. The news broke in March of this year that Zolgensma would be available on the NHS, following a ‘landmark’ deal being brokered during the evaluation consultation stage of the National Institute of Health and Care Excellence (NICE) process. Once the deal– which consists of a confidential simple discount for patients aged 0-6 months and a managed access agreement for patients aged 7-12 months – was agreed, NHS England announced they would fast track access to Zolgensma and not wait for NICE to publish final guidance, given the aggressive nature of SMA making it critical to administer this treatment early. Consequently, the first patient receiving Zolgensma did so days before NICE published its draft guidance on the drug, which recommended its use. Final guidance is expected to be published early July. 

Elsewhere in Europe, Zolgensma has achieved mixed access. Some countries have requested more data. In Germany, Novartis was requested to collect further data on Zolgensma’s use in routine clinical practice through a registry study for an additional benefit assessment; in France, Zolgensma received an ‘improvement of actual benefit’ ASMR rating of III for patients with type I SMA but with the need for the creation of a registry to collect long-term data. Payment-by-results agreements have also been utilised by AIFA, as a condition of reimbursement of Zolgensma in Italy. The Beneluxa Initiative recommended in May both a ‘pay-for-performance’ agreement and a 50% discount. 

A complex access situation

Thus far, it could be concluded that although Zolgensma has had a complicated path to access, HTA agencies have ensured patients can access the drug by using solutions such as financial agreements. This is reflected in the access seen for the other nine advanced therapy medicinal products (ATMPs) approved by the European Medicines Agency (EMA), which consist of both cell and gene therapies. Returning to NICE, eight of these nine ATMPs have final guidance from NICE, of which seven received positive guidance and five utilised either simple discounts or managed access agreements. Some of these ATMPs, such as Luxturna and Strimvelis, like Zolgensma, have also benefitted from NICE’s Highly Specialised Technology (HST) appraisal process and its elevated cost-effectiveness threshold of £100,000 per QALY gained. 

For a situation that is already complex, it is unlikely it will get simpler any time soon. For one thing, the cost of gene therapies could be on the rise. This potential trend is already visible when comparing Zolgensma’s eye-watering price tag with Luxturna’s per patient cost of £613,410 from 2018 (the cost a single dose in each eye). This can also be seen in Bluebird Bio’s Zynteglo, which is currently undergoing NICE appraisal with a price tag of £1.45 million for a one-time infusion. The economic burden of these therapies to healthcare systems is likely to continue to grow. 

New gene therapies in the pipeline could be priced at a premium to even Zolgensma. BioMarin’s failed haemophilia A gene therapy was predicted to reach a price in the range of $2 to $3 million (£1.4-2.1 million) before being rejected by the Food and Drug Administration (FDA). This was not a range set by the company, but rather by The Institute for Clinical and Economic Review (ICER) who concluded that such a range would be cost-effective for the aspirational gene therapy. The high price is justified by offsetting the cost of treating haemophilia over a lifetime with one, curative shot. Haemophilia is a clear example of the potential cost offsets of gene therapy, since it costs approximately $50,000 (£35,000) per year to treat, with haemophilia patients having an average life expectancy of 63 years.1,2 

Even if the growing costs of individual products can be justified, there is another trend that will increase the burden to healthcare systems. Not only are individual prices going up, but the number of gene therapies on the market will also grow. According to a report by America’s Biopharmaceutical Companies, the current pipeline encompasses over 360 cell and gene therapies and is dominated by oncology, with the areas of genetic, eye and blood disorders also well represented.3 One simulation study suggests that 1.09 million patients will be treated with gene therapy from January 2020 to December 2034 in the US alone, with a total expense of $306 billion (£216 billion).4 Justified price tags or not, it is likely that governments and healthcare systems will need to find new ways to rise to this challenge. 

Looking to the future

As seen in haemophilia, the cost of gene therapies is often justified by offsetting lifetime costs with a one-off payment. Nevertheless, one-off payments at the scale we are seeing come with immense risk, particularly where there are uncertainties on the curative ability of these therapies. Further to this, whilst for rare diseases such as SMA the high risk can be acceptable to payers when there are so few patients, two thirds of the pipeline for cell and gene therapies target diseases that are not rare.3 

Current Solutions 

Novel payment models are already in place in some markets. In Germany, an annuity model has already been utilised for Zolgensma, with another model agreed for Zynteglo in the country as well. The Zynteglo model combines annuity payment with risk-sharing, the four subsequent annual payments only made if milestones are reached (i.e. no transfusions for the treatment of transfusion-dependent beta thalassaemia). 

Future Solutions 

A subscription payment model has been proposed. Subscription models involve healthcare systems paying a fixed amount for unlimited access to therapies and is being trialled in antibiotics. The benefit of this to payers is balancing the fluctuation of costs that can be seen from year to year, especially in very rare diseases, but could prove difficult when the eligible populations for these therapies are often less than clear. 

Moving away from the one-drug, one-price model completely was suggested at a 2021 ISPOR panel by the Chief Medical Officer of a US-based health insurance group.5 The nature of gene therapies mean that they could have wildly different effects depending on stage of disease, for example when treating a new-born as compared to an adult who has lived with a genetic disease for decades. To avoid excluding some patients from accessing these life-changing therapies, two different price-tags for the two different ‘values’ could be a solution. 


It seems clear that the pricing and access landscape of gene therapies will be rapidly evolving, and it is very possible we will see the introduction of novel strategies, either those discussed here or even others, in the near future. HTA submissions for gene therapies are going to continue to be complex, and will require care, expertise and critical strategic planning. 

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