Recent launches of new cell and gene therapies (CGTs) have yielded mixed results. In some cases, patient outcomes have been promising, with high rates of therapeutic success and transformed lives. In others, the desired therapeutic gains have been undercut by the difficulty of getting the right therapy to the right patients.
Few CGTs have reached the market over the past decade. However, judging from the pipeline of products in Phase III clinical trials, the number of approvals is likely to rise dramatically in the near future. In 2024 alone, up to 21 cell therapy launches and as many as 31 gene therapy launches—including more than 29 adeno-associated virus (AAV) therapies—are expected (Exhibit 1).1
Companies have worked hard to develop these exciting new products.2 It behooves them to launch those products effectively so that the therapies can benefit as many patients as possible while ensuring a sufficient return on manufacturers’ investments—thereby encouraging continued research and development in CGT.
CGTs face steeper challenges at launch than traditional drugs do, potentially limiting their adoption and thus their potential to transform patients’ lives. In an environment in which the population of prospective patients for these therapies is small—and where patients frequently switch payers (at least in the United States)—the current payer system is not well suited to accommodate single-dose therapies for which long-term treatment efficacy, risk–benefit ratios, and safety remain uncertain.
Moreover, CGT patients face a highly complex and costly path to treatment, including long trips to widely spaced healthcare sites and frequent genetic testing and counseling. For healthcare providers, finding and training personnel at new sites requires significant investments of time and the development of new relationships with clinicians and administrators. Furthermore, payers can be reluctant to take on the increased financial risk inherent in treatments with higher one-time costs. Finally, companies themselves face considerable supply chain, manufacturing, and distribution challenges in the effort to make sure just-in-time doses are available when and where they are needed.
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To overcome these obstacles, companies planning for future launches must rethink their go-to-market models, moving away from the models they’ve long used for traditional drug launches. This shift requires adequate preparation to ensure that potential patients, providers, and payers are in place; payment and risk-sharing mechanisms between pharmaceutical companies and providers have been established; and the therapy itself is readily available.
Drawing on our work across several recent CGT launches, as well as on interviews with experts and recent roundtables with experienced launchers, we have identified eight priorities that can significantly improve the likelihood that CGT launches will succeed. These priorities fall into three overarching categories: preparing the market, the product, and the company itself (Exhibit 2).
Preparing the market
The sheer novelty of CGTs means that companies cannot rely on past methods of launching new drugs. Instead, they should prepare patients, caregivers, payers, and healthcare systems for the complexity of new CGTs.
1. Identify appropriate sites of care
Traditional drug launches typically follow a prescriber-based approach, with treatment decisions for prescriptions made in a variety of local and community settings. Given the complexity and preparation needed for the typical CGT, the go-to-market model should shift to centralized sites of care—regional, specialized centers with the ability to make treatment recommendations to the right patients and to scale up CGT delivery to them.
Patients living within 60 miles of sites offering gene therapy are more than twice as likely to receive therapy, according to McKinsey analysis of data from Compile, a data provider for the healthcare industry. Locations should thus be carefully chosen to maximize patient concentration. Ensuring complete care models with clear roles and standardized practices can help sites manage the burden of preparing patients and providing care.
A well-functioning site-of-care model depends on three factors: site willingness, capabilities, and scalability. The first is a function of the attitude of site leaders (including both physicians and administrators) toward CGT and their perspectives on local demand for CGT. The second requires drug companies to carefully track the ability of prospective sites to run their first patients through its system; simple checklists for the infrastructure needed to provide therapy will allow companies to quickly determine a site’s suitability. The third requires that prospective sites have the capacity to meet patient and caregiver demand for CGTs (for more on these requirements, see sidebar “Equipping the front lines”).
2. Support patients and caregivers
Providing CGTs to patients typically requires a highly complex, often multiyear journey, so it is essential that both patients and their caregivers understand the process through well-designed patient services and associated infrastructure. Rather than offering standard information hotlines and patient-facing websites, some companies are providing personalized nurse educator programs and regular one-to-one patient outreach along with documentation to monitor the progress of each therapy and to ensure routine follow-up care. To support patients and caregivers with logistics and travel, some companies are now offering travel concierge services with full reimbursement of costs.
Identifying patients who would benefit most from CGT is also important given that CGTs are typically designed for rare diseases. Real-world evidence (RWE) collected both prior to and after launch—including insurance claims, lab data, diagnostic codes, and claims for other medications that potential patients are taking—can help providers discover likely patients and optimize their care. For example, by using natural-language processing and key words from electronic medical records and insurance claims, companies can identify pockets of potential patients and tailor physician outreach accordingly.
In general, each CGT patient has a unique set of needs. Some will require a full suite of services, while others may need only limited support, as with conventional therapies. The key lies in understanding each patient’s individual needs and delivering personalized support.
3. Offer innovative payment structures
To enable access to and reimbursement for CGTs, companies are experimenting with novel outcomes-based pricing models. These models include the following:
- Outcomes-based payments. The payer covers a fraction of the full price up front and pays the remainder if the therapy achieves prespecified outcomes. One cell therapy company explored this method in the United States, arranging for payment only if a response was achieved after 30 days.
