By Amy DuRoss, Vineti, and Chris Zant and Sanjay Srivastava, Ph.D., Deloitte
In today’s breakthrough medicines, personalization changes everything. Patients are used to receiving medications. Now, in the era of cell and gene therapies, patients are actually providing the key components to create them.
When patients are the source of cells and other “raw material” needed to manufacture these breakthrough living drugs, the entire patient journey is profoundly transformed. Patients and their clinicians become part of the manufacturing process and the supply chain. Patient onboarding and reimbursement processes take on new complexities, and long-term monitoring requirements make the journey a lengthy, involved commitment.
Amid the very well-founded excitement about the science of cell and gene therapies, it is easy to underestimate the challenges of managing what has become medicine’s most complex patient journey. The initial regulatory approvals to commercialize the first cell and gene therapies fundamentally changed the landscape for biopharma. The historic acts singlehandedly created an entirely new supply chain for the industry.
The nature of these therapies definitively reshapes the patient experience. Now a key player in the supply chain, the patient begins their journey early in the onboarding and cell collection processes. They play a role in the complex logistical and manufacturing processes leading up to treatment, and then have to participate in extensive post-treatment monitoring, sometimes for as long as a decade or more. Plus, with a single-dose price tag in the hundreds of thousands of dollars, the pre-authorization, order-to-cash, and reimbursement processes face new requirements and procedures.
For gene and cell treatments to be marketable and benefit those most in need, the biopharmaceutical industry must simplify the patient experience in a way that enables scalability, while minimizing risk across the supply chain. New logistical demands, such as creating, delivering, and tracking multiple personalized therapeutic doses for a single patient, will also emerge as the science continues to advance.
To expand the availability of these therapies in the market, biopharma companies have to address a number of different challenges. Here are four strategies to help align the journey and move personalized therapies into mainstream medicine.
1. Define a robust informatics capability.
There is an increasing amount of data being generated as more patients undergo CAR T-cell treatments in both clinical and commercial settings. Implementing a robust informatics strategy to uncover value in this information is key to turning cell and gene therapy into a second or first line treatment.
Informatics capabilities help you collate and correlate large data sets obtained from disparate sources — EMR data, manufacturing data, clinical outcomes data, etc. — across the entire patient journey from diagnosis to post-therapy outcomes. The convergence of this data can help you identify the combination of factors that lead to successful treatment. Biomarker identification, specifically, can help predict patient cohorts with a higher likelihood for successful treatment, and those more susceptible to adverse events, to improve the overall viability for all patients in the marketplace.
The right strategy will demonstrate treatment viability and inform key business decisions to enhance market access, streamline internal processes, improve reporting accuracy and consistency, and demonstrate overall process competencies. By improving the entire process, you can reduce the cost, increasing availability and coverage.
2. Prioritize collaboration at every stage.
Cell and gene therapies are highly collaborative. As a patient’s cells become a therapy, there is continuous interaction between patient care teams, logistics and manufacturing facilities, insurance companies, and other stakeholders throughout the entire procedure to create transparency and ensure safety. Therefore, building effective communication into your strategy is essential.
There are some key points to consider when laying the groundwork for your strategy. Biopharmaceutical developers are most effective when they understand, from the start, that the therapeutic ecosystem is broad and goes beyond the four walls of manufacturing. It is impossible to separate the patient, the physician, and the manufacturer. All stakeholders are living within the same ecosystem and sharing the same resources – even though ecosystem is varied and distributed. It is essential to develop an approach that respects the healthcare provider’s world — versus one that disrupts it — and integrates with the other systems they use to care for their patients.
When it comes to how you communicate, consider that each player in the ecosystem has a different understanding of the complex science behind individualized therapies. Transparency will go a long way towards creating an experience that is as reliable and reassuring as possible. Clear explanations about clinical benefits offered at multiple points in the patient journey, for example, can help motivate patient and provider engagement.
Cell and gene therapy is already a crowded field, destined to become only busier. Without effective collaboration, you will create barriers to the clinical process. If you enable effective collaboration, you can make swifter progress, which is essential amid today’s accelerated regulatory environments for these promising new therapies.
3. Keep it simple for health care providers.
The collaborative, high-regulated nature of the cell and gene therapy patient journey requires new systems for healthcare providers to deliver cell and gene therapies. Yet, like everything else in health care, adding too many new processes or systems — or throwing too many at healthcare providers at once — can trigger resistance. The ongoing demands of patient care already take up much of a clinician’s day; healthcare providers most likely do not have time to convince their hospital to implement big IT or infrastructure changes.
