Effective Medical Communications During CGT Clinical Trials

By Kimberly Cash and Kyle Felmet, Two Labs

As the cell and gene therapy market has exponentially grown in recent years — with some reports predicting it will reach $24.4 billion with 54 to 74 approvals by 2030 — many manufacturers are learning the hard way just how complex it can be to launch these products, and that’s after already making it through the arduous development and clinical trial process.

Compared to traditional prescription products, cell and gene therapies present several unique challenges and opportunities. What sets the stage for much of how the launch plan is put together is the size of the company, often small, which results in constrained budget and capacity resources. That is intensified by additional budget and cost concerns because of the complex nature of the cell and gene therapies themselves.

In this space, each therapy has a unique product journey. Different from traditional prescription products, cell and gene therapies often require direct distribution to the site of care. Because of the growing cell and gene therapy market, we’re seeing a growing need for specialty pharmacy utilization, especially with the anticipated ability for outpatient administration for several pipeline cell and gene therapies. Another unique challenge of cell and gene therapies, ironically, is the unprecedented success these therapies could potentially provide for patients. These treatments often have long-term durability and may even cure diseases through a one-time administration. This is changing the treatment landscape as we know it, which impacts healthcare providers, payers, and patients. These unique challenges contribute to higher product development costs. The short duration of these therapies compounds the impact of the high cost even more because now the high cost of the specialty discovery and development phase needs to be balanced against the concentrated time of delivery.

All these unique traits of cell and gene therapies translate into multilayered reimbursement, manufacturing, and supply chain complexities that are difficult to sell to investors. In addition to navigating everything above, the manufacturer also must put together an irresistible value proposition to help investors overlook the increased commercial obstacles that the therapies may face, as well as medical communications (medcomms) during CGT clinical trials for patients and healthcare providers that can help crystalize the nuances of cell and gene therapies. Tackling these challenges requires early attention to the medcomms plan, by experts familiar with strategies to build a clear and concise scientific story, to effectively inform stakeholders of the value of the therapy in development.

The Patient Perspective

In contrast to a more traditional therapy, cell and gene therapies require a special focus on patient-facing materials. The primary aims for patient communications are to reduce knowledge gap barriers, provide layperson-level education about the novel MOA and administration requirements, and set therapeutic outcome expectations.

Another unique piece, which brings both advantages and challenges, is that there is a much smaller number of patient candidates for these targeted therapies. This can make assembling patient communications easier as there are fewer variations within a population to navigate, but it is trickier in that the small patient population will be expecting the most tailored communication and messaging that you can offer. This dynamic is also fluid and expected to ebb and flow in the future, as we track pipeline cell and gene therapies that address much broader disease states, such as sickle cell.

Because the clinical pathway is not always clear in the disease areas targeted by cell and gene therapies, patients often have a prolonged and circuitous journey to obtain a correct diagnosis. This results in active and highly networked patient advocacy groups that form to offer support and education to their members. Thus, patient advocacy groups are very important engagement targets, as they can provide a consistent channel for your message. These groups are also a vital source of input to product development, distribution, and administration plans as patient-centricity is a key success factor in cell and gene therapy.

Why Integrate Medcomms Early?

Given the multiple and unique complexities that an emerging manufacturer is managing when launching a cell and gene therapy – costs, patient perspective, transforming therapeutic landscapes, working investors, supply chains, etc., it might sound like medical communications is just one more thing on the list during CGT clinical development. However, that would be a disastrous misconception. Infusing medical communications into your launch plan early on, in late Phase 1/early Phase 2, sets the foundation for success in each part of the plan. Medical communications aren’t just another part of the list; it’s how the rest of your list gets done.

The groundbreaking science of cell and gene therapies is complicated, and without an expert on your team refining the details into digestible, stepwise communications, you can be left with complex messaging that your patients, HCPs, and payers all struggle to understand. When tackling this piece of a plan, there’s a lot to factor in:

• The scientific platform must identify the core data and the resulting messages needed to establish product value relative to the higher costs to put the cost in context.
• Beginning as early as Phase 1, the medical communications outputs need to set the stage for value-based care, to inform key stakeholders who are often still thinking in the traditional reimbursement model framework.
• With regard to rare diseases and cell and gene therapies, treatment guidelines may be lacking or may require a different clinical pathway that needs to be mapped out and updated.
• Since the therapies are newer, the evidence base is not yet as robust as other more traditional options, and the data that is there often requires a specialized expert to interpret. Medical communications strategies and tactics tailored to cell and gene therapy launches make this information easier to understand and apply for HCPs and patients.
• Less competition in the field means a different approach for publication planning (e.g., less need for strategic comparisons with competitor agents, more need for disease awareness and diagnostic approaches).

For emerging manufacturers, all these extra considerations need to be included in a much smaller budget that inherently begs for cutting line items. Recognizing that medical communications are part of the solution for building value at each step of cell and gene therapy development is the bottom-line case for bringing this service in early in a launch strategy. Failing to do so can leave a manufacturer vulnerable to a number of gaps: inconsistent messaging that results in weak product understanding and uptake, lack of evidence of product differentiation for compendia entries, and/or incomplete scientific publication planning that won’t convey the complexities of the therapy to HCPs, payers, and patients. Large pharma has greater budget and resources to address these gaps in hindsight, which are often more costly as compared to proactively addressing them up front, due to lost opportunities at launch. Such costs weigh more heavily on an emerging manufacturer and can jeopardize a successful product launch with unforeseen costs that are not covered by more streamlined budgets.

How To Build An Effective Medcomms Strategy

To make the most of adding medcomms to your strategy, initiate it early in your cell and gene therapy clinical development journey. Think of it as a two-way function that synthesizes internal and external information into a consistent message that will inform product development, as well as educate external stakeholders as your product’s story builds.

The team behind these efforts should include experts across disciplines to ensure a comprehensive view of the product development objectives, while also building lines of open communication to effectively facilitate collaboration. As you build this team, consider bringing in representatives from the drug discovery, clinical development, medical affairs, commercial strategy, and regulatory teams to provide the variety of unique perspectives that a medcomms strategy needs to be successful.

In the earlier stages, your medcomms efforts should be focused on assessing your product’s therapeutic area landscape, relevant standards of care, patient journeys, and scientific context to identify gaps and inform the detailed elements of the communication plan. These plans should then be routinely reviewed and revised to reflect the internal and external influences in the therapeutic area and the general scientific space. These changes might include new data or competitors.

This process of continual review and revision should continue to and through the product’s launch, with medcomms strategies that are built to anticipate changes and quickly pivot to address any stakeholder knowledge gaps that surface after the product becomes commercially available. Specific to the cell and gene therapy sector, this often includes communication of post-approval studies that address real-world use scenarios, as well as late-breaking updates that might result from conference or congress proceedings.

Cell and gene therapies are proving to be game-changers in the lab, in the market, and in the healthcare provider's office, where they are changing patients' lives. Make sure that yours is set up for success by including medical communications in your launch plan from the very beginning.

About The Authors:

Kimberly Cash is a general manager of communications at Two Labs. She works with biotech/biopharma companies to deliver strategic medical affairs tactics and impactful strategic medical communications. Cash has led medical affairs development at small to large pharmaceutical companies.

Kyle Felmet is vice president of specialty commercialization at Two Labs. He has supported clients in the oncology, rare disease, and cell and gene therapy spaces for more than 10 years.