- Outcomes-based rebates. The payer pays the full price of the drug up front but receives a rebate if the drug fails to achieve prespecified outcomes within a predefined period. A US company providing a new gene therapy offered outcomes-based rebates to payers based on both short-term efficacy (30 to 90 days) and long-term durability (30 months).
- Outcomes-based annuity. The payer pays a fixed price, with payments spread over many installments, but only if the drug continues to meet certain prespecified outcomes. A US company launching a new gene therapy used this model to arrange outcomes-based payments for up to five years.
Gene therapy coming of age: Opportunities and challenges to getting ahead
Companies should consider engaging early with payers and sites of care to design the most suitable payment model. Enabling outcomes-based models usually requires involving intermediaries, such as pharmaceutical distributors, that can act as a risk-sharing vehicle, as well as writing a complex set of contracts. In the case of one gene therapy launch, the market access team began holding monthly meetings with payers three years before approval to educate them on the disease and the therapy. By engaging early with payers and providers on payment models, companies can get a head start on assessing the cost–benefit ratio of the CGT they plan to launch.
Preparing the product
A successful CGT launch will also depend on providing full support for the therapy itself by offering evidence-based information to providers and payers and delivering adequate supplies of the therapy.
4. Demonstrate long-term outcomes
RWE can address a range of unique challenges faced by CGT stakeholders. For instance, it can help provide payers with early visibility into the total health system costs related to a particular disease. This is especially important for CGTs because the disease burden is often not quantified for rare subpopulations indicated for CGT. Suitable price anchors and comparisons also may not exist due to a poor or missing standard of care. Some companies are using RWE to quantify the impact of specific diseases across mortality, morbidity, and financial measures, as well as to track long-term outcomes of patients using their therapy, thereby providing payers with the data needed to engage in outcomes-based contracts. Tactically, this can be achieved by identifying the drivers of each outcome measure and then using machine learning to extract the causal relationships between the disease and outcomes.
5. Optimize the supply chain
Given the uncertainties of the CGT supply chain, manufacturing capacity3 should be matched carefully with demand so that patient-specific doses are delivered just in time to sites of care. To do so, companies should carry out dynamic, scenario-based demand forecasting, beginning as early as three years before the actual launch. If done well, a digital supply chain thread can provide track-and-trace data on critical information (such as the location and quality of cells extracted from patients for autologous cell therapies), demonstrate outcomes, provide timely information regarding when caregivers should bring patients in for treatment, and help automate key steps along the value chain.
Preparing the company
Companies should consider preparing themselves for successful CGT launches, reconsidering their go-to-market models across all potential markets, and updating their organizational models for efficiency and scale.
6. Rethink the go-to-market model
Across the patient journey, patients and caregivers face a more complex path with CGT than with traditional therapies. Companies looking to fully support patients along the journey will need to create new CGT-specific roles. For example, appointing CGT account coordinators who can serve as single representatives to site leaders can help build the necessary business relationships between the company and CGT site physicians and administrators. CGT coordinators can also ensure proper synchronization between apheresis—when the cells are extracted from patients—and when the cells are reinfused during therapy. Network coordinators can work with hospital networks and manage referrals to facilitate patient and data flow from one site of care to another as patients move. Across all field roles, tight cross-functional coordination is required to streamline the experience for patients, caregivers, and sites (see sidebar “A team effort”).
7. Enable and promote access across regions
When preparing to launch CGT internationally, companies should consider several areas. First, they need to prioritize markets carefully, considering several factors: the number of potential patients, how patients are treated, how treatments are reimbursed, and whether the infrastructure exists to provide care. This can include the availability of sites equipped to provide therapies and the sophistication of the cold chain for transport of therapies.
Second, companies should determine which model works best for which country: going direct, entering via a partnership, using licensing agreements, or some other arrangement. The model should consider the potential for clustering countries together and the dynamics of extraregional hubs. A regional headquarters with at-scale medical and commercial resources in Singapore, for example, could treat patients in other markets who can travel for treatment. Providers in neighboring countries, such as Malaysia and Thailand, could be supported remotely from the regional headquarters through education or virtual visits from sales representatives.
8. Reorganize for effectiveness and scale
Companies looking to meet the unique needs of CGT patients, caregivers, and payers at scale will likely have to rethink their operating models for maximum efficiency. When commercializing CGT within a larger established business, companies can choose between several models. At one end of the spectrum, companies can create an independent CGT unit for all relevant commercial functions across all markets; at the other end, they can fully integrate the CGT business into the central footprint, with dedicated CGT resources within each function.
Companies that have recently acquired CGT assets have chosen a variety of models depending on circumstances. Some prefer not to integrate the new CGT asset at all to avoid disrupting ongoing efforts to launch it. If integrating the new asset—given that specialized CGT therapies often need distinct capabilities—many prefer to keep specific commercial functions (such as patient engagement, site operations, and market access) independent. In almost all examples, the model evolves over time, and learnings from CGT launches are integrated into parent organizations (see sidebar “Merging capabilities”).
With so many new CGTs in the pipeline and poised for commercialization, the imperative to enable their benefits as widely as possible is as urgent as ever. Successful launches are key to maximizing the benefits of CGTs for patients, for healthcare providers, and for the companies that develop and distribute these life-changing therapies. Ensuring that the market, the product, and the company itself are fully prepared for an effective launch will enable success. The time to resolve the challenges of commercializing CGT therapies is now.
