Editor’s note: This article is part one of three in a series on bringing cell and gene therapies to market.
Complexity is a common term for professionals in the pharmaceutical industry. The medical advancements that have allowed us to create more targeted, innovative and – hopefully – curative treatments for patients often come with new implications that challenge generally accepted supply chain management and commercialization practices. We saw this when biologics came to market with cold chain requirements and high-touch hub services to overcome prior authorization requirements and manage the patient journey. More recently, innovation has led to developments in cell and gene therapies, which are poised to radically transform how doctors treat and cure diseases, as well as how innovators manufacture and commercialize these therapies. The nature of these therapies and their utilization of living cells or genetic materials create new challenges that redefine the complexity of commercialization. And, that’s because of one specific, highly unique and variant factor in the supply chain: the patient.
Scaling Clinical to Commercial
Cell and gene therapies disrupt the prevailing supply chain model. Traditionally we think of supply chains evolving linearly from clinical trials to commercialization. A manufacturer finds an unmet need, develops a product, conducts trials for safety and efficacy, and once approved the treatment goes on market. While individual components of this model may be complex, there are only marginal dependencies. But with cell and gene therapies, that’s not the case. Decisions cannot be made separately without compromising the overall result.
Logistics partners can help manufacturers optimize access and commercialize their products by creating a highly-customized program that is as unique as that therapy’s patients. First, there is no distinct hand-off from clinical to commercial manufacturing of cell and gene therapies, and decisions made during clinical development influence the future viability of the product to an unprecedented degree. As a result, manufacturers should consider the barriers that will limit patients’ access and customers’ utilization of the product preferably at least 2 years prior to launch. Manufacturers can reduce their risk by accessing the expertise and best practices of a partner who has vast experience designing and executing product and patient specific commercialization solutions within rare disease, oncology, and other highly complex therapeutic areas that require uniquely designed, high-touch, and integrated solutions.
With the magnitude of variables involved in production, distribution and delivery, cell and gene therapy manufacturers must have a supply chain that synchronizes near perfectly. This high-level of interconnectivity can often require a partner who is dedicated to creating efficiencies and ensuring coordination in logistics. As products move through development and approach commercialization, a logistics partner should provide guidance on how to take the processes in place from clinical trials and scale them up to support the product in the commercial market. Even if the patient population isn’t getting much bigger, the variability of where and how a patient presents becomes an important factor.
Because autologous therapies are comprised of living cells with a limited lifespan, logistics expertise will be incredibly important to coordinate and connect services that are designed as a unique program – tailored to perfectly sync with the apheresis center, provider, manufacturer and patient for each cell therapy. Some of the many services that a manufacturer may require include: the fulfillment of kitting at apheresis site, the coordination and tracking of shipments, temperature monitoring and real-time temperature tracking, notification of interruptions or changes, chain of custody tracking, product data monitoring, and advanced cryopreservation and cryoshipping technologies that add an element of flexibility in the supply chain. Manufacturers should also ask logistics partners about their experience with contingency planning to assess their capability to resolving a variety of unexpected situations.
When considering how the supply chain will influence the product’s commercial uptake, a key question to ask at the outset of clinical trials is, “What is the target end state?” Or, once commercialized, where will manufacturing take place? While the complexity of cell and gene products favors minimizing manufacturing sites, it is important for manufacturers to recognize that patients who may benefit from these therapies live across the globe.
For example, Phase I studies for a cell therapy may happen at one location and move to multiple sites for Phase II and III studies, with apheresis happening at one of those sites. When the product moves to commercialization, manufacturing and apheresis may each occur at several sites. Logistics that work in harmony mean apheresis scheduling in a patient hub program links to an available manufacturing slot, which triggers logistics preparations like shipment with temperature-controlled packaging and a time-definite pickup, which should in turn initiate invoicing. When working with logistics providers, all of the points from A to Z can be coordinated together, and manufacturers are able to focus on further clinical innovation or advancing other aspects of commercialization.
