The better the question
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, Strategy and Transactions, 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.”
The better the answer
Designing a new operating model and enabling a digital ecosphere
Re-imagining the biopharma supply chain, processes and tools to help provide the right patient with the truly individualized cell and gene therapies at the right time and place, every time.
A cross-functional EY team studied each step and connectivity along the value chain, asking what issues could arise. What are the required changes to existing supply chains and infrastructure to address them? How could EY leverage its strengths to create an effective solution that would enable delivery of these therapies at scale? To develop our appropriate approach, the team reached into EY’s diverse multi-disciplinary capabilities and geographies to find the right experience and bring the best of the organization to solve this complex problem.
Overcoming representative challenges at every step
- Genetic sampling and sequencing: Core to an individualized treatment may be the collection of a patient’s blood and tumor biopsy, analyzing their genetic makeups, identifying genetic mutations in the tumor, and then developing a therapy that targets specific genetic mutations. However, there is no industry standard that exists today to do genetic sequencing at the required level of specificity and commercial scale to serve a global market, so the key question was how do you 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. Approaches had to be found that allowed end-to-end operations to exist within different legal and regulatory guidelines.
- Administration of the therapies: Today, most cancer drugs are administered to thousands of people, so how would health systems and oncologists deal with the issue of receiving, storing and administering a therapy that is intended only for a single individual patient? The EY team conducted market research of more than 150 specialists (practicing oncologists, oncology thought leaders, health care providers, payers and other stakeholders) across multiple geographies (US, France, Germany, Spain), to compile this data and understand the current paradigm and needed behavioral and process change from a commercial standpoint.
- Care management: As life expectancy after cancer diagnosis and treatment continues to improve, there is growing recognition of the need to address patients’ health, wellness, emotional and psychosocial needs from the time of diagnosis through treatment and survivorship. The key question was how to leverage the large body of data accumulated on the patient to improve the patients’ overall health and quality of life by suggesting appropriate treatments to manage comorbidities, avoiding unnecessary treatment or suggesting appropriate lifestyle modifications.
Furthermore, new tools that engage patients in their own care, such as web programs for symptom monitoring, psychological support and overall health management are needed. These new therapies offer a unique opportunity to directly engage patients and empower them to take better control of their treatment and overall health and the question was how to make that possible in an end-to-end care management system.
“By providing tissue samples and genetic and clinical data, patients are providing invaluable information and contributing to innovation that offers the promise of better treatments for themselves and other patients. We must devise approaches that offer patients greater control over their health data, and help them to make more informed decisions regarding their treatment and overall health,” says Spence.
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 has developed a private, secure information exchange and related end-to-end services to enables the reliable delivery of individualized therapies for cancer and other chronic diseases. This is a fully-connected data infrastructure, with associated capabilities and services, that we call PointellisTM.
- 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.
By enabling each of these core functional areas, PointellisTM aims to build an end-to-end solution to help each client manage its own ecosphere to deliver the individualized cell and gene therapies of the future.
“The partnership with Microsoft provides EY engineering team with access to the latest Azure services and approaches. With these Azure services, EY teams are able to rapidly build, test and deploy the platform and features which bring the exchange of information to key oncology stakeholders in the digital ecosphere”, says Frank Traina, Managing Director, Digital & Emerging Technologies, Data and Analytics, Ernst & Young LLP. The EY team provides clients with support on the decision to buy, partner or build the capabilities to deliver this ecosphere. Joint ventures, alliance management and other partnership structures will be critical to building and connecting the various capabilities and systems needed. New incentives structures and performance metrics are also needed to keep the ecosphere operationally and economically viable for all the stakeholders.
“Combining EY and Microsoft’s experience was fundamental to developing PointellisTM, which connects the many stakeholders involved in providing individualized treatments,” says Adlai Goldberg, EY Global Digital, Social and Commercial Innovation Life Sciences Leader. “PointellisTM enables a supply chain as bespoke 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, 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.
By enabling this digital ecosphere, EY and Microsoft are laying the foundation that will bring the potential of individualized treatments to a global market.
And this won’t be the end of the road. To implement individualized care properly there needs to be transformation across the industry and the health care ecosystem.
The different stakeholders involved in providing care need to rethink their operating model and develop an entirely new value chain. One that requires timely, error-free coordination, communication and hand-offs between often-disconnected health care stakeholders; the secure sharing of unprecedented amounts of medical and genetic data; and the efficient transportation of sensitive, patient-specific blood samples, biopsy tissue and the resulting individualized therapies.
“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.”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.
Individualized therapies can dramatically improve patient outcomes and help alleviate the burden of cancer and other chronic diseases. EY and Microsoft are laying the foundation to enable these revolutionary therapies reach patients in need and transform oncology care and other chronic conditions worldwide. Please visit PointellisTM to learn more.