The field of gene therapy is at a pivotal moment. With a recent explosion of clinical activity, it is well-poised for further growth — but there are also existing challenges it will need to overcome. As the industry continues to evolve, here are five key trends Novartis Gene Therapies believes will be critical to watch.
Gene therapy will drive transformation for patients, science and industry as it expands
Gene therapies have already transformed patient lives, with four gene therapy products approved in the United States.1 But this is just the beginning.
The field is poised to grow exponentially, with investigational new drug (IND) applications with the FDA for ongoing gene therapy clinical studies, and several new gene therapy indications expected to secure US and EU market approval by 2030.1,2
Further scientific and technological advancements will continue to grow the number of therapies available, expanding their impact to more people and treating more prevalent diseases. The ability to introduce inhibition into the disease pathways through gene therapy, thereby blocking some function that is dysregulated in the course of the disease, will be one breakthrough the industry needs to move beyond genetic diseases and into chronic diseases that impact broader populations.
Global health authorities will push higher standards, but also show greater flexibility
For a time, there was an assumption that companies could file for approval of a gene therapy based on Phase 1, open-label studies. There may still be occasional approvals of this kind, but they will be rarer.
As applications and the potential patient pool grows, there is a move toward regulators increasing requirements across the industry, seeking both broader efficacy data, as well as more robust, long-term safety data even in earlier phases of clinical study. This reflects regulatory authorities’ increasing understanding of the field, and an increased focus on how a gene therapy program fits into the context of the broader treatment landscape.
In parallel, global health authorities are likely to show greater flexibility in the form and type of evidence that is required. In the future, there will be a move toward embracing a wider variety of “fit-for-purpose” evidence types to allow companies to put together robust data packages for review.
These packages may include real-world evidence, novel study designs, greater attention given to patient voice and relevant outcomes, and efficient data generation processes designed to meet the needs of all stakeholders.
Manufacturing will be key to the success of gene therapy growth and innovation
While traditional therapeutics are designed to be easily replicated, gene therapies require an innovative manufacturing process supported by specialized equipment and personnel.
It has become apparent that regulators prefer sponsors have commercial-grade process and product ready at the time of clinical development. While investing in manufacturing is historically difficult without a close-to-market product, an early focus on manufacturing to advance a product is now non-negotiable for a product to be commercially viable and reach patients.
For example, Novartis Gene Therapies developed a manufacturing process where none existed, and as the field advances, the company continues to innovate. It has continually made process improvements — including scaling to larger bioreactors, shortening turnaround time, and other efficiency gains — allowing Novartis Gene Therapies to meet the sizeable need for treatment and unlock potential for new patients.
Stakeholders will work together to encourage health systems to adopt new paradigms that reflect the value gene therapies bring to patients and, more broadly, society
One-time gene therapies provide incredible innovation, but they also challenge a healthcare system built around chronic treatment. Higher one-time upfront payment for gene therapy benefits is a vastly different and new concept.
Innovative financing models will play a significant role in realizing the potential benefits of these therapies. For example, outcomes-based financing models effectively articulate the value of a product and provide an opportunity for payers to recognize the potential of one-time, interventional strategies.
In the future, patient perspective will be at the heart of all value assessments. Patients — particularly those living with rare genetic diseases — may experience significant impacts on health and quality of life, including disability and a mental and emotional toll driven by anxiety due to disease progression, inability to conduct activities of daily living, and feelings of isolation. Yet, these collateral impacts are not always taken into account when evaluating medicines.
The future is likely to incorporate multi-stakeholder dialogue, centered around the patient, for a greater consensus on the determinants of a product’s value.
There will be no one-size-ﬁts-all approach for a therapy’s path to market
The path of each therapy, from development to commercialization, will differ based on factors like patient needs and reimbursement pressures.
The field will learn to adapt to this new reality and collect best practices as each new therapy is brought to market. Already-established platforms will play a vital role moving forward, providing consistency and experience across manufacturing, analytics, biodistribution, and safety.
Novartis Gene Therapies is reimagining medicine to transform the lives of people living with rare genetic diseases. Beginning with the company’s transformative gene therapy for spinal muscular atrophy, Novartis Gene Therapies has played a leading role in the journey the field has taken to date. Visit our website to learn more about how the company is poised to leverage these trends into the future.
1US Food & Drug Administration. FDA continues strong support of innovation in development of gene therapy products. https://www.fda.gov/news-events/press-announcements/fda-continues-strong-support-innovation-development-gene-therapy-products. Accessed June 3, 2021.
2Quinn C, Young C, Thomas J, Trusheim M. Estimating the clinical pipeline of cell and gene therapies and their potential economic impact on the US healthcare system. Value Health. 2019;22(6):621-626.
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