The better the question
How can we harness the potential of cell and gene therapy for all?
Each year, there will be 23.6 million new cases of cancer worldwide by 2030, according to the World Health Organization (WHO).
The International Agency for Research on Cancer estimates there will be 27.5 million new cancer cases and 16.3 million cancer deaths worldwide by 2040, up from 17 million new cancer cases and 9.5 million cancer deaths in 2018. The World Health Organization (WHO) attributes an anticipated rise in cancer cases to people living longer; growing exposure to risk factors, especially in low- and middle-income countries; and an expected increased incidence among younger people.
While we haven’t yet won the war on cancer, we are winning some battles. Over the past 30-plus 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.
On the scientific front, we are witnessing what experts are calling a “golden age” of cancer research and discovery. We know that cancer is not one, but many diseases – more than 200 in fact. And our knowledge and management of those diseases is increasing at a rapid pace.
Some of the most exciting, life-changing possibilities for patients with cancer and other serious illnesses are in cell and gene therapies (CGT). Cell therapy aims to treat disease by restoring or altering certain sets of cells or by using cells to carry a therapy through the body. Gene therapy replaces, inactivates, or introduces genes into cells. Therapies that are considered both cell and gene therapies alter genes in specific types of cells and insert them into the body.
A mother who spends hours each day hooked up to an artificial kidney machine has a chance for a normal life.
A father who’s lost his sight has the chance to see again.
A son dying from cancer has the chance of a cure.
Health outcomes that were once unimaginable are now within reach.
“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.”
Gene therapy has been a work in progress for almost 40 years, but it is now experiencing an acceleration. In 2017, the U.S. Food and Drug Administration (FDA) approved the first gene therapy treatments. While 2019 only saw one approval, the agency expects to see a doubling of new gene therapy applications every year. Scott Gottlieb, the former FDA commissioner, predicted that by the year 2025, the US will be approving between 10 and 20 different gene therapies every year.
However, the evolving cancer treatment paradigm from a mass market (1-for-Many) to an individualized (N of 1) poses significant challenges that will multiply as CGT options and their usage increase.
“These treatments use the patient’s own blood or tissue, which is transformed into a personalized therapy and then reinfused. There is no room for error – the system must deliver the right drug for the right patient at the right time and right place, every time,” says Adlai Goldberg, EY Global Digital, Social and Commercial Innovation Life Sciences Leader.
In addition to being difficult to create and administer, cell and gene therapies are also very expensive and only available to a handful of people. EY examined how it could help transform the treatment of oncological and other serious diseases by enabling highly effective, efficient and safe delivery of individualized therapies at commercial scale.
The operational requirements for individualized care
The only way to make cell and gene therapies a reality at scale is to create an ecosystem of different stakeholders working together and sharing data and information they all trust. This includes patients, health care providers, health services companies, insurers/payers, and the biopharmaceutical industry. No single player – regardless of how smart or well-funded – can do this alone.
“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, EY Managing Director, Strategy & Operations, Strategy and Transactions. “How did EY approach this challenge? By asking the right questions.”
The better the answer
Designing a new operating model and enabling a digital ecosphere
Reimagining the biopharma supply chain, processes, and tools to help provide the right patient with truly individualized cell and gene therapy at the right time and place, every time.
A cross-functional EY team studied each step and connectivity along the CGT value chain. It asked: what issues could arise? What changes are required to address them? The team leveraged EY’s multi-disciplinary capabilities and diverse geographies and experiences to further define and solve this complex problem.
Identifying challenges at every step of the CGT process – by example
- Genetic sampling and sequencing: Core to an individualized treatment may be the collection of a patient’s blood and tumor biopsy, analysis of their genetic makeup, identification of genetic mutations in the tumor and development of a therapy that targets those genetic mutations. However, there is no industry standard for conducting genetic sequencing at the required level of specificity and commercial scale to serve a global market.
