The field of gene therapy is witnessing unprecedented advancements, marked by innovative treatments and expanding possibilities. As we navigate this dynamic landscape, it is crucial to centre our efforts around the very individuals these therapies aim to benefit—the patients.
We recently attended the Royal Society of Medicine’s (RSM) “Unveiling Gene Therapy: Transforming medical practice through ATMPs” event. In this blog, Dan Gibson, Head of Collaborations at the Cell and Gene Therapy Catapult, reflects on his thoughts following the meeting, exploring how patient-centric approaches are not just a moral imperative but a scientific necessity for the future of gene therapy.
The heart of innovation: Patients first
Gene therapy has come a long way, evolving from conceptual stages to real-world applications that promise to transform lives. Yet, amid this rapid progress, the importance of keeping patients at the forefront cannot be overstated. This importance was made apparent at the start of the meeting, where Professor Uta Griesenbach, Professor of Molecular Medicine, National Heart and Lung Institute, Imperial College London, set the scene for the day by highlighting the potential impact of cell and gene therapies for patients.
Patient-centricity involves more than just involving patients in clinical trials; it encompasses understanding their needs, preferences, and experiences to inform every stage of the therapeutic process. A key aspect of this approach is recognising the diverse and unique challenges faced by patients with rare diseases. These individuals often confront prolonged diagnostic journeys, limited treatment options, and significant social and emotional burdens. By prioritising their voices, we can tailor therapies that not only address clinical symptoms but also enhance quality of life.
Redefining clinical trials
Traditional randomised clinical trials (RCTs) have long been the gold standard for evaluating new treatments. However, for many rare diseases, RCTs are neither practical nor ethical. Patients and advocates argue that single-arm trials, which compare new treatments against historical control data, can offer a viable alternative. This method not only respects the urgency of providing treatment to those in need but also leverages high-quality registry data to ensure robust comparisons. However, it does put a greater dependency on real-world data monitoring processes and or patient registries
Alternatively, there are more novel and intelligent approaches, such as integrating in silico trials utilising simulated control arm data, extrapolated from real world data, and statistically driven computer models. These models can predict how different patient populations might respond to new therapies, providing valuable insights while minimising risks. By combining real-world data with advanced analytics, we can create a more flexible and responsive trial design that better serves patient needs.
Addressing healthcare inequities
Equity in healthcare is a critical issue, particularly in the context of gene therapy. This was emphasised by John James, Chief Executive Officer at the Sickle Cell Society.
Diseases like sickle cell anaemia, predominantly affecting individuals of African descent, have historically seen less research funding and fewer treatment options compared to other conditions. Addressing these disparities requires a multifaceted approach.
Firstly, increasing representation in clinical trials is essential. Diverse participation ensures that the benefits of gene therapies are accessible to all segments of the population. Additionally, equitable investment in research for underfunded diseases can drive innovation and bring much-needed treatments to marginalised communities.
Education and public awareness campaigns also play a vital role. By fostering understanding and trust in gene therapies, we can encourage broader participation in clinical trials and ensure that new treatments are widely adopted.
Navigating the path to approval and beyond
The role of Health Technology Assessment bodies like NICE (National Institute for Health and Care Excellence) is pivotal, as highlighted by Dr Sam Roberts, Chief Executive Officer at NICE, at the event. Their balanced approach to approving therapies, considering both efficacy and cost-effectiveness, ensures that treatments are accessible while maintaining healthcare system financial sustainability. Post-approval, the delivery of gene therapies must transition from clinical trial frameworks to scalable models. This typically requires significant investment in manufacturing and NHS infrastructure.
The current value-based pricing model used by NICE was discussed, Dr Roberts explained, as a pricing strategy, value-based pricing is a fair assessment of health impact, ensuring that every pound spent in the NHS has the same impact, whether that’s into cancer treatment or diabetes. However, some argue that this doesn’t fully account for the impact of cell and gene therapies and suggest that rather, we should perhaps explore an ecosystem wide impact approach, considering overall returns on investment and overall quality adjusted life years (QALYs), as the shared infrastructure, skills and iterative discoveries will continue to provide benefits beyond the impact of a single therapy. A more circular economic approach to cell and gene therapies could be adopted, recognising the investment needed to improve market access to stimulate growth, competition, innovation and economic growth.
Whilst NICE’s value-based pricing appraisals are robust and equitable on impact, there are still opportunities for us, as an industry to improve accessibility by increasing scalability and decreasing the cost of goods. It is no secret that these living drugs are expensive to manufacture and the required infrastructure to maintain them it is expensive. A concerted and collaborative effort to streamline, automate and digitise processes will ultimately help lower the cost of goods, improve negotiation positioning and ultimately lower the price paid.
Embracing a collaborative future
The future of gene therapy is bright, whilst recognising the need for scientific breakthroughs to deliver fully on its promise for patients. However, to fully realise its potential, we must embrace a collaborative, patient-centric approach. By listening to patients, addressing healthcare inequities, and fostering innovation through adaptable clinical trials and robust regulatory frameworks, we can create a future where gene therapies transform lives on a global scale. It was great to hear this all come together in the panel discussion at the end of the RSM event on “Regulating for the future: How do we balance patient safety, efficiency, effectiveness, and cost?”, promoting the importance of working collaboratively to accelerate the advancement of the sector to support more patients.
As we forge ahead, let us remember that the ultimate measure of our success lies in the impact we have on the lives of patients. Their stories, struggles, and triumphs should guide our path, inspiring us to push the boundaries of science and further deliver on the promise of cell and gene therapies.