To support collaboration, make the process easy for healthcare providers by working within their ecosystem. Help them balance the demands coming from the patient’s overall treatment program, their other patients, and their schedule — instead of prioritizing your schedule or process. Your therapy may be a breakthrough, but it probably isn’t the only option in play.
One way to do this is to create simple, easy-to-use systems and processes that enable transparency and feedback. Consider some of the aspects of the patient journey that are of most interest to healthcare providers:
The significance of transparency and real-time data
The critical importance of scheduling the cell and gene therapy patient journey alongside the rest of the patient’s care
Chain of identity / chain of custody / chain of condition
Stages of the patient journey:
Has the patient gone in for their cell collection?
What is going on with the collection?
What is going on with the manufacturing?
When will the finished product be back for infusion?
Healthcare providers need this information to be readily available, up-to-date, and accurate. Again, it is about simplicity. A smooth, easy process for ordering, collection, scheduling, and transparency will help drive healthcare provider adoption and loyalty in cell and gene therapies. For critically-ill patients, a difference of day or two in treatment can make a major difference. Make this life-changing process as simple as possible for healthcare providers.
4. Proactively use post-therapy outcomes data.
CAR T therapies have demonstrated the ability to save lives when other treatments have not. Yet, they are in their infancy and scientists have a limited understanding into the causes of long-term recurrences or other abnormalities, triggering skepticism in the marketplace.
The strategic use of clinical outcomes data creates an opportunity to demonstrate success and improve delivery of this emerging therapy. The more we can learn about the long-term impact and apply that knowledge to the patient journey, specifically the recruitment, manufacturing, and treatment process, the sooner we can move it up to a second or first line treatment. Biopharma companies need to demonstrate proof of success, both with regards to the treatment and patient selection, to convince insurance companies to cover the therapies.
CAR T treatments began clinical trials as early as 2013, providing five years of data that is currently being used in specific case studies to predict the occurrence of adverse events and response to therapies. Proactive application of outcomes data will play a significant role in the development of cell and gene therapies and demonstrating its clinical efficacy, and this is bound to get easier with advancements in data-gathering technologies and our understanding of the therapies’ success.
Emerging tools will help drive deep analytics to help extract critical insights and promote data-driven clinical and commercial development of future CAR T therapies. It would be prudent to embrace such trends and learnings from the real world and realize the full potential of clinical data in accelerating innovation and gaining the opportunity to bring an even better therapy to patients.
About The Authors:
Amy DuRoss is CEO and cofounder of Vineti Solutions. Before cofounding Vineti, she focused on healthcare new business creation for GE Ventures/healthymagination. Prior to GE, DuRoss was chief business Officer at Navigenics, a genomics company sold to Life Technologies in 2012. She was cofounder and executive director of Proposition 71, California’s $3B stem cell research initiative passed in 2004, as well as chief of staff at the resulting state grant oversight agency. DuRoss holds an MBA and MA/BA in English from Stanford University. She was named a 2016 Health Innovator Fellow by the Aspen Institute.
Christopher Zant is a principal at Deloitte Consulting LLP and chief digital officer of ConvergeHEALTH. As Deloitte’s digital life sciences leader, helps companies transform their capabilities through effective and compliant delivery of digital strategy, process, and technology. With more than 24 years of life sciences consulting experience, Zant understands the business requirements and enablers that help optimize the patient and health care practitioner experience while streamlining operations and improving financial performance. He is passionate about empowering patients to manage their journey through digital technology and sponsors Deloitte’s Patient Connect platform, which helps life sciences companies leverage digital capabilities to develop and strengthen bonds with patients, caregivers, and health care practitioners.
Sanjay Srivastava, Ph.D., is a senior manager and the lead for Deloitte's NextGen consulting practice, helping biopharma and biotech companies define and implement fundamentally new and transformative capabilities to develop, produce, and commercialize next-generation therapies. His project work centers around defining and implementing innovative R&D and commercial strategies to design value-added activities that optimize operations and deliver therapies to the market. Srivastava earned his Ph.D. in bio-organic chemistry from the University of Maryland with post-doctoral training at Johns Hopkins University and Cornell University with a focus on neurodegenerative diseases, such as Parkinson’s Disease.