The question was: how do we create an environment where that could exist?
- Transportation logistics: Moving not just patient samples but very large amounts of patient data safely and securely is a central challenge to implementing individualized therapies. For instance, crossing national borders means crossing different jurisdictions, which may have different regulations pertaining to the movement of data, tissue and therapeutic products.
The question was: how do we allow end-to-end operations to exist across different legal entities and regulatory guidelines?
- Administration of therapies: Traditionally, the same cancer drugs were administered to thousands of people.
The question was: how can health systems and oncologists handle receiving, storing and administering individualized therapies for every patient?
- Care management: As life expectancy after cancer diagnosis and treatment continues to improve, there is growing recognition of the need to address patients’ health and wellness, emotional and psychosocial needs from the time of diagnosis through treatment and survivorship.
The question was: how can we leverage the large body of patient data to create an end-to-end care management system that engages and empowers patients and improves their overall health and quality of life?
The EY team also conducted market research of more than 250 specialists (practicing oncologists, oncology thought leaders, health care providers, payers and other stakeholders) across multiple geographies (US, France, Germany, Spain), to better understand the current care management paradigm and determine needed behavioral and process change from patient, health care, manufacturing, intermediary and reimbursement standpoints.
Building a fully connected infrastructure of data, capabilities and services with Microsoft
Working with Microsoft, a multi-disciplinary EY team from across multiple geographies and industries developed a private, secure broad services information platform to help enable the reliable delivery of individualized therapies for cancer and other chronic diseases. This is a fully connected data infrastructure, with associated capabilities and services.
We call it PointellisTM.
PointellisTM introduces new industry data capabilities focused on enabling six core functional areas of the digital ecosphere:
- Biometric chain of identity and custody, to track and trace biopsy tissue and blood samples from point of care when the patient first enters the system, all the way through medicine administration.
- Supply chain, to allow for enrollment, scheduling, demand and capacity planning as well as coordination of logistics and delivery.
- Patient engagement, to share appropriate information and keep the patient engaged through the waiting process as well as provide disease management information.
- Care management, to provide oncologists and health care providers who treat and support these critically-ill patients with up-to-date information and decision support based on the wealth of data captured through the platform.
- Health outcomes, to measure more accurately and allow providers, health systems, payers and manufacturers to adapt treatment and maximize clinical benefit.
- Funds allotment, to manage the complexities of paying all who are involved with the successful treatment of a patient.
“Combining EY and Microsoft’s experience was fundamental to developing Pointellis™, which connects the many stakeholders involved in providing individualized treatments,” says Goldberg. “Pointellis™ enables a supply chain as individual as the treatment itself, one for each patient. It safeguards the chain of custody and identity, validating every handoff from patient through to manufacturer and back again, and ensuring patients are treated safely and securely.”
The better the world works
A game changer for cancer treatment … and for chronic diseases
The new Digital Ecosphere expedites a flow of information along the end-to-end value chain, helping patients to receive the care they need.
EY is working with organizations across health, life sciences and other sectors to make cell and gene therapies available at scale. We’re helping them understand the role they play in this new era of health care and what it takes to get there. We’re using our expertise in business model design, information flow and data analytics to help them develop and shape the ecosystems that CGT requires. And we’re continuing to build the information platform needed to make these ecosystems work for patients: Pointellis™.
Our hope is that by providing this trusted platform, we’re liberating everyone else in the ecosystem to focus on what they do best – from developing new science, to delivering outstanding logistics, to keeping patients and their health providers connected and engaged.
“We’re approaching a major inflection point in driving the transformation of oncology care to dramatically improve outcomes, extend and improve the quality of lives and greatly reduce human suffering,” says Roberts. “What a remarkable way to help build a better working world.”
Leaders who embrace this potential now and actively come together won’t just change the lives of people with cancer and other serious diseases. They will put their organizations at the forefront of next generation health.
Please visit PointellisTM to learn more.