ISPOR 2021 Issue Panel: Can We All Afford A Cure? The Greatest Challenges Now Facing Payers

“How can we afford gene therapies?” is not a new question. Back in 2019, we saw the spinal muscular atrophy gene therapy, Zolgensma, priced at a record $2.125 million (1). As medicines priced in the million-dollar range are unlike anything the healthcare industry has seen before, different payment mechanisms for gene therapies have been frequently debated over the last few years.

I don’t yet believe we can truly answer the question “how can we afford gene therapies?”. However, 2021 brings us to an exciting point in time within the gene therapies space. As a number of these transformative treatments have now been on the market for several years, we can look to understand how payers have responded to funding and access challenges, and what the remaining, enduring challenges that must be tackled are.

I was therefore delighted to moderate an issue panel on this topic at virtual ISPOR 2021! Below is a summary of some of the key themes that were discussed. 

How have gene therapies been funded to date?

• Michael commented that, generally speaking, payers have not found funding gene therapies to be overly problematic, as available therapies have typically been indicated for a very small number of patients with rare diseases – in other words, these therapies have not broken the bank. However, with the FDA expected to be approving 10–20 cell and gene therapies every year by 2030, the current financing system will struggle to continue to provide access to all patients   
• Michael also noted that, whilst some of the existing risk-based agreements are good, further improvements are needed. More specific outcome measures that are able to measure the benefit of a therapy need to be incorporated into agreements, especially when the price hinges on benefit. At a minimum, payers would want outcome measures used to gain FDA approval to be included in contracts

What changes are needed to improve access to gene therapies in the future?   

• Michael argued that manufacturers must be willing to assume more risk than payers in risk-based agreements, where there is uncertainty around the long-term effectiveness of therapies
• Ramesh explained that long-term follow-up data is therefore extremely important, and patient organisations should work closely with manufacturers to develop digital tools that can aid the collection of long-term data, whilst reducing the burden on patients
• Payers and manufacturers must consider entering into agreements where therapies are priced differently for clearly-defined subpopulations i.e. not just a ‘fixed-price’ treatment. For example, the benefits of a gene therapy for a patient in an advanced stage of their disease may be very different to a newly-diagnosed patient. Manufacturers must collaborate with key opinion leaders early on in the clinical development process to design and validate disease severity scales, as they can be of paramount importance for future pricing negotiations
• Ramesh highlighted that manufacturers must continue to optimise data generation and collection methods, to aid pricing discussions and minimse risk for both themselves and payers:

1. Consider from the outset how durability of effect will be measured (for Zolgensma, it was important to demonstrate how patients could maintain a sitting position in the long-term, beyond the clinical trial setting)
2. Where pivotal trials are single arm studies, ensure relevant data has also been collected in a natural history study. This comparative data allows for effectiveness to be measured, and can help inform risk-based agreements
3. Consider what data needs to be collected to demonstrate a neutral or favourable effect on budget impact. Whilst the US does not have a national healthcare budget that is used to reimburse medicines, individual states have limited healthcare budgets that affect public insurance programs

What are potential roadblocks that may affect access to gene therapies?

• Health insurers are unable to budget for such expensive therapies without extensive planning. Discussions between insurers and manufacturers that happen too close to FDA approval may delay access, therefore early engagement between stakeholders is vital
• Debra noted that, whilst it is now commonplace for manufacturers and patient organisations to collaborate on clinical development programs, payers and patient organisations must also join forces to educate each other, which will aid access for patients
• Portability issues when patients switch healthcare providers, and complexities stemming from fully-insured versus self-insured healthcare plans are still unresolved issues. Whilst there are some positive discussions taking place to address these issues, it is difficult to get the relevant stakeholders ‘round the table’ until the issue is pressing. If more can be done to engage stakeholders to tackle these challenges sooner, this could minimise access issues

The issue panel made clear that whilst progress continues to be made surrounding affordability and access to gene therapies, it is important for payers who have provided access to gene therapies to share their experiences with others. It is only through collaboration and engagement that we will ensure access for all.  

References

Institute for Clinical and Economic Review. 2021. ICER Comments on the FDA Approval of Zolgensma for the Treatment of Spinal Muscular Atrophy. Available at: https://icer.org/news-insights/press-releases/icer_comment_on_zolgensma_approval/. Last accessed 29.06.2

Kate Hanman is a member of the Rare Diseases Division at Costello Medical, providing scientific support to the healthcare industry in the analysis, interpretation and communication of clinical and health economic data. Due to the growing demand across an increasing range of service offerings and geographies, Costello Medical has grown organically since its foundation in 2008 to a team of over 200 based in Cambridge, London and Manchester in the UK and internationally in Singapore, China and the US. Alongside our evolving technical and creative capabilities, we remain committed to our core values of high-quality scientific work coupled with exceptional customer service at competitive and transparent prices. Our talented team has experience with a variety of leading pharmaceutical and device companies across an extensive range of therapy areas and geographies. For more information on our services, please visit our website. If you would like any further information on the article above, please do not hesitate to contact Kate.