How can individualized cell and gene therapies go from ambition to reality?
Each year, there will be 23.6 million new cases of cancer worldwide by 2030, according to the World Health Organization (WHO).
The fight against cancer has made huge strides over the past 30 years. Survival rates have doubled and people are living longer with cancer than ever before. Novel treatments are helping patients delay the worsening of their disease or lower the chance of recurrence, while improving their overall quality of life.
Rising rates of cancer23.6 million
new cases of cancer worldwide in 2030, according to WHO.
However, cancer remains one of the world’s most pressing health care challenges. Because of an aging and growing population worldwide, there will be more patients with cancer seeking treatment. According to WHO, there will be 23.6 million new cases of cancer worldwide in 2030, and it is imperative that scientific and technological advancements outpace the growing burden of this deadly disease.
We continue to make rapid progress with the application of knowledge about mechanisms underlying cancer. This has allowed the evolution of the treatment paradigm for most cancers, from the traditional one-size-fits-all approach to one that is bespoke to a person’s specific disease. The ultimate goals of this individualized therapy are to be able to analyze a patient’s tumor for genetic abnormalities, develop a drug (or combination of drugs) that target those abnormalities and track the response of the tumor using molecular markers.
While targeted therapies that act on specific molecular targets have been the focus of cancer drug development in the past decade, we are witnessing the emergence of individualized treatments that uniquely target each patient’s specific bespoke need. Individualized therapies may take many forms (e.g., adoptive cell immunotherapy, peptide- or mRNA-based vaccines, gene-based). The first few therapies using an adoptive cell immunotherapy approach (CAR-T cells) have already received marketing approval and other therapies targeting specific mutations are showing great promise in early-stage clinical trials.
“Individualized therapies can dramatically improve outcomes in cancer, and alleviate the burden of these catastrophic diseases,” says Pamela Spence, EY Global Health Sciences and Wellness Industry Leader. “This is not just a game changer for cancer care, but has the potential for application in a number of other very debilitating diseases.”
This changing clinical paradigm in cancer medicine from a mass-market “1 for Many” treatment paradigm to an individualized “N of 1” approach has significant implications for the different stakeholders involved, including patients, health care providers, health services companies, insurers/payers, and the pharmaceutical industry. At EY, we have been examining how we can help to transform oncology care (and possibly a cure) and other chronic conditions by enabling highly effective, expedited and safe delivery of individualized therapies at commercial scale.
The operational requirements for individualized care
One of the key requirements to move cancer care to an individualized “N of 1” approach is to have an operating model and supply chain in place that can make certain each therapy is designed, manufactured, delivered and administered to the right patient, at the right time and place, every time. The main challenge beyond “N of 1” manufacturing is the logistics involved, including systems, processes, training and behavior change needed to coordinate the accurate and timely flow of materials and information across an end-to-end value chain that spans many unrelated organizations.
“Truly individualized care requires you have an error-free outcome from beginning to end. It means you need a fail-safe value chain with far better transparency and a real time understanding of what's going on with every individual patient throughout the course of their therapy at any point in time. And since patients are already quite ill, it needs to take as little time as possible,” says Karl Roberts, Managing Director, Strategy & Operations, Transaction Advisory Services, Ernst & Young LLP.
“As a key advisor to major global biotechnology companies, we realized there is a crucial need to help clients overcome the myriad challenges of successfully bringing individualized treatments to market,” says Adlai Goldberg, EY Global Digital, Social and Commercial Innovation Life Sciences Leader. “How are we approaching this challenge? The starting point is asking the right questions.”