46 - Time for change

Medical innovation has always been key to both saving and improving lives worldwide. Collectively, new and emerging therapies are the most significant contributors to increased life expectancy. But with progress comes change – evolving treatments call for adaptive regulatory frameworks to ensure patients can access life-changing medication. Gene therapy is one such example. Advances in gene therapies have disrupted the European regulatory landscape in 2020, with therapies, such as the recent breakthrough treatment for spinal muscular atrophy (SMA), successfully making their way through approval. There are still few one-time (single-dose) gene therapies available on the European market; however, it’s clear that this innovation is here to stay and that healthcare systems will need to make room.

Change is always present, and sometimes it can be accelerated, as seen last year when a global pandemic had life-altering impact on both healthcare systems and personal lives. Medical breakthrough is pushing us forward and, as demonstrated by the approval of COVID-19 vaccines, healthcare systems need to be responsive and adapt to situational needs. Global efforts are underway to speed up access to new medicines by initiatives, such as Project Orbis and Access Consortium, aiming to bring world-leading regulators together to improve the approval process. Although speed is the primary objective, new treatments like gene therapy will continue to challenge all aspects of the process.

Currently, healthcare systems are built to support chronic, lifetime treatments, and are not set up for one-time, transformative therapies. Value is measured over a temporary period, whereby treatments are given and paid for in frequent doses. For a rare paediatric disease such as SMA, for instance, ten-year cumulative chronic treatment costs are an estimated 2-5.6 million euros, but one-time gene therapies can reduce the overall cost of treatment over a patient’s lifetime. It is clear that one-time costs do not conform to traditional payment and reimbursement models. We must mould perspectives to appreciate the value of lifetime benefit with one-time treatments.

…we won’t have changed healthcare systems completely, but if the pandemic has taught us anything, it’s that change is not only possible but necessary.

There are 20-25 gene therapies expected to reach market approval by 2030 and they are expected to account for only 1.30-1.61% of the annual healthcare spend in Europe. Manufacturing is another critical challenge facing the gene therapy industry because processes are long, complex and require multiple biological steps. But, eventually, gene therapy manufacturing will achieve economies of scale.

There are still many diseases that are thought to be untreatable, but developments in gene therapy have laid the groundwork for the new generation of healthcare. The pharmaceutical industry is continuing to innovate and extend into new therapy areas with limited or no treatment options, and to patients who have been living with genetic diseases for years.

I am excited to be at the forefront of this new era in healthcare, changing perceptions, healthcare systems and the lives of patients and families all over the world.