Genetic Engineering & Biotechnology News

AUG 2018

Genetic Engineering & Biotechnology News (GEN) is the world's most widely read biotech publication. It provides the R&D community with critical information on the tools, technologies, and trends that drive the biotech industry.

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24 | AUGUST 2018 | Genetic Engineering & Biotechnology News | Layne Martin Gene therapy has the potential to revolution- ize the treatment of many cancers and rare diseases. Each treatment becomes a personal- ized drug by re-engineering a patient's immune cells or by introducing a new or modified gene into the body to help treat or fight disease. However, gene therapies also present some complex logistical challenges that can poten- tially jeopardize patient safety and limit access if these challenges are not managed well. A comprehensive portfolio of patient sup- port services is needed across the lifecycle of a product, but smaller gene-therapy manu- facturers often lack the resources and scale to be able to implement them. This article discusses the elements of a successful com- mercialization strategy including high-touch patient services, which are key to maximiz- ing access and enhancing patient outcomes. Patient Registry and Data Capture Kymriah™ (tisagenlecleucel) is a chime- ric antigen receptor (CAR) T-cell therapy approved for the treatment of acute lym- phoblastic leukemia (ALL) in pediatric and young adult patients. Each dose is a person- alized treatment created using the patient's T cells, which are collected and sent to a manu- facturing facility. The T cells are genetically modified to include a new gene containing a protein that directs the T cells to target and kill leukemia cells with a specific antigen when they are infused back into the patient. Commercialization Strategies for CAR T-cell therapies illustrate many of the market access challenges. A successful commercialization strategy must carefully consider how to: • Execute complex logistical processes • Maintain the quality and integrity of the product • Minimize the risk of adverse events • Achieve patient access to a very expensive drug • Assist stakeholders with financial risk • Enhance the customer/stakeholder experience • Collect longitudinal data to prove clinical outcomes • Access claims and outcomes data to gain reimbursement in a value-based environment With complex requirements for handling, storage, transportation, and administration, gene therapies require robust logistics and monitoring. Patient data flows through the entire process and is critical to the manufac- turer's ability to track the product across the entire supply chain to get the right medica- tion to the right patient at the right time. Beyond optimizing distribution and logis- tics for gene therapies, McKesson is focused on supporting a commercial strategy that op- timizes patient access and enables physicians, pharmacists, and other healthcare providers to care for patients throughout their treat- ment journey. As a provider of comprehensive patient and physician support programs, the company captures key patient information during the enrollment process. This includes unique identifiers designed to support real- time monitoring of the product throughout the supply chain as well as enable the manu- facturer to deliver the CAR-T cells back to the right patient at the point of care. Concierge Care Coordination As the treatment of cancer becomes more targeted, it also increases in complexity. Pa- tients must navigate multiple care teams, including primary care, oncology, and other specialists; they often have to manage a num- ber of prescriptions; coordinate blood draws; potentially receive additional therapies while waiting for CAR T-cell therapy, get a flu shot to strengthen their immune system; and, with gene therapies, schedule an infusion session. Patients frequently have a limited under- standing of the healthcare system and treat- ment process, and older, very sick patients often have more difficulty. Ongoing education and patient-centric support are critical to helping patients navi- gate this treatment journey and improving patient outcomes. While oncology practices own primary responsibility for patient care, specialty pharmacies with comprehensive Hub services can help manage clinical com- plexities such as educating patients on how to properly take their medications, control- ling side effects, and helping patients deal with the intricacies of their care. Concierge care coordination features clini- cal counseling and one-on-one support to help patients manage their treatment, includ- ing high-touch services, ranging from sched- uling, appointment reminders, and coordi- nating between care teams to ensure that the patient does what they need to do in order to get the best possible care. While some Cen- ters of Excellence may provide this support in person, most patients will receive regular calls and emails from dedicated clinical nurses from a Patient Support Center. Monitor Treatment and Measure Outcomes In addition to providing patient support, clinical nurses also play a critical role in monitoring treatment results and the onset of side effects, such as cytokine release syn- drome (CRS). While CRS demonstrates that CAR-T cells are active in the body, if not monitored properly it can result in serious, potentially fatal side effects. To maintain end-to-end visibility into a patient's journey, it is important to evaluate clinical data points (biomarkers, the location of metastases, disease progression) as well as elements of the patient experience (ad- herence rates, toxicities, functional status). Observational studies using real-world evi- dence (RWE) offer insight into how a drug performs in real-world clinical settings. RWE also provides the opportunity to better un- derstand rare disease types of smaller popu- lations, where clinical trials may be difficult to perform and evidence is sparse. Another important aspect of commercial- ization is understanding how the therapy will be utilized once approved, which is essential for the future approval, pricing, and reim- bursement decisions. An excellent example of the potential of a RWE/registry database is McKesson Specialty Health's large iKnowMed database, which collects clinical and reim- bursement data from approximately 2,200 providers and 650 sites of care across the Unit- ed States. This robust electronic health record (EHR) and economic data delivers real-world evi- dence and observational insights that biophar- ma companies can use to support regulatory approvals for new indications and improved patient care. The ability to analyze real-time, large, detailed data sets allows researchers to discover patterns that may not be visible in smaller sample sizes, helping biopharma companies to conduct effective pre- and post- approval studies. Collection of long-term clinical and economic data after a gene-based therapeutic intervention could: • Confirm the therapeutic durability • Identify any long-term safety issues • Support value-based reimbursement strategies • Support ongoing cost-utility estimations over time • Be used to assess revised economic and clinical burden of illness which could be used to validate therapy cost assumptions High-Touch Services Put the Patient at the Treatment Epicenter Gene therapies are rapidly emerging as an important new treatment option for cancer and other rare diseases. The successful com- mercialization of gene therapies involves pro- actively addressing some complex logistical and market challenges. Yet it is not enough to simply optimize supply chain logistics. Bio- pharma companies must implement patient- centric commercialization strategies that op- timize patient outcomes. These are expensive treatments; however, the value of gene thera- pies warrants high-touch support and ongo- ing contact. Utilizing a comprehensive portfo- lio of high-touch patient support services pro- vides end-to-end visibility of where the patient is along their journey and enables pharmacists and physicians to care for patients throughout their treatment. Layne Martin (accessolutions@mckesson. com) is vice president, supply chain services, McKesson Specialty Health. Website: The ability to analyze real-time, large, detailed data sets allows researchers to discover patterns that may not be visible in smaller sample sizes. Metamorworks/Getty Images Translational Medicine Commercializing Gene Therapies Using High-Touch Services to Enhance Patient Outcomes

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