Key Considerations For Successful Launch Of Cell & Gene Therapies
By Sanobar Syed, BeiGene
Continuing advances in cell and gene therapy (CGT) are transforming how biopharma companies treat and potentially cure certain diseases. Recognizing CGT’s potential, 16 of the world’s largest (by revenue) 20 biopharma companies now have CGT assets in their product portfolios. Yet most companies are moving cautiously — only two of the top 20 have CGT assets making up more than 20 percent of their pipelines. They are still considering whether, when, and how to reposition their portfolios. In the meantime, biotech companies remain leaders in CGT innovation.
As this sector grows further, there is hope that more and more complex therapies will enter the market, leading to a consequent increase in the number of treated patients. The data volume is increasing significantly, and that is where the concept of Big Data analytics will kick in.
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.
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.
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.
One of the most important points is to align the company top down and bottom up in the same “thinking” fabric. This is imperative in the pharmaceutical world but more so in CGT where the stakes are quite high, and speed is a necessity. This will not only make a cohesive point but also ensure that the forecasting, insight, and strategy align with the overall objectives set by the company.
Strategic Market Access
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.
Rethink the go-to-market model to optimize support for patients, healthcare providers, sites of care, and payers. 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.
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.
Consider your company’s geographic presence and its vision to expand or operate locally. For example, if your company is in the U.K., your strategy would prioritize the U.K.’s neighboring countries to fulfill the speed and access. If your company is in the U.S., Canada or Brazil might be a priority for expansion. Brazil is your top choice in Latin America if your company is looking to establish itself in that region.
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 extra-regional hubs. Two notable European clusters are Spain and Portugal (as one cluster) and Germany and Austria (as another cluster). Not only they are geographically close but also their healthcare authorities and systems mimic each other very closely. They are used as a “reference” of each other.
Making these decisions is very important as it leads to valuable data input for various forecasting, analytical, and strategic functions.
Payer Costs
While the potential benefits of CGT to patients seem clear, the high costs, concentrated over a short period of time, are hard for healthcare systems to carry. A single dose of a curative treatment can cost millions of dollars, and cash-strapped authorities may have to weigh the benefits of relatively low-cost treatments for common conditions against high-cost treatments for rare ones.
The payer cost dilemma will likely become more acute as more centers specializing in the delivery of CGTs are established and many new CGT treatments emerge. Yet payers face an additional cost challenge as many have the ethical ambition, and often legal obligation, to make treatments equally available to all patients without discrimination, which could stretch budgets still further.
In contrast, the fragmented market can make reimbursement a complex, lengthy, and costly process, as CGT companies have to negotiate terms with every country and, in some instances, with regional authorities within a country, too (e.g., in Spain and Italy).
Manufacturing, Supply Chain, And Inventory
The manufacture and logistics of CGTs are highly complex, and manufacturing capacity is a major limiting factor to treating more patients. While pharmaceutical companies and contract development and manufacturing organizations are working to fill that gap, a shortage of manufacturing capacity and talent is exacerbated by logistical issues. The sensitivity of supply chains was highlighted during the COVID-19 crisis, when two-thirds of cell therapy companies surveyed reported supply chain disruptions because of travel restrictions.
Integrate your strategic forecasting and planning early in the process and time the launch excellence exercise. Often companies do not include this function in the early development cycle and that can cause misalignment of revenue or demand numbers. If the forecasting and strategic team is involved, they can incorporate the changes (say, expedited approval or expanded access, etc.) into the model to run the revised numbers and scope of the business opportunity. This will also lead to alignment in the process and across your organization.
Weaving Your Strategy Together
Insights, forecasting, and analytics will ultimately drive the understanding and adoption of the best practices in the success of CGT. The above points are imperative to drive and understand the realistic scenarios and get ROI in this space.
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.
All the views and opinions expressed are fully independent and belong to the author only. They do not represent the views or opinions of the author's employer or organization.
About The Author:
Sanobar Syed, M.Sc., MBA, is associate director of market insights and strategy at BeiGene. She has more than 14 years of experience in establishing and leading business strategy and forecasting in leading global pharmaceutical firms. She regularly speaks at industry conferences and guest lectures, and has developed academic modules in this field. She has a master’s in organic chemistry and an MBA in marketing.