Cell and gene therapies (CGTs) are positioned currently as last-chance, âmiracleâ cures for patients who have severe illnesses. Such promises require innovation. Despite the cutting-edge science and significant investment that goes into CGT development, fundamental challenges remain, including patient access. The highly personalized nature of autologous-therapy development presents myriad logistical, financial, and manufacturing challenges to ensuring global access to treatment. Understanding a patientâs journey to treatment is vitally important to achieving that goal.
Barriers to Cell and Gene Therapy Access
Awareness: At Terumo Blood and Cell Technologies, weâve learned that educating patients and clinicians about available technologies and therapeutic approaches â such as treatment of sickle cell disease using automated red blood cell exchange â can broaden access to treatment. Even if a cure existed for the condition, many sickle cell patients in the United States would go untreated simply because they and their physicians did not know about available options. The CGT industry still has much work to do in this respect, which is why my company has increased its efforts not only to promote patient awareness, but also to inform clinicians about how different treatments raise distinct benefits for quality of life and health economics.
Geography: Wide disparities compromise patientsâ treatment access across different regions. The United States has enough medical infrastructure to build broad awareness about many conditions. However, in regions where such infrastructure is less developed, the problem of patient unfamiliarity with treatment options is amplified.
In Africa, where over a third of the population (>360 million people) has the sickle cell trait, the importance of patient awareness becomes more obvious than it might in other regions. Without widespread education, severely ill patients are unlikely to know about their treatment options, including new CGTs.
Cost remains particularly prohibitive to CGT availability. The highly personalized nature of autologous-therapy development exacts high production costs that are passed down to patients and ultimately can bar many people from receiving treatment. Those costs need to be reduced through development and implementation of process and manufacturing efficiencies.
Developing Global Solutions
The biopharmaceutical industry is working to enhance CGT manufacture and delivery. Automation is the clearest solution. Cutting out manual work and streamlining manufacturing processes could increase repeatability and scalability, facilitating transfer of what can be highly complex CGT systems to new markets while significantly cutting costs. To that end, my company is contributing its full range of automation technologies and scientific expertise to the recently formed Cell and Gene Therapy Catapult Process Analytical Technology (PAT) Consortium, which was formed in June 2021 to accelerate technology development and minimize CGT manufacturing costs (1).
At Terumo BCT, we also believe that creating both globally interconnected and local treatment ecosystems can increase cell and gene therapy access worldwide. CGT manufacturers and technology providers need to understand the regions in which their patients live, then structure their operations around those patientsâ needs. Terumo intends to lead the industry in this respect. Whereas other companies might concentrate on developing the next âwidget,â device or feature for their technology offerings, we consider a patient-first approach to be vital to increasing cell and gene therapy access. Beginning with patient needs and then working âoutwardâ yields a far better understanding of how to help than a standard operational approach can.
To expand patient access, my company is investing in developing regions. Many biotechnology vendors still do not operate in Africa, for example. In such places, we have invested in long-term infrastructure. Serving those patients today increases both health and business tomorrow.
Terumo aims to create fertile ground for future, localized development. Opportunities for collaboration within the CGT industry certainly will yield returns on investment. More important, working together will establish the infrastructure needed to manufacture lifesaving therapies locally and the framework required to make treatments available to the people who need them.
1 Cell and Gene Therapy Catapult Brings Together Over 20 Organisations with the Aim to Accelerate Technology Development and Potentially Lower Cost in Cell and Gene Therapy Manufacturing. Cell and Gene Therapy Catapult: London, UK,
8 June 2021; https://ct.catapult.org.uk/news-media/general-news/press-release-cell-and-gene-therapy-catapult-brings-together-over-20.
Antoinette Gawin is president and chief executive officer of Terumo Blood and Cell Technologies, 10811 West Collins Avenue, Lakewood, CO 80215; 1-303-231-4357; https://www.terumobct.com.