Jill Maddux, McKesson Life Sciences

With the potential to revolutionize treatment of cancers and many rare diseases, cell and gene therapies (CGTs) have created a new movement in medicine. Two CAR T-cell therapies were approved by FDA in 2017, and thousands of CGTs are in the development and commercialization pipeline. [1] According to Evaluate Pharma, by 2024 the market could reach $36B.

Because these therapies harness the patient’s own cells to engineer a treatment, patients are not only the end users, but the first step in the therapy’s creation. For the first time, patients are at the center of the supply chain. Because the process can be fraught with complexity, McKesson works with biopharma companies to get life-saving medications to patients at the right time, in the right place, in order to drive the best outcomes.

Addressing complex logistics and market challenges

With this innovation comes complex logistical challenges. To avoid limitations to patient access and jeopardized patient safety, biopharma companies must not only optimize supply chain logistics, but implement patient-centric strategies that support the broader commercial life cycle of CGTs. Lack of resources and scale can be a challenge.

To drive the best outcomes, McKesson creates seamless connectivity between commercial support services, like patient access and outcomes-oriented adherence support, third-party logistics (3PL), and advanced analytics and insights:

Access for Patients - helping patients avoid delays in starting treatment, patient on-boarding, prior authorizations, medication benefit investigation and resolution are critical administrative tasks to ensure patients have affordable access to the treatments they need.

Supply Chain Management – maintaining accurate inventory and order accuracy and managing complex logistics is the price of entry in today’s market. Supply chain and commercial strategies must also support the broader commercial life cycle of CGTs. The ability to support patients and physicians through the complexities of choosing, starting and staying on therapy directly correlates to commercial success.

Market Intelligence – leveraging deep clinical electronic health record (EHR) data and robust analytics, McKesson helps biopharma companies identify patient populations, identify patterns of care around the indication, and develop a clinical education and communications plan to engage patients and impact prescribing patterns

Improving patient access and enabling treatment monitoring

While they have the potential to transform the treatment of cancers and other complex diseases, CGTs can come with a high price tag. Concerns over affordability can keep patients from initiating gene therapy. Additionally, many patients experience treatment delays due to complex prior authorization processes and access requirements.

Patients are our number one priority, and we are constantly looking for ways to improve their experience and access to medications. The key to this approach is to enable informed and personalized support to help manage the clinical, financial and emotional challenges associated with disease state-specific treatment. Our portfolio of high-touch patient support services provides end-to-end visibility of a patient’s journey and arms pharmacists, physicians, case managers and care-takers with information to care for patients throughout their treatment.

We also care about the safety of patients. Due to potentially fatal side effects, approximately, 80-85% of CAR T-cell therapies have high-risk safety profiles that require a Risk Evaluation and Mitigation Strategy (REMS) program. McKesson works closely with manufacturers to build and implement robust evaluation and mitigation strategies to satisfy FDA and patient monitoring requirements.

Optimizing the supply chain for patients and manufacturers

With complex needs for handling, storage, transportation and administration, gene therapies require robust logistics and monitoring. A personalized treatment such as Kymriah (tisagenlecleucel), a CAR T-cell therapy for acute lymphoblastic leukemia created using the patient’s own T-cells, requires meticulous logistical oversight. Our technology tracks the product across the entire supply chain -- from cell’s extraction from the patient, through cell engineering, and patient infusion -- tracking patient data through the entire process. Unique identifiers support real-time monitoring of the product throughout the supply chain and enable the manufacturer to deliver the right CAR T-cells back to the right patient at the point of care.

Real-world data is the cornerstone to visibility into the patient journey

We help biopharma companies capture clinical data points and maintain visibility throughout a patient’s journey. McKesson’s iKnowMed EHR database, which collects clinical and reimbursement data from more than 1,950 providers in approximately 145 practices across the U.S., delivers real-world evidence and observational insights that biopharma companies can use to support regulatory approvals for new indications and improved patient care. Collection of long-term clinical and economic data after a gene-based therapeutic intervention can:

support value-based reimbursement strategies

support ongoing cost-utility estimations over time

be used to assess revised economic and clinical burden of illness which in turn could be used to validate therapy cost assumptions.