With this degree of planning, manufacturers will remove barriers for patients and provider customers, ensuring that the product fits efficiently into their treatment journey and operational workflow. Only by ensuring all barriers are overcome will treatments be able to seamlessly transition from the clinical stage to commercial, allowing as many potential patients as possible to benefit.
The Importance of Data to Overcome Reimbursement Challenges
In order for patients to realize the promising clinical benefits of cell and gene therapies, manufacturers must have access to integrated data and understand what data will be important to share with the stakeholders who will influence the therapy’s utilization. Thankfully, integrated data is a key solution logistics partners can provide manufacturers. Manufacturers should seek partners who can coordinate and integrate data to create “needle to needle” visibility of how the patient and product move through the supply chain. Such visibility will enable manufacturers and their logistics partners to work together to continually optimize that journey because there’s no margin for error in the time-sensitive and temperature-controlled transport and storage of cell therapies. The personalized nature of each therapy means there’s generally no back-up product to ship. A mistake in the supply chain means the manufacturing process must begin all over again, impacting everyone involved in the process, but most of all, it would cost crucial time for the patient .
At the clinical trial stage, aggregated data provides visibility for supply chain requirements and filters down to the provider and patient. This visibility informs requirements for the commercial supply chain. Additionally, data housed at a single source allows for tracking and chain of custody purposes. Most importantly, with any new therapy a single source of data can allow for expedited analysis if any serious adverse events occur.
In order to support value-based coverage and reimbursement decisions, different types of data will be required prior to, and following, launch. When regulatory bodies, pharmacy and therapeutics (P&T), medical policy and/or health technology assessment (HTA) committees evaluate coding, coverage, or reimbursement, they will assess not only the therapy’s clinical differentiation, efficacy and safety data, but also its cost-effectiveness. Therefore, designing a solution that enables collection of the right data will support an effective product value proposition both at launch and as new competitors emerge. In cases of curative therapies, longitudinal patient outcomes data will need to justify its cost effectiveness by demonstrating the therapy’s ability to lower the overall cost of the care.
As mentioned previously, it will be critical for manufacturers to design solutions to minimize the barriers patients and providers are likely to experience as they seek access to these therapies and incorporate them into treatment workflow. Beyond supply chain logistics, patient care will require intensive scheduling and coordination, a more complex patient-centered supply chain will need sophisticated data aggregation, high price points will invite more payer restrictions, and the reimbursement model will be redefined to fit one-time therapies that potentially offer curative treatment. An effective hub model will be a critical tool for these high-cost therapies. From ensuring patients overcome the access barriers that correspond with payers’ high utilization management, to facilitating consistent patient education, reliable scheduling, and data collection; the hub will coordinate services and be a source of data and insights across every stage of the patient’s journey.
Because the complexity of this therapeutic category is so immense, the services to support each product must be extremely customizable, inherently high-touch and tightly integrated. Even breakthrough clinical therapies will require a solid market access strategy and tailored patient services program to ensure payer coverage, provider uptake, a seamless patient experience and the data necessary to continue to improve product performance. Working with a logistics partner that can develop end-to-end solutions where none currently exists will give manufacturers an edge by simplifying some of the complexity in bringing cell and gene therapies to market.
Pete Belden is President for ICS, where he has strategic management and operational responsibility for the leading provider of third-party logistics for healthcare.
Belden previously served in several leadership roles with AndersonBrecon, which was part of AmerisourceBergen from 2003 until its sale to PCI in 2013. At AndersonBrecon, Belden established a track record for driving company growth in each of his executive roles: Vice President, Sales & Marketing, Senior Vice President & Managing Director and Senior Vice President of Commercial Services. Following AndersonBrecon’s sale to PCI, he served as Vice President & General Manager, where he led the two companies’ integration and oversaw operations to ensure business continuity.
Filed Under: Drug Discovery