How gene therapies are redefining value and access

One-time gene therapies can positively reshape the lives of people living with rare disease — raising questions about how value should be ascribed to these transformative treatments

Gene therapies are an unprecedented breakthrough area of medicine. The entire field is undergoing a rapid evolution as it matures, and many from academia to industry are responding to the new challenges and opportunities being presented based on this scientific progress. As we start to look beyond the lab and clinic and toward the realm of commercialization, one of the biggest challenges — but also opportunities — that comes into play is access.

“The access vocabulary has made its way into boardrooms,” according to Tay Salimullah, Global Head of Value and Access at Novartis Gene Therapies. “‘Access’ is to some extent the most [frequently] used word in biotech and pharma now – and rightly so!”

This is understandable as gene therapies are an emerging therapeutic category and a whole new era in how we treat and manage disease. This type of paradigm shift has implications not just for patients and healthcare providers, but for societies more broadly and the health systems that underpin them. With this comes a need to properly evaluate gene therapies to ensure global and equitable access.

The new challenges that gene therapies present for payers and health care systems

Payers and health systems are accustomed to the traditional model: a chronic treatment that a patient takes for an extended period of time – perhaps indefinitely – that the system pays for in parallel. The concept of making a one-time payment to cover years or even a lifetime of potential benefits for the patient presents new challenges for payers.

Firstly, payers often face short-term affordability constraints against annual siloed budgets, and they fear that these new therapies will overburden them financially.

Ongoing studies suggest that new reimbursement models may be needed to provide adequate patient access to gene therapies.¹ 

Secondly, because these therapies are so new, uncertainty and concern remain among payers about long-term benefits when they are paying upfront.  Real-world registries are contributing to an increasing understanding of the long-term efficacy of gene therapies, as are ongoing long-term follow-up studies.^2-4 As these data continue to mature, health systems may start to embrace gene therapies and the value they bring to patients, societies and health systems themselves.

Healthcare systems must adapt to help ensure access for patients with rare disease

Through experience in manufacturing and commercializing a gene therapy worldwide, Novartis Gene Therapies has experienced this firsthand. To continue to ensure gene therapies reach the patients who might benefit from them, it is critical to engage with stakeholders in a collaborative way.

This means partnering with advocacy groups in the early stages of research and development to deeply understand the unmet needs with existing treatments, as well as working with local health authorities to understand the potential long-term value these products bring and help tailor solutions to their local markets. Without collaboration and flexibility between industry, regulators and patient communities, innovative solutions will be hard to reach, and patients will suffer. Proactively converging Health Technology Assessment with early regulatory thinking to shape down-stream access is imperative for one-time gene therapies.

It is also critical to ensure that innovative financing models continue to play a significant role in addressing uncertainty and affordability concerns. Outcomes-based financing models and annuity options are examples of these.

To successfully implement these models, the industry will need to continually revisit how these frameworks are executed, evaluate their performance, and make adjustments accordingly.

Finally, health systems need to take a broader perspective on value assessment, increasingly incorporating the most important voice — that of the patients. Systems must also align early with stakeholders on endpoints that matter to patients and caregivers, as well as on evidence that will form part of the payor evaluation to grant coverage and reimbursement.

Patients, particularly those living with rare genetic diseases, experience a significant impact on health and quality of life, including disability, mental and emotional toll driven by anxiety due to disease progression, inability to conduct activities of daily living and feelings of isolation.⁵ In the past, these factors were rarely taken into account when evaluating the value of medicines.

However, the patient perspective has increasingly grown to be the heart of this assessment, allowing for a multi-stakeholder dialogue for a greater consensus on the determinants of a therapy’s value. If this continues, we will see a new health system be built on the cornerstone of value and access.

A glimpse into the future

As more one-time gene therapies come to market, there is reason to believe that health systems will adapt to embrace their value. As the field expands to tackle not only rare diseases, but also more common conditions, partnership between stakeholders will be critical to build innovative and sustainable access solutions. Health systems will also take a broader perspective on value, increasingly incorporating the patient voice, and ensuring that delays to access are minimized and not based on outdated models.

“Moving forward, progressive, value-based solutions that focus on the holistic value a therapy can provide is a reality that the industry is going to have to take the lead on,” says Salimullah. “We must take the business model into the 21st century, moving away from the cumulative and transactional approaches of the past.”

Novartis Gene Therapies is reimagining medicine to transform the lives of people living with rare genetic diseases. Utilizing cutting-edge technology, we are working to turn promising gene therapies into proven treatments. Visit our website to learn more about how we are poised to leverage these trends into the future.

References
¹Quinn C, et al. Value Health. 2019;22(6):621-626.
²Reinhardt B, et al. Blood. 2021;138(15):1304–1316.
³George LA, et al. N Engl J Med. 2021;385:1961-1973
⁴Biousse V, et al. J Neuroopthalmol. 2021;41(3):309-315
⁵Lenderking, WR, et al. J Patient Rep Outcomes. 2021;5(1). 

US-UNB-21-0108 12/2021
©2021 Novartis Gene Therapies, Inc. Bannockburn, IL 60015.