The US Oncology Network delivers frontline oncology treatment expertise

Our physician partners in The US Oncology Network, a group of over 1,200 doctors who treat more than 1 million patients a year, are studying how to best treat patients with CGTs and are collaborating with biopharma to answer important scientific questions relevant for drug development and improving outcomes.

According to Dr. James Essell, oncologist with Oncology Hematology Care (OHC), “It’s important to understand that community oncology clinics have significant experience and capabilities in complex treatments and are highly capable of administering CAR-T treatment. In fact, many community clinics, including mine in Ohio, have been conducting scientific research, including Phase I-IV clinical trials, for many years. Care for stem-cell and bone-marrow transplant patients, which is similar in complexity to CAR-T treatment, is also effectively provided in the community setting.” [2]

Some clinical trials The US Oncology Network currently leads or participates in include:

* "Can CAR T-cell therapy be done in the office setting?" This study is designed to evaluate if this therapy can be administered safely in the community setting, which could increase access for more patients and reduce costs to the patient and the healthcare system by alleviating the cost of in-patient stays.

* "Expanding CAR T-cell therapy to patients with fewer treatment options." After chemotherapy, patients with relapsed lymphoma normally receive an autologous stem cell transplant, but for various reasons, some patients cannot tolerate transplants. This study is designed to enable the biopharma company to define to the FDA, which patients, who are not eligible for stem cell transplant, may be eligible for CAR-T.

* "Can CAR T-cell therapy replace autologous transplant?" Typically, patients with relapsed diffused large cell lymphoma have an autologous transplant after chemotherapy. This study is designed to evaluate whether using CAR-T therapy as the primary therapy results in a higher cure rate and could be a viable option to replace autologous transplant as the treatment of choice.

McKesson works to expand treatment settings for CGTs

Currently, the two FDA-approved CAR T-cell therapies can only be administered in a hospital setting as an in-patient procedure. This has the potential to limit patient access and increase costs. So, we are working with oncologists who have clinical experience with CGTs to collaborate with the international advocacy organization Alliance for Regenerative Medicine (ARM) and CMS to establish stringent standards to guide the extraction and reinfusion processes that support the administration of cell and gene therapies in community oncology practice settings.

With thousands of therapies in development, CGT represents the next generation of drug treatment for cancer and other rare diseases. CGTs require a new approach to ensure patient access and safety. Not only does this require patient-centric support throughout the treatment journey, it requires a whole new mindset placing the patient at the epicenter of everything from clinical development to supply chain management and ongoing engagement and adherence. This will move CGT from an emerging new therapeutic approach to a critical mechanism for delivering life-saving medications to the right patients at the right time.

Jill Maddux is the director of Cell and Gene Therapy Product Strategy for McKesson Life Sciences, which aligns business assets, technology and innovations through the lens of its life sciences partners. McKesson Life Sciences leverages deep functional and therapeutic expertise, actionable data and insights as well as extensive reach across the healthcare spectrum to solve for its customers’ biggest challenges in connecting patients to and keeping them on their therapies. For more information, visit www.mckesson.com.

1. Q2 2019 Alliance for Regenerative Medicine (ARM) Quarterly Global Regenerative Medicine Sector Report http://alliancerm.org/wp-content/uploads/2019/07/ARM_Q2_2019_Web_FINAL.pdf

2. April 11, 2019, The Columbus Dispatch, “Column: Clinic is safe, convenient setting for cancer therapy,” Dr. James Essell, https://www.dispatch.com/opinion/20190411/column-clinic-is-safe-convenient-setting-for-cancer-therapy

Jill Maddux, McKesson Life Sciences

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