Cell and gene therapies (CGT) are an innovative form of medical treatment that uses cells and genetic material to fight disease. Cancer, cycstic fibrosis, and diabetes (to name just a few) can all be treated by CGT, and as it stands, outcomes are promising. The potential is driving scientists to look for innovations in the field to make medical breakthroughs. The demand for CGT is ripe, and the space is primed for significant growth: BCC Research expects the market to grow at an eye watering CAGR of 33.6% to reach $17.4 billion by 2026. But if the industry wants to fully realize this potential, it must first overcome some huge hurdles.
While it's not the only medical field that incurs some seriously high costs, CGT demands a lot of monetary investment that could stop the treatment becoming widespread. CGTs are personalized for each patient and have an unusually complex manufacturing process. Covering these costs isn’t affordable for most patients – nor companies that are required to fork out huge upfront investments.
Currently, most cell therapies are produced one dose at a time, unlike the traditional ready-to-stock approach. This makes it difficult to estimate the patient pool and increases the complexity in demand and capacity forecasting. Not only this, but the manufacturing requires quality testing of each individual product instead of a whole batch. Subsequently, this is highly labor-intensive, prone to failure and difficult to scale with manual methods.
These overheads hinder the access and affordability of CGT therapies in both developed and developing countries. Let’s give a concrete example: The first two approved CAR-T-cell therapies, Kymriah and Yescarta, are priced at $475,000 and $373,000 respectively per treatment in the US. Unsurprisingly, the inaccessibility is escalated in low- and middle-income countries. According to WHO, comprehensive treatment options for cancer are available in over 90% of high-income countries while it is available to less than 15% of patients in low-income countries. Even in high-income countries such as those in the European Union, there is constant pressure for cost-containment by the government and payors for these therapies. For instance, bluebird, which manufactures Zynteglo, a one-time gene therapy product for beta-thalassemia, costs $1.8 million. The company pulled the drug off the market in Germany due to pricing disagreements with the health authorities.
As a result of these sky-high prices, there’s a growing focus on cost-effective and scalable manufacturing for CGTs, which should hopefully address this issue in the future.