With Beneluxa Leading the Way, Will International HTA Collaboration Become the Norm?

By Ines Dieringer, Associate Consultant at Global Pricing Innovations

Belgium, the Netherlands, and Ireland are aiming to undertake a joint Health Technology Assessment (HTA) through the Beneluxa agreement of Zolgensma, Novartis’ gene therapy medication to treat spinal muscular atrophy (SMA)1.

Zolgensma is a gene therapy that has been in the pharma headlines for several years now, including speculations over its possible price, which is currently estimated to be $2.1 million for the USA. While this is quite a high-cost treatment, it is unsurprising as Zolgensma has the potential to cure SMA after just one dose. Zolgensma also has the potential to target pre- symptomatic patients, which would lead to a significant increase in patient benefit and quality of life, especially when compared to Spinraza’s regular chronic treatment2.

Although there are still uncertainties over Zolgensma’s long-term effects, especially the potential need to re-administer treatment at a later stage, the current clinical data has been promising, which justifies the high price-tag2.

The joint HTA decision by Belgium, the Netherlands and Ireland is in response to Zolgensma’s reimbursement application, which is conditionally approved in Europe to treat infants and young children under 21kg with SMA, while more data on it safety is collected3. Other HTA agencies, such as NICE, have also started appraising Zolgensma. In the UK, Zolgensma will be assessed for its potential treatment of SMA as of June 8th 20204. While this therapy has been approved, Novartis is yet to set a price for treatment in Europe. Novartis is however allowing for national reimbursement systems to choose from a variety of customizable reimbursement options, including2:

  • Retroactive rebates to ensure early access costs are aligned with negotiated prices after respective assessment processes
  • Deferred payments and installment options for reimbursement bodies to minimize budget impact during early access
  • Outcomes-based rebates following HTA assessments can be applied to treated patients during early access
  • Training for institutions on administration and follow-up care
  • Access to RESTORE, a registry of patients with SMA5

The Beneluxa agreement allows for the evaluation of treatments that are considered incredibly effective but very expensive. The agreement arose to provide patients with significant increases in their quality of life. Through Beneluxa, Belgium, the Netherlands, Luxembourg, Austria, and Ireland are able to collaborate extensively. Cooperative policies include: sharing resources and policies on best practices, improving transparency on pricing, and increasing ‘bargaining power’ with pharmaceutical companies to reach an equitable price for these emerging treatments6. The member states of this agreement, through their increased bargaining power, aim to lower the price of Zolgensma to ensure that the demand can be met7.

While the treatment is promising, some member states have argued that there may be uncertainties regarding Zolgensma’s long-term effects. As such, Beneluxa has welcomed an open dialogue with Novartis concerning Zolgensma – an invitation which Novartis has accepted as of May 19th, 2020. Thus far, the open dialogue has proved effective; HTA agencies from member states will now be aligning their timing and content of HTA procedures to continue with the market access process of this treatment7.

The Beneluxa agreement is not unique in its aims. The European Network for Health Technology Assessment (EUnetHTA) was established in 2006 to create a sustainable and effective network for HTA collaboration across Europe. Its key purpose is to ensure that appraisal processes are streamlined by sharing resources and reducing the duplication of work, while still allowing for individual countries to make their own decision8.

EUnetHTA is at its infancy and significant challenges remain. In particular, friction arising from methodological issues which require further research attention from EUnetHTA’s member states9. As such, individual country decisions such as Belenuxa are taking the lead in advance of an EU wide approach.

Other agreements, such as FINOSE, a collaborative effort between Finland, Norway, and Sweden, also try to foster collaboration in order to increase the efficiency and equity of emerging therapies10. FINOSE recently agreed to negotiate a price for Zynteglo, a gene therapy for beta thalassaemia11.

In addition, there are also other examples of cross-border cooperation where therapies are approved in certain EU countries, but patients have to travel to another country to have the treatment administered. This is the case with Strimvelis, a gene therapy for the treatment of adenosine deaminase deficiency, which is approved for use in the UK but only has one approved manufacturing plant in Milan, Italy. Due to its six-hour shelf life, patients can only receive this treatment in Italy, even though Strimvelis is approved in the UK12.

Clearly, the development of highly effective but expensive treatments has led to an increase in cross-border cooperation. As high-cost pharmaceutical sectors, such as gene therapy and wider personalized medicine, continue to expand and become more common-place, it will be interesting to see whether international collaboration trend discussed above will continue to develop.

The article above naturally raises some questions:

  • How will this impact the price of Zolgensma given the increased purchasing power from reimbursement agencies?
  • Are these cross-border collaborations something that can only be done across healthcare markets that operate within a similar space?
  • What can we expect from the interplay between universalized HTA decisions and non-universalized HTA agencies? How will they affect payer hurdles?

At GPI, we believe that the monitoring of these trends is key to understanding the interplay of market forces in the procurement of emerging pharmaceutical products. As such GPI has developed services, such as the Access Module on its GPI pulse platform and personalised HTA Trackers, to provide customers with the most up-to-date information on HTA and Access intelligence.

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(1) https://www.apmhealtheurope.com/story.php?objet=68846&idmail=.O.oQ4xQ03Sib7LrDKvHBQowPolyG_7c4N_hHJ7SPNZ6oggXt6Bar9Na76G-kum1kDOv6Ofqoh97h-Y228lPJjQ07BqnjeUHDUdBNFdTzB2SeASlD8CfvQFrTuJAHA9Ht7f3IN2QReXoCpLJsyMYg6jeokWWG3es-PBiHbWz32vXe-64amlt4kLV-cw6RwjFGO8pA69-OggnxrFl71DvlG3ctvKvnO-s2K0lKA6Qb6fCTtJgW528mStiYQ9frA6R5HU

(2) https://pharmaphorum.com/news/novartis-reimbursement-options-as-eu-approves-ultra-pricey-sma-drug/

(3) https://www.ema.europa.eu/en/documents/product-information/zolgensma-epar-product-information_en.pdf

(4) https://www.treatsma.uk/2019/06/nice-starts-appraising-zolgensma-to-treat-spinal-muscular-atrophy/

(5) https://www.novartis.com/news/media-releases/avexis-receives-ec-approval-and-activates-%22day-one%22-access-program-zolgensma-only-gene-therapy-spinal-muscular-atrophy-sma

(6) https://beneluxa.org/collaboration

(7) https://beneluxa.org/news3

(8) https://eunethta.eu/eunethta-project-2006-2008/

(9) https://www.eunethta.eu/scientific-needs-of-hta-methodological-issues-requiring-further-research-attention/

(10) https://www.tlv.se/in-english/international-collaboration/finose—a-nordic-cooperation.html

(11) https://www.healthtalk.no/alle-artikler/de-nordiske-landene-skal-forhandle-pris-p%C3%A5-genterapien-zynteglo/

(12) https://www.nice.org.uk/guidance/hst7/chapter/3-